Biotech Stocks

Novo Nordisk A/S, a healthcare company, engages in the research, development, manufacture, and marketing of pharmaceutical products worldwide. It operates in two segments, Diabetes and Obesity care, and Biopharm. The Diabetes and Obesity care segment provides products in the areas of insulins, GLP-1 and related delivery systems, oral antidiabetic products, obesity, and other chronic diseases. The Biopharmaceuticals segment offers products in the areas of haemophilia, growth disorders, and hormone replacement therapy. The company collaboration agreements with Gilead Sciences, Inc. Novo Nordisk A/S also has a research collaboration with Lumen Bioscience, Inc. to explore strategies for delivering oral biologics for cardiometabolic disease. The company was founded in 1923 and is headquartered in Bagsvaerd, Denmark.

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 6 years of age or older who have at least one F508del mutation. Its pipeline includes VX-864 for the treatment of AAT deficiency, which is in Phase 2 clinical trial; VX-147 for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases which is in Phase 2 clinical trial; VX- 880, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-548, a NaV1.8 inhibitor for treatments of acute, neuropathic, musculoskeletal pain which is in Phase 2 clinical trial; and CTX001 for the treatment severe SCD and TDT which is in Phase 3 clinical trial. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. It has collaborations with Affinia Therapeutics, Inc.; Arbor Biotechnologies, Inc.; CRISPR Therapeutics AG.; Kymera Therapeutics, Inc.; Mammoth Biosciences, Inc.; Moderna, Inc.; Obsidian Therapeutics, Inc.; and Skyhawk Therapeutics, Inc.; as well as Ribometrix, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany, and X-Chem, Inc. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

Regeneron Pharmaceuticals, Inc. discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide. The company's products include EYLEA injection to treat wet age-related macular degeneration and diabetic macular edema; myopic choroidal neovascularization; and diabetic retinopathy, as well as macular edema following retinal vein occlusion, including macular edema following central retinal vein occlusion and macular edema following branch retinal vein occlusion. It also provides Dupixent injection to treat atopic dermatitis and asthma in adults and pediatrics; Libtayo injection to treat metastatic or locally advanced cutaneous squamous cell carcinoma;Praluent injection for heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in adults; REGEN-COV for covid-19; and Kevzara solution for treating rheumatoid arthritis in adults. In addition, the company offers Inmazeb injection for infection caused by Zaire ebolavirus; ARCALYST injection for cryopyrin-associated periodic syndromes, including familial cold auto-inflammatory syndrome and muckle-wells syndrome; and ZALTRAP injection for intravenous infusion to treat metastatic colorectal cancer; and develops product candidates for treating patients with eye, allergic and inflammatory, cardiovascular and metabolic, infectious, and rare diseases; and cancer, pain, and hematologic conditions. It has collaboration and license agreements with Sanofi; Bayer; Teva Pharmaceutical Industries Ltd.; Mitsubishi Tanabe Pharma Corporation; Alnylam Pharmaceuticals, Inc.; Roche Pharmaceuticals; and Kiniksa Pharmaceuticals, Ltd., as well as has an agreement with the U.S. Department of Health and Human Services, as well as with Zai Lab Limited; Intellia Therapeutics, Inc.; Biomedical Advanced Research Development Authority; and AstraZeneca PLC. The company was incorporated in 1988 and is headquartered in Tarrytown, New York.

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases. Its marketed products include ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria (AHP); and OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1). In addition, the company is developing givosiran for the treatment of adolescent patients with AHP; patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; ALN-AAT02 for the treatment of AAT deficiency-associated liver disease; ALN-HBV02 to treat chronic HBV infection; Zilebesiran to treat hypertension; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia and rare bleeding disorders, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1 and recurrent renal stones, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Sanofi Genzyme to discover, develop, and commercialize RNAi therapeutics. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

argenx SE, a biotechnology company, focuses on developing various therapies for the treatment of autoimmune diseases in the United States, the Netherlands, Belgium, Japan, Switzerland, Germany and France. Its lead product candidate is efgartigimod for the treatment of patients with myasthenia gravis, immune thrombocytopenia, pemphigus vulgaris, and chronic inflammatory demyelinating polyneuropathy in Phase III clinical trials; bullous pemphigoid and idiopathic inflammatory myopathy in Phase II/III clinical trials; and ENHANZE SC in Pre-clinical study. The company is also developing immunology innovation programs, including cusatuzumab for hematological cancer, as well as high risk MDS; ARGX-119, an antibody that targets muscle-specific tyrosine kinase stage; ARGX-117 for severe autoimmune indications; ARGX-118 for airway inflammation; and ARGX-120 to treat autoimmune diseases. In addition, its partnered product candidates include ARGX-115 for the treatment of cancer; ARGX-112 to treat interleukin-22 receptor; and ARGX-114, an antibody directed against the MET receptor. The company has strategic partnership with AbbVie S.À.R.L., Zai Lab Limited, and LEO Pharma A/S; and collaboration and license agreement with Genor Biopharma Co. Ltd, Université Catholique de Louvain, Sopartec S.A., NYU Langone Health, Leiden University Medical Center, AgomAb Therapeutics NV, Broteio Pharma B.V., University of Texas, BioWa, Inc., Cilag GmbH International, Staten Biotechnology B.V., and Shire International GmbH. argenx SE was incorporated in 2008 and is based in Breda, the Netherlands.

BioNTech SE, a biotechnology company, develops and commercializes immunotherapies for cancer and other infectious diseases. The company is developing FixVac product candidates, including BNT111, which is in Phase II clinical trial for advance melanoma; BNT112 that is in Phase I/IIa clinical trial for prostate cancer; BNT113, which is in Phase II clinical trial to treat HPV+ head and neck cancers; BNT114 that is in Phase I clinical trial for triple negative breast cancer; BNT115, which is in Phase I clinical trial in ovarian cancer; and BNT116, a preclinical stage product for non-small cell lung cancer. It also develops neoantigen specific immunotherapies, such as Autogene cevumeran (BNT122), which is in Phase II clinical trial for first-line melanoma, as well as in Phase 1a/1b clinical trial to treat multiple solid tumors; mRNA intratumoral immunotherapy comprising SAR441000 that is in Phase I clinical trial for solid tumors; and BNT141 and BNT142 that are in Phase I clinical trial to treat multiple solid tumors. In addition, the company develops RiboCytokines, which include BNT151, BNT152, and BNT153 to treat solid tumors; chimeric antigen receptor T cell immunotherapies, such as BNT211 to treat multiple solid tumors, and BNT221 for other cancers; and checkpoint immunomodulators consisting of GEN1046 and GEN1042, which are in Phase I/II clinical trial to treat solid tumors. Further, it develops BNT321, an IgG1 monoclonal antibody in Phase II clinical trial for pancreatic cancer; BNT411, a small molecule immunomodulator product candidate for solid tumors; prophylactic vaccine for COVID-19 and Influenza; and infectious disease immunotherapies and rare disease protein replacement therapies. The company has collaborations with Genentech, Inc.; Sanofi S.A.; Genmab A/S; Pfizer Inc.; Shanghai Fosun Pharmaceutical (Group) Co., Ltd.; and Regeneron Pharmaceuticals, Inc. BioNTech SE was incorporated in 2008 and is headquartered in Mainz, Germany.

Moderna, Inc., a biotechnology company, discovers, develops, and commercializes messenger RNA therapeutics and vaccines for the treatment of infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases, and auto-immune diseases in the United States, Europe, and internationally. Its respiratory vaccines include COVID-19, flu, respiratory syncytial virus, Endemic HCoV, and hMPV+PIV3 vaccines; latent vaccines comprise cytomegalovirus, epstein-barr virus, human immunodeficiency virus, herpes simplex virus, and varicella-zoster virus vaccines; and public health vaccines consists of Zika and Nipah vaccines. The company also offers systemic secreted and cell surface therapeutics; cancer vaccines, such as personalized cancer, KRAS, and checkpoint vaccines; intratumoral immuno-oncology products; localized regenerative, systemic intracellular, and inhaled pulmonary therapeutics. It has strategic alliances with AstraZeneca PLC; Merck & Co., Inc.; Vertex Pharmaceuticals Incorporated; Vertex Pharmaceuticals (Europe) Limited; Carisma Therapeutics, Inc.; Metagenomi, Inc.; the Defense Advanced Research Projects Agency; Biomedical Advanced Research and Development Authority; Institute for Life Changing Medicines; and The Bill & Melinda Gates Foundation, as well as a collaboration and license agreement with Chiesi Farmaceutici S.P.A. The company was formerly known as Moderna Therapeutics, Inc. and changed its name to Moderna, Inc. in August 2018. Moderna, Inc. was founded in 2010 and is headquartered in Cambridge, Massachusetts.

BeiGene, Ltd., a biotechnology company, focuses on discovering, developing, manufacturing, and commercializing various medicines worldwide. Its products include BRUKINSA to treat relapsed/refractory (R/R) mantle cell lymphoma; Tislelizumab to treat R/R classical Hodgkin's lymphoma; REVLIMID to treat multiple myeloma; VIDAZA to treat myelodysplastic syndromes, chronic myelomonocyte leukemia, and acute myeloid leukemia; XGEVA to treat giant cell tumor of bone; BLINCYTO to treat acute lymphoblastic leukemia; KYPROLIS to treat R/R multiple myeloma; SYLVANT to treat idiopathic multicentric castleman disease; QARZIBA to treat neuroblastoma; Pamiparib for the treatment of various solid tumors; and Pobevcy to treat metastatic colorectal cancer, liver cancer, and non-small cell lung cancer (NSCLC). The company's clinical stage drug candidates comprise Zanubrutinib, a BTK inhibitor to treat lymphomas; Tislelizumab, an anti-PD-1 antibody to treat solid and hematological cancers; Lifirafenib and BGB-3245 to treat melanoma, NSCLC, and endometrial cancer; and Sitravatinib, a multi-kinase inhibitor to treat NSCLC, melanoma, and other solid tumors. Its clinical stage drug candidates also include BGB-A333, a PD-L1 inhibitor to treat various solid tumors; Ociperlimab, a TIGIT inhibitor to treat various solid tumors; BGB-11417, a small molecule Bcl-2 inhibitor to treat mature B-cell malignancies; BGB-A445, an OX40 agonist antibody to treat solid tumors; Zanidatamab, a bispecific HER2 inhibitor to treat breast and gastric cancer; BGB-A425, a T-cell immunoglobulin and mucin-domain containing-3 inhibitor to treat various solid tumors; and BGB-15025, a small molecule inhibitor of HPK1. The company has strategic collaborations with Shoreline Biosciences, Inc., Amgen Inc., Novartis AG, and Bristol Myers Squibb company. BeiGene, Ltd. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.

United Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. Its commercial therapies include Remodulin to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Tyvaso, an inhaled formulation of prostacyclin analogue treprostinil to enhance the exercise ability in PAH patients and pulmonary hypertension associated with interstitial lung disease (PH-ILD); Orenitram, a tablet dosage form of treprostinil to enhance the exercise capacity in PAH patients; Unituxin, a monoclonal antibody for treating high-risk neuroblastoma; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. The company also engages in developing Tyvaso DPI, a dry powder inhalation form of Tyvaso; Remunity Pump, a small, lightweight, durable pump and separate controller; RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Tyvaso PERFECT and TETON studies, which are the studies of Tyvaso in patients with World Health Organization (WHO) Group 3 pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD). It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.

Summit Therapeutics Inc., a biopharmaceutical company, discovers, develops, and commercializes medicines to treat infectious diseases in the United States and Latin America. It conducts clinical programs focusing on Clostridioides difficile infection (CDI). The company's lead product candidate is ridinilazole, an orally administered small molecule antibiotic that is in Phase III clinical trials for the treatment of CDI. It also offers SMT-738, for combating multidrug resistant infections primarily carbapenem-resistant Enterobacteriaceae infections; and DDS-04 series for the potential treatment of infections caused by the Enterobacteriaceae. The company was founded in 2003 and is based in Cambridge, Massachusetts.

Genmab A/S develops antibody therapeutics for the treatment of cancer and other diseases primarily in Denmark. The company markets DARZALEX, a human monoclonal antibody for the treatment of patients with multiple myeloma (MM); teprotumumab for the treatment of thyroid eye disease; ofatumurnab, a human monoclonal antibody to treat chronic lymphocytic leukemia (CLL) and multiple sclerosis; and Amivantamab for advanced or metastatic gastric or esophageal cancer and NSCLC. Its products include daratumumab to treat MM, non-MM blood cancers, and AL amyloidosis; GEN1047; tisotumab vedotin for treating cervical, ovarian, and solid cancers; DuoBody-PD-L1x4-1BB, and DuoBody-CD40x4-1BB for treating solid tumors; Epcoritamab for relapsed/refractory diffuse large B-cell lymphoma and chronic lymphocytic leukemia; and HexaBody-CD38 and DuoHexaBody-CD37 for treating hematological malignancies. The company's also develops products, which is in Phase 2 comprise Teclistamab for vaso-occlusive crises; Camidanlumab tesirine to treat hodgkin lymphoma and solid tumors; JNJ-64007957 and JNJ-64407564 to treat MM; PRV-015 for treating celiac disease; Mim8 for treating haemophilia A; and Lu AF82422 for treating multiple system atrophy disease. In addition, it has approximately 20 active pre-clinical programs. The company has a commercial license and collaboration agreement with Seagen Inc. to co-develop tisotumab vedotin. It also has a collaboration agreement with CureVac AG for the research and development of differentiated mRNA-based antibody products; AbbVie for the development of epcoritamab; and collaborations with BioNTech, Janssen, Novo Nordisk A/S, BliNK Biomedical SAS, and Bolt Biotherapeutics, Inc. Genmab A/S was founded in 1999 and is headquartered in Copenhagen, Denmark.

Vaxcyte, Inc., a clinical-stage biotechnology vaccine company, develops novel protein vaccines to prevent or treat bacterial infectious diseases. Its lead vaccine candidate is VAX-24, a 24-valent investigational pneumococcal conjugate vaccine that is in Phase 1/2 clinical trials to treat invasive pneumococcal disease and pneumonia. The company also develops VAX-XP to protect against emerging strains and address antibiotic resistance; VAX-A1, a conjugate vaccine candidate designed to treat Group A Strep; and VAX-PG, a novel protein vaccine candidate targeting keystone pathogen responsible for periodontitis. The company was formerly known as SutroVax, Inc. and changed its name to Vaxcyte, Inc. in May 2020. Vaxcyte, Inc. was incorporated in 2013 and is headquartered in San Carlos, California.

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Incyte Corporation, a biopharmaceutical company, focuses on the discovery, development, and commercialization of proprietary therapeutics in the United States and internationally. The company offers JAKAFI, a drug for the treatment of myelofibrosis and polycythemia vera; PEMAZYRE, a fibroblast growth factor receptor kinase inhibitor that act as oncogenic drivers in various liquid and solid tumor types; and ICLUSIG, a kinase inhibitor to treat chronic myeloid leukemia and philadelphia-chromosome positive acute lymphoblastic leukemia. Its clinical stage products include ruxolitinib, a steroid-refractory chronic graft-versus-host-diseases (GVHD); itacitinib, which is in Phase II/III clinical trial to treat naïve chronic GVHD; and pemigatinib for treating bladder cancer, cholangiocarcinoma, myeloproliferative syndrome, and tumor agnostic. In addition, the company engages in developing Parsaclisib, which is in Phase II clinical trial for follicular lymphoma, marginal zone lymphoma, and mantel cell lymphoma. Additionally, it develops Retifanlimab that is in Phase II clinical trials for MSI-high endometrial cancer, merkel cell carcinoma, and anal cancer, as well as in Phase II clinical trials for patients with non-small cell lung cancer. It has collaboration agreements with Novartis International Pharmaceutical Ltd.; Eli Lilly and Company; Agenus Inc.; Calithera Biosciences, Inc; MacroGenics, Inc.; Merus N.V.; Syros Pharmaceuticals, Inc.; Innovent Biologics, Inc.; Zai Lab Limited; Cellenkos, Inc.; and Nimble Therapeutics, as well as clinical collaborations with MorphoSys AG and Xencor, Inc. to investigate the combination of tafasitamab, plamotamab, and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma, and relapsed or refractory follicular lymphoma. The company was incorporated in 1991 and is headquartered in Wilmington, Delaware.

Bio-Techne Corporation, together with its subsidiaries, develops, manufactures, and sells life science reagents, instruments, and services for the research and clinical diagnostic markets worldwide. The company operates through two segments, Protein Sciences, and Diagnostics and Genomics. The Protein Sciences segment develops and manufactures biological reagents used in various aspects of life science research, diagnostics, and cell and gene therapy, such as cytokines and growth factors, antibodies, small molecules, tissue culture sera, and cell selection technologies. This segment also offers proteomic analytical tools for automated western blot and multiplexed ELISA workflow consists of manual and automated protein analysis instruments and immunoassays for use in quantifying proteins in various biological fluids. The Diagnostics and Genomics segment develops and manufactures diagnostic products, including controls, calibrators, and diagnostic assays for regulated diagnostics market, exosome-based molecular diagnostic assays, advanced tissue-based in-situ hybridization assays for spatial genomic and tissue biopsy analysis, and genetic and oncology kits for research and clinical applications; and sells products for genetic carrier screening, oncology diagnostics, molecular controls, and research, as well as instruments and process control products for hematology, blood chemistry and gases, and coagulation controls and reagents used in various diagnostic applications. It offers its products under R&D Systems, Tocris Biosciences, Novus Biologicals, ProteinSimple, Advanced Cell Diagnostics, Exosome Diagnostics, and Asuragen brands. The company was formerly known as Techne Corporation and changed its name to Bio-Techne Corporation in November 2014. Bio-Techne Corporation was founded in 1976 and is headquartered in Minneapolis, Minnesota.

Royalty Pharma plc operates as a buyer of biopharmaceutical royalties and a funder of innovations in the biopharmaceutical industry in the United States. It is also involved in the identification, evaluation, and acquisition of royalties on various biopharmaceutical therapies. In addition, the company collaborates with innovators from academic institutions, research hospitals and not-for-profits, small and mid-cap biotechnology companies, and pharmaceutical companies. Its portfolio consists of royalties on approximately 35 marketed therapies and 10 development-stage product candidates that address various therapeutic areas, such as rare disease, cancer, neurology, infectious disease, hematology, and diabetes. The company was founded in 1996 and is based in New York, New York.

Karuna Therapeutics, Inc., a clinical-stage biopharmaceutical company, creates and delivers transformative medicines for people living with psychiatric and neurological conditions. Its lead product candidate is KarXT, an oral modulator of muscarinic receptors that is in Phase III clinical trial for the treatment of acute psychosis in patients with schizophrenia; and for the treatment of central nervous system disorders, such as negative and cognitive symptoms of schizophrenia and psychosis, as well as for the treatment of dementia-related psychosis. The company also focuses on developing other muscarinic-targeted drug candidates. Karuna Therapeutics, Inc. has a license agreement with Eli Lilly and Company, and Zai Lab (Shanghai) Co., Ltd; patent license agreement with PureTech Health LLC; and drug discovery partnership with Charles River Laboratories, as well as drug discovery collaboration with PsychoGenics, Inc. The company was formerly known as Karuna Pharmaceuticals, Inc. and changed its name to Karuna Therapeutics, Inc. in March 2019. Karuna Therapeutics, Inc. was incorporated in 2009 and is headquartered in Boston, Massachusetts.

Insmed Incorporated, a biopharmaceutical company, develops and commercializes therapies for patients with serious and rare diseases. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It is also developing Brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1 for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases; and Treprostinil Palmitil Inhalation Powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil for the treatment of pulmonary arterial hypertension and other rare pulmonary disorders. Insmed Incorporated was founded in 1988 and is headquartered in Bridgewater, New Jersey.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; and VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. The company is also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

Prometheus Biosciences, Inc., a biopharmaceutical company, engages in the discovery, development, and commercialization of novel therapeutics and companion diagnostics products for the treatment of inflammatory bowel diseases (IBD). Its lead product includes PRA023, a humanized IgG1 monoclonal antibody (mAb), which is in Phase IIa clinical trial for the treatment of ulcerative colitis and Crohn's disease, as well as systemic sclerosis-associated interstitial lung disease. The company also develops PR600, an anti-tumor necrosis factor mAb for IBD; PR300, a G-protein coupled receptor modulator small molecule for IBD; PR1100, an anti-cytokine receptor mAb for IBD and other immune-mediated diseases; PR1800, an anti-chemokine mAb for IBD; and PR2100, an anti-inflammatory cytokine mAb for IBD. It has a diagnostics development and collaboration agreement with Takeda Pharmaceutical Company Limited; co-development and manufacturing agreement with Dr. Falk Pharma GmbH; license agreement with Cedars-Sinai Medical Center; and strategic collaboration with Millennium Pharmaceuticals, Inc. The company was formerly known as Precision IBD, Inc. and changed its name to Prometheus Biosciences, Inc. in October 2019. Prometheus Biosciences, Inc. was incorporated in 2016 and is headquartered in San Diego, California.

Legend Biotech Corporation, a clinical-stage biopharmaceutical company, through its subsidiaries, engages in the discovery and development of novel cell therapies for oncology and other indications in the United States, China, and internationally. Its lead product candidate, LCAR- B38M, is a chimeric antigen receptor for the treatment of multiple myeloma (MM), as well as a comparison of the treatment with standard triplet therapy in revlimid-refractory multiple myeloma. The company also has a portfolio of earlier-stage autologous product candidates, which are in Phase I clinical trials for the treatment of gastric cancer and T cell lymphoma. In addition, it is developing CAR-T product candidates targeting CD20/CD22/CD19 for the treatment of non-hodgkins lymphoma, diffuse large B-cell lymphoma, and acute lymphoblastic leukemia. Further, the company has product candidates in early preclinical and clinical development for the treatment of solid tumors as well as infectious diseases. It has collaboration and license agreement with Janssen Biotech, Inc. for the development and commercialization of ciltacabtagene autoleucel. Legend Biotech Corporation was founded in 2014 and is based in Somerset, New Jersey. Legend Biotech Corporation is a subsidiary of Genscript Biotech Corporation.

Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapeutics for unmet medical needs. The company offers SKYTROFA for treating patients with growth hormone deficiency (GHD). It also develops TransCon Growth Hormone (hGH) for pediatric GHD in Japan; TransCon hGH for adults with GHD; TransCon parathyroid hormone for adult hypoparathyroidism; and TransCon CNP for pediatric achondroplasia. In addition, the company develops TransCon toll like receptors 7/8 agonist for intratumoral delivery; and TransCon IL-2 ß/g for systemic delivery. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Roivant Sciences Ltd., a biopharmaceutical and healthcare technology company that researches and develops medicines. The company develops product candidates for the treatment of various therapeutics, including solid tumors, sickle cell diseases, hypophosphatasia, oncologic malignancies, psoriasis, atopic dermatitis, vitiligo, hyperhidrosis, acne, myasthenia gravis, warm autoimmune hemolytic anemia, thyroid eye diseases, sarcoidosis, and staph aureus bacteremia. The company was founded in 2014 and is based in London, the United Kingdom.

Cerevel Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, engages in the development of various therapies for neuroscience diseases. It is developing emraclidine, a positive allosteric modulator (PAM) that is in phase 1b clinical trials for the treatment of schizophrenia; and Darigabat, a PAM, which is in Phase 2 proof-of-concept trial in patients with drug-resistant focal onset seizures in epilepsy or focal epilepsy, as well as in phase 1 trial to treat acute anxiety. The company's products also comprise Tavapadon, a selective dopamine D1/D5 partial agonist that is in phase 3 clinical trial for the treatment of early- and late-stage Parkinson's disease; CVL-871, a selective dopamine D1/D5 partial agonist, which is in Phase 2a clinical trial to treat dementia-related apathy; CVL-936, a selective dopamine D3-preferring antagonist, which is in Phase I clinical trial for the treatment of substance use disorder; CVL-354, a selective kappa-opioid receptor antagonist to treat major depressive disorder and substance use disorder; and CVL-047, selective PDE4 inhibitor for the treatment of major depressive disorder and substance use disorder. It is also involved in the development of an M4 agonist program for the treatment of psychosis and related indications; and an LRRK2 inhibitor program to address disease progression in Parkinson's. The company was founded in 2018 and is headquartered in Cambridge, Massachusetts.

Revolution Medicines, Inc., a clinical-stage precision oncology company, focuses on developing therapies to inhibit frontier targets in RAS-addicted cancers. The company is developing RMC-4630, an inhibitor of SHP2, which is in Phase 1/2 clinical trial for the treatment of solid tumors, such as gynecologic and colorectal cancer tumors. It also develops RMC-5845, a selective inhibitor of SOS1, a protein that converts RAS (OFF) to RAS (ON) in cells; and RMC-5552, a hyperactivated selective inhibitor of mTORC1 signaling in tumors. In addition, the company is developing RMC-6291, a mutant-selective inhibitor of KRASG12C(ON) and NRASG12C(ON); and RMC-6236, a RAS-selective inhibitor of multiple RAS(ON) variants. Further, it develops RAS(ON) Inhibitors targeting KRASG13C(ON) and KRASG12D(ON). The company has a collaboration agreement with Sanofi for the research and development of SHP2 inhibitors, including RMC-4630. Revolution Medicines, Inc. was incorporated in 2014 and is headquartered in Redwood City, California.

Exelixis, Inc., an oncology-focused biotechnology company, focuses on the discovery, development, and commercialization of new medicines to treat cancers in the United States. The company's products include CABOMETYX tablets for the treatment of patients with advanced renal cell carcinoma who received prior anti-angiogenic therapy; and COMETRIQ capsules for the treatment of patients with progressive and metastatic medullary thyroid cancer. Its CABOMETYX and COMETRIQ are derived from cabozantinib, an inhibitor of multiple tyrosine kinases, including MET, AXL, RET, and VEGF receptors. The company also offers COTELLIC, an inhibitor of MEK as a combination regimen to treat advanced melanoma; and MINNEBRO, an oral non-steroidal selective blocker of the mineralocorticoid receptor for the treatment of hypertension in Japan. In addition, it is developing XL092, an oral tyrosine kinase inhibitor that targets VEGF receptors, MET, AXL, MER, and other kinases implicated in growth and spread of cancer; XB002, an antibody-drug conjugate composed of human mAb against tissue factor (TF) for the treatment of advanced solid tumors; XL102, an orally bioavailable cyclin-dependent kinase 7 (CDK7) inhibitor for the treatment of advanced or metastatic solid tumors; and XB002 for the treatment of non-hodgkin's lymphoma. Exelixis, Inc. has research collaborations and license agreements with Ipsen Pharma SAS; Takeda Pharmaceutical Company Ltd.; F. Hoffmann-La Roche Ltd.; Redwood Bioscience, Inc.; R.P. Scherer Technologies, LLC; Catalent Pharma Solutions, Inc.; NBE Therapeutics AG; Aurigene Discovery Technologies Limited; Iconic Therapeutics, Inc.; Invenra, Inc.; StemSynergy Therapeutics, Inc.; Genentech, Inc.; Bristol-Myers Squibb Company; and Daiichi Sankyo Company, Limited. The company was formerly known as Exelixis Pharmaceuticals, Inc. and changed its name to Exelixis, Inc. in February 2000. Exelixis, Inc. was incorporated in 1994 and is headquartered in Alameda, California.

Halozyme Therapeutics, Inc. operates as a biopharma technology platform company in the United States, Switzerland, Ireland, Belgium, Japan, and internationally. The company's products are based on the ENHANZE drug delivery technology, a patented recombinant human hyaluronidase enzyme (rHuPH20) that enables the subcutaneous delivery of injectable biologics, such as monoclonal antibodies and other therapeutic molecules, as well as small molecules and fluids. Its flagship product is Hylenex recombinant, a formulation of rHuPH20 to facilitate subcutaneous fluid administration for achieving hydration to enhance the dispersion and absorption of other injected drugs in subcutaneous urography and to improve resorption of radiopaque agents. The company also develops Perjeta; RITUXAN HYCELA and MabThera SC for the treatment of non-Hodgkin lymphoma and chronic lymphocytic leukemia (CLL); RITUXAN SC for patients with CLL; and HYQVIA for the treatment of immunodeficiency disorders. In addition, it is developing Tecentriq for non-small cell lung cancer; OCREVUS for multiple sclerosis; DARZALEX for the treatment of patients with amyloidosis, smoldering myeloma, and multiple myeloma; nivolumab for the treatment of solid tumors; ARGX-113, a human neonatal Fc receptor; ARGX-117 to treat autoimmune diseases; and BMS-986179, an anti-CD-73 antibody. The company has collaborations with F. Hoffmann-La Roche, Ltd.; Hoffmann-La Roche, Inc.; Baxalta US Inc.; Baxalta GmbH; Pfizer Inc.; Janssen Biotech, Inc.; AbbVie, Inc.; Eli Lilly and Company; Bristol-Myers Squibb Company; Alexion Pharma Holding; ARGENX BVBA; Horizon Therapeutics plc; National Institute of Allergy and Infectious Diseases; Centre for the AIDS Programme of Research in South Africa; and ViiV Healthcare Limited for small and large molecule targets for the treatment and prevention of HIV. Halozyme Therapeutics, Inc. was founded in 1998 and is based in San Diego, California.

Nuvalent, Inc., a clinical stage biopharmaceutical company, develops therapies for patients with cancer. Its lead product candidates are NVL-520, a brain-penetrant ROS1-selective inhibitor to inhibit ROS1 fusions that express the normal ROS1 kinase domain without any drug-resistant mutations and remain active in the presence of mutations conferring resistance to approved and investigational ROS1 inhibitors, which is under Phase I development; and NVL-655, a brain-penetrant ALK-selective inhibitor, to address the clinical challenges of emergent treatment resistance, central nervous system-related adverse events, and brain metastases that might limit the use of first-, second-, and third-generation ALK inhibitors that is under Phase I/II clinical trial. The company was incorporated in 2017 and is headquartered in Cambridge, Massachusetts.

Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapies for metabolic and endocrine disorders. The company's lead drug candidate is VK2809, an orally available tissue and receptor-subtype selective agonist of the thyroid hormone receptor beta (TRß), which is in Phase IIb clinical trials to treat patients with biopsy-confirmed non-alcoholic steatohepatitis, as well as NAFLD. It also develops VK5211, an orally available non-steroidal selective androgen receptor modulator that is in Phase II clinical trials for the treatment of patients recovering from non-elective hip fracture surgery; VK0612, an orally available Phase IIb-ready drug candidate for type 2 diabetes; and VK0214, an orally available tissue and receptor-subtype selective agonist of the TRß for X-linked adrenoleukodystrophy. The company was incorporated in 2012 and is headquartered in San Diego, California.

Jazz Pharmaceuticals plc, a biopharmaceutical company, identifies, develops, and commercializes pharmaceutical products for various unmet medical needs in the United States, Europe, and internationally. The company has a portfolio of products and product candidates with a focus in the areas of neuroscience, including sleep medicine and movement disorders; and in oncology, including hematologic and solid tumors. Its lead marketed products include Xyrem, an oral solution for the treatment of cataplexy and excessive daytime sleepiness (EDS) in narcolepsy patients seven years of age and older; Sunosi for the treatment of EDS in patients with narcolepsy and obstructive sleep apnea; Erwinaze to treat acute lymphoblastic leukemia; Defitelio for the treatment of adult and pediatric patients with hepatic veno-occlusive disease; Vyxeos liposome for injection, a product for the treatment of adults with newly-diagnosed therapy-related acute myeloid leukemia; and Zepzelca for the treatment of adult patients with metastatic small cell lung cancer. The company also offers Xywav, an oxybate product candidate, to treat EDS and cataplexy with narcolepsy and idiopathic hypersomnia; JZP-324, a low sodium oxybate formulation with the potential to provide a clinically meaningful option for narcolepsy patients; JZP385, a T-type calcium channel modulator, for the treatment of essential tremor; JZP458, a recombinant Erwinia asparaginase, for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients; and JZP150 for treatment of post-traumatic stress disorder. The company has licensing and collaboration agreements with ImmunoGen, Inc.; Codiak BioSciences, Inc.; Pfenex, Inc.; XL-protein GmbH; and Redx Pharma plc. Jazz Pharmaceuticals plc was incorporated in 2003 and is headquartered in Dublin, Ireland.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of TTR amyloidosis; Olezarsen for patients with severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Tofersen to inhibit the production of superoxide dismutase 1. In addition, the company develops medicines for metabolic diseases, infectious diseases, renal diseases, ophthalmic diseases, and cancer. It has a strategic collaboration with Biogen Inc.; and collaboration and license agreement with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

Cytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases. The company develops small molecule drug candidates primarily engineered to impact muscle function and contractility. Its drug candidates include omecamtiv mecarbil, a novel cardiac myosin activator that is in Phase III clinical trial in patients with heart failure; and reldesemtiv, a skeletal muscle troponin activator, which is in Phase III clinical trial to treat amyotrophic lateral sclerosis and spinal muscular atrophy. The company also develops CK-136, a novel cardiac troponin activator that is in Phase I clinical trial; aficamten, a novel cardiac myosin inhibitor, which is in Phase III clinical trial for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy; and CK-3772271, a small molecule cardiac myosin inhibitor that is in Phase I clinical trial. Cytokinetics, Incorporated has a strategic alliance with Astellas Pharma Inc. The company was incorporated in 1997 and is headquartered in South San Francisco, California.

Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of non-advanced SM and other mast cell disorders; and Fisogatinib, an orally available and potent inhibitor for the treatment of hepatocellular carcinoma. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered solid tumors, medullary thyroid carcinoma, and other solid tumors; BLU-701 and BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the development of oligonucleotide-based therapies. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat a range of serious diseases. The company's lead product candidate AOC 1001 is used for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease; AOC 1044 for the treatment of Duchenne Muscular Dystrophy; and AOC 1020 designed to treat facioscapulohumeral muscular dystrophy which are in preclinical stage. It also offers Lumizyme therapy for Pompe diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.

Krystal Biotech, Inc., a clinical stage biotechnology company, engages in the field of redosable gene therapy to treat serious rare diseases in the United States. Its lead product candidate is beremagene geperpavec (B-VEC), which is in Phase III clinical study to treat dystrophic epidermolysis bullosa. The company is also involved in developing KB105 that is in Phase I/II clinical study for treating patients with deficient autosomal recessive congenital ichthyosis; KB301, which is in Phase I/II clinical stage for treating wrinkles and other presentations of aged or damaged skin; KB407 that is in preclinical stage for cystic fibrosis; and KB104, which is in preclinical stage for netherton syndrome. Its discovery stage product candidates include KB5xx for treating chronic skin diseases, KB3xx to treat aesthetic skin conditions, and KB3xx product. Krystal Biotech, Inc. was founded in 2015 and is headquartered in Pittsburgh, Pennsylvania.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

Corcept Therapeutics Incorporated discovers, develops, and commercializes drugs for the treatment of severe metabolic, oncologic, and neuropsychiatric disorders in the United States. The company offers Korlym (mifepristone) tablets as a once-daily oral medication for the treatment of hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome, who have type 2 diabetes mellitus or glucose intolerance, and have failed surgery or are not candidates for surgery. It is developing relacorilant to treat patients with Cushing's syndrome; and nab-paclitaxel in combination with relacorilant, which has completed Phase II clinical trial to treat patients with advanced ovarian tumors, as well as for the treatment of cortisol excess. The company is also developing selective cortisol modulator to treat patients with metastatic castration-resistant prostate cancer; selective cortisol modulator for the treatment of antipsychotic-induced weight gain and other disorders; and FKBP5 gene expression assays. Corcept Therapeutics Incorporated was incorporated in 1998 and is headquartered in Menlo Park, California.

BridgeBio Pharma, Inc. engages in the discovery, development, and delivery of various medicines for genetic diseases. The company has a pipeline of 30 development programs that include product candidates ranging from early discovery to late-stage development. Its products in development programs include AG10 and BBP-265, a small molecule stabilizer of transthyretin, or TTR that is in Phase 3 clinical trial for the treatment of TTR amyloidosis-cardiomyopathy, or ATTR-CM; BBP-831, a small molecule selective FGFR1-3 inhibitor, which is Phase 2 clinical trial to treat achondroplasia in pediatric patients; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 2 proof-of-concept clinical trial for Autosomal Dominant Hypocalcemia Type 1, or ADH1; and BBP-711 for the treatment of hyperoxaluria, as well as patients suffering from recurrent kidney stones. In addition, it engages in developing products for Mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and The Regents of the University of California; Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.

Biohaven Pharmaceutical Holding Company Ltd., a biopharmaceutical company, develops products candidates targeting neurological and neuropsychiatric diseases, and rare disorders in the United States. It offers NURTEC ODT (rimegepant) for the acute treatment of migraine, as well as developing Rimegepant for preventive treatment of migraine; Zavegepant that is in phase III clinical trials for acute and preventive treatment of migraine, as well as respiratory complications and non-migraine studies; and BHV-3100 for non-migraine indications. The company also offers Troriluzole for spinocerebellar ataxia and obsessive compulsive disorders, as well as Alzheimer diseases; BHV-0223, a product for amyotrophic lateral sclerosis; BHV-5000/5500 for neuropsychiatric indications; and Verdiperstat, a product for multiple system atrophy and amyotrophic lateral sclerosis. Biohaven Pharmaceutical Holding Company Ltd. has a collaboration agreement with Cove to facilitate telemedicine evaluation for migraine sufferers. The company was incorporated in 2013 and is headquartered in New Haven, Connecticut.

ADMA Biologics, Inc., a biopharmaceutical company, engages in developing, manufacturing, and marketing specialty plasma-derived biologics for the treatment of immune deficiencies and infectious diseases in the United States and internationally. It offers BIVIGAM, an intravenous immune globulin (IVIG) product indicated for the treatment of primary humoral immunodeficiency (PI); ASCENIV, an IVIG product for the treatment of PI; and Nabi-HB for the treatment of acute exposure to blood containing Hepatitis B surface antigen and other listed exposures to Hepatitis B. The company also develops a pipeline of plasma-derived therapeutics, including products related to the methods of treatment and prevention of S. pneumonia infection for an immunoglobulin. In addition, it operates source plasma collection facilities. The company sells its products through independent distributors, sales agents, specialty pharmacies, and other alternate site providers. ADMA Biologics, Inc. was incorporated in 2004 and is headquartered in Ramsey, New Jersey.

Alkermes plc, a biopharmaceutical company, researches, develops, and commercializes pharmaceutical products to address unmet medical needs of patients in various therapeutic areas in the United States, Ireland, and internationally. Its marketed products include ARISTADA, an intramuscular injectable suspension for the treatment of schizophrenia; VIVITROL for the treatment of alcohol and prevention of opioid dependence; RISPERDAL CONSTA for the treatment of schizophrenia and bipolar I disorder; INVEGA SUSTENNA for the treatment of schizophrenia and schizoaffective disorder; XEPLION, INVEGA TRINZA, and TREVICTA to treat schizophrenia and schizoaffective; and VUMERITY for the treatment of relapsing forms of multiple sclerosis in adults, including clinically isolated syndrome, relapsing-remitting and active secondary progressive diseases. The company is also developing LYBALVI, an oral atypical antipsychotic drug candidate for the treatment of adults with schizophrenia and bipolar I disorder; and nemvaleukin alfa, an engineered fusion protein to expand tumor-killing immune cells and to avoid the activation of immunosuppressive cells. It has collaboration agreements primarily with Janssen Pharmaceutica N.V., Janssen Pharmaceutica Inc, and Janssen Pharmaceutica International. Alkermes plc was founded in 1987 and is headquartered in Dublin, Ireland.

Madrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapeutic candidates for the treatment of cardiovascular, metabolic, and liver diseases. Its lead product candidate is resmetirom, a liver-directed selective thyroid hormone receptor-ß agonist, which is in Phase III clinical trials for the treatment of non-alcoholic steatohepatitis. The company also develops MGL-3745, a backup compound to resmetirom. It has research, development, and commercialization agreement with Hoffmann-La Roche. Madrigal Pharmaceuticals, Inc. is headquartered in West Conshohocken, Pennsylvania.

Arcellx, Inc., a clinical-stage biotechnology company, engages in the development of various immunotherapies for patients with cancer and other incurable diseases. The company's lead ddCAR product candidate is CART-ddBCMA, which is in phase 1 clinical trial for the treatment of patients with relapsed or refractory (r/r) multiple myeloma (MM). It is also developing ACLX-001, an immunotherapeutic combination composed of ARC-T cells and bi-valent SparX proteins targeting BCMA to treat r/r MM; ACLX-002 and ACLX-003 for treating r/r acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS); and other AML/MDS product candidates, as well as solid tumor programs. The company was formerly known as Encarta Therapeutics, Inc. and changed its name to Arcellx, Inc. in January 2016. Arcellx, Inc. was incorporated in 2014 and is headquartered in Gaithersburg, Maryland.

Alpine Immune Sciences, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of protein-based immunotherapies for the treatment of cancer, autoimmune/inflammatory disorders, and other diseases. Its product pipeline includes ALPN-101, an inducible T cell costimulator (ICOS)/cluster of differentiation 28 (CD28) antagonist program for the treatment of autoimmune and inflammatory diseases; ALPN-202, a conditional CD28 costimulator and dual checkpoint inhibitor for the treatment of cancer; and ALPN-303, a dual B cell cytokine antagonist for the treatment of B cell-mediated inflammatory and autoimmune diseases. The company has a collaboration agreement with AbbVie Ireland Unlimited Company for the development of ALPN-101; and Adaptimmune Therapeutics plc to develop next-generation SPEAR T cell products. Alpine Immune Sciences, Inc. was incorporated in 2007 and is headquartered in Seattle, Washington.

Axsome Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel therapies for central nervous system (CNS) disorders in the United States. The company's product pipeline includes AXS-05, a therapeutic for the treatment of major depressive disorder and resistant depression disorders; and that is in the Phase III clinical trial to treat Alzheimer's disease agitation, as well as that has completed phase II clinical trial for the treatment of smoking cessation. It is also developing AXS-07, a novel, oral, rapidly absorbed, multi-mechanistic, and investigational medicine that has completed two Phase III trials for the acute treatment of migraine; AXS-12, a selective and potent norepinephrine reuptake inhibitor, which is in Phase III trial to treat narcolepsy; and AXS-14, a novel, oral, and investigational medicine that is in Phase III trial for the treatment of fibromyalgia. Axsome Therapeutics, Inc. has a research collaboration agreement with Duke University for evaluating AXS-05 in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.

Immunovant, Inc., a clinical-stage biopharmaceutical company, develops monoclonal antibodies for the treatment of autoimmune diseases. It develops batoclimab, a novel fully human monoclonal antibody that selectively binds to and inhibits the neonatal fragment crystallizable receptor, which is in Phase IIa clinical trials for the treatment of myasthenia gravis and thyroid eye disease, as well as completed initiation of Phase II clinical trials for the treatment of warm autoimmune hemolytic anemia. The company was incorporated in 2018 is headquartered in New York, New York. Immunovant, Inc. is a subsidiary of Roivant Sciences Ltd.

Denali Therapeutics Inc., a biopharmaceutical company, discovers and develops therapeutic candidates for neurodegenerative diseases in the United States. It offers leucine-rich repeat kinase 2 (LRRK2) inhibitor product candidate, including BIIB122/DNL151, a small molecule inhibitor, which is in phase I and phase Ib clinical trials for the treatment of Parkinson's disease. The company also develops DNL310 that is in Phase I/II clinical trials for the treatment of hunter syndrome; DNL343, which is in phase 1 clinical trial the treatment of amyotrophic lateral sclerosis (ALS); AR443820/DNL788 completed a phase I clinical trial for the treatment of ALS, multiple sclerosis (MS), and Alzheimer's disease; and SAR443122/DNL758, which is in phase II clinical trial for the treatment of cutaneous lupus erythematosus. It has collaboration agreement with Takeda Pharmaceutical Company, Genentech, Inc., Sanofi, F-star Gamma Limited, F-star Biotechnologische Forschungs-Und Entwicklungsges M.B.H, F-star Biotechnology Limited, SIRION Biotech GmbH, Genzyme Corporation, Harvard University, the Michael J. Fox Foundation, and Centogene; and a research and option agreement with Secarna Pharmaceuticals GmbH & Co. KG. to develop antisense therapies in the field of neurodegenerative diseases. The company was formerly known as SPR Pharma Inc. and changed its name to Denali Therapeutics Inc. in March 2015. Denali Therapeutics Inc. was incorporated in 2013 and is headquartered in South San Francisco, California.

Crinetics Pharmaceuticals, Inc., a clinical stage pharmaceutical company, focuses on the discovery, development, and commercialization of therapeutics for rare endocrine diseases and endocrine-related tumors. Its lead product candidate is Paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist that has completed phase III clinical trial for the treatment of acromegaly, as well as completed phase II clinical trial to treat carcinoid syndrome and nonfunctional neuroendocrine tumors (NETs). The company is also developing CRN04777, an oral selective nonpeptide somatostatin type 5 receptor agonist, which is in phase I clinical trial for the treatment of congenital hyperinsulinism; and CRN04894, an oral adrenocorticotrophic hormone antagonist that is in phase I clinical trial for the treatment of Cushing's and congenital adrenal hyperplasia diseases. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious diseases using its proprietary Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. Its CRISPR/Cas9 is a gene editing technology that allows for precise directed changes to genomic DNA. The company has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta-thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It also develops CTX110, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19 positive malignancies; CTX120, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and CTX130, a donor-derived gene-edited allogeneic CAR-T investigational therapy targeting Cluster of Differentiation 70 to treat various solid tumors and hematologic malignancies. In addition, the company develops VCTX210, a gene-edited immune-evasive stem cell-derived product candidate for the treatment of treatment of type 1 diabetes; and pursues various in vivo gene-editing programs that target the liver, lung, muscle, and central nervous system diseases. It has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.

TG Therapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the acquisition, development, and commercialization of novel treatments for B-cell malignancies and autoimmune diseases. Its therapeutic product candidates include Ublituximab, an investigational glycoengineered monoclonal antibody for the treatment of B-cell non-hodgkin lymphoma, chronic lymphocytic leukemia (CLL), and relapsing forms of multiple sclerosis; and Umbralisib, an oral inhibitor of PI3K-delta and CK1-epsilon for the treatment of CLL, marginal zone lymphoma, and follicular lymphoma. The company also develops Cosibelimab, a human monoclonal antibody of IgG1 subtype that binds to programmed death-ligand 1 (PD-L1) and blocks its interactions with PD-1 and B7.1 receptors; TG-1701 is an orally available and covalently-bound Bruton's tyrosine kinase (BTK) inhibitor that exhibits selectivity to BTK compared to ibrutinib in in vitro kinase screening; and TG-1801, a bispecific CD47 and CD19 antibody. In addition, it has various licensed preclinical programs for BET, interleukin-1 receptor associated kinase-4, and GITR; and collaboration agreements with Checkpoint Therapeutics, Inc., Jiangsu Hengrui Medicine Co., Novimmune SA, Ligand Pharmaceuticals Incorporated, and Jubilant Biosys. The company has strategic alliances with LFB Biotechnologies S.A.S; GTC Biotherapeutics; LFB/GTC LLC; Ildong Pharmaceutical Co. Ltd.; and Rhizen Pharmaceuticals, S A. TG Therapeutics, Inc. was incorporated in 1993 and is headquartered in New York, New York.

Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapeutics. The company was incorporated in 2017 and is headquartered in Waltham, Massachusetts..

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. The company's lead product candidate is pegcetacoplan that is in Phase III clinical trials for the treatment of geographic atrophy (GA) in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria (PNH) diseases. It also develops EMPAVELI (systemic pegcetacoplan) for the treatment of cold agglutinin disease (CAD), and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) in hematology; C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN) in nephrology; and amyotrophic lateral sclerosis (ALS) in neurology. In addition, the company develops APL-2006, a bispecific C3 and VEGF inhibitor for treating complement-mediated disorders; APL-1030, a C3 inhibitor for the treatment of multiple neurodegenerative diseases; and the combination of EMPAVELI and a small interfering RNA, or siRNA for reducing the production of C3 proteins by the liver. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) to co-develop pegcetacoplan; and a research collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. Apellis Pharmaceuticals, Inc. was incorporated in 2009 and is based in Waltham, Massachusetts.

Apogee Therapeutics, Inc., through its subsidiary, operates as a biotechnology company that develops biologics for the treatment of atopic dermatitis (AD), chronic obstructive pulmonary disease (COPD), and related inflammatory and immunology indications. The company primarily develops APG777, a subcutaneous (SQ) extended half-life monoclonal antibody (mAb) for AD; and APG808, an SQ extended half-life mAb for COPD. Its earlier-stage programs include APG990, an SQ extended half-life mAb for the treatment of AD; and APG222, an extended half-life SQ antibodies for AD. The company was founded in 2022 and is based in Waltham, Massachusetts.

Merus N.V., a clinical-stage immuno-oncology company, engages in the discovery and development of bispecific antibody therapeutics in the Netherlands. Its bispecific antibody candidate pipeline includes Zenocutuzumab (MCLA-128), which is in a phase 2 clinical trials for the treatment of patients with metastatic breast cancer, as well as in Phase 1/2 clinical trials for the treatment of solid tumors that harbor Neuregulin 1. The company is also developing MCLA-158, which is in a phase I clinical trial for the treatment of solid tumors; MCLA-145, which is in phase 1 clinical trials for the treatment of solid tumors; MCLA-129, which is in phase 1/2 clinical trials for the treatment of patients with advanced non-small cell lung cancer and other solid tumors; and ONO-4685 that is Phase 1 clinical trial to treat relapsed/refractory T cell lymphoma. In addition, it has collaboration agreement with Betta Pharmaceuticals Co. Ltd for the research and development of stage bispecific antibody candidates include MCLA-129; and collaboration with Incyte Corporation for the development of MCLA-145. The company was incorporated in 2003 and is headquartered in Utrecht, the Netherlands.

Rhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of therapeutics for the treatment of rare genetic diseases of obesity. The company's lead product candidate is IMCIVREE, a potent melanocortin-4 receptor for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1, leptin receptor (LEPR) deficiency obesity, and Bardet-Biedl and Alström syndrome. It is also developing setmelanotide, which is in Phase II clinical trials for treating POMC or LEPR heterozygous deficiency obesities, steroid receptor coactivator 1 deficiency obesity, SH2B1 deficiency obesity, MC4 receptor deficiency obesity, Smith-Magenis syndrome obesity, POMC epigenetic disorders, and other MC4R disorders. Rhythm Pharmaceuticals, Inc. has a collaborative research agreement with the Clinical Registry Investigating Bardet-Biedl Syndrome. The company was formerly known as Rhythm Metabolic, Inc. and changed its name to Rhythm Pharmaceuticals, Inc. in October 2015. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.

MoonLake Immunotherapeutics, a clinical-stage biopharmaceutical company, engages in developing therapies. It is developing Sonelokimab, a novel investigational Nanobody therapy for the treatment of inflammation. The company is involved in conducting Phase II trials for hidradenitis suppurativa, psoriatic arthritis, ankylosing spondylitis, or radiographic axial spondyloarthritis. MoonLake Immunotherapeutics was founded in 2021 and is headquartered in Zug, Switzerland.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data. It also develops AT-GAA, a novel treatment paradigm for Pompe disease; enzyme replacement therapies for Pompe diseases; CLN3, which is in Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of an AAV serotype AT-GTX-502 gene therapy in patients with CLN3; and CDKL5, a gene on the X-chromosome encoding the CDKL5 protein that regulates the expression of essential proteins for normal brain development. The company has collaboration and license agreements with Nationwide Children's Hospital; University of Pennsylvania; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Philadelphia, Pennsylvania.

Kymera Therapeutics, Inc., a biopharmaceutical company, focuses on discovering and developing novel small molecule therapeutics that selectively degrade disease-causing proteins by harnessing the body's own natural protein degradation system. It engages in developing IRAK4 program, which is in Phase I clinical trial for the treatment of immunology-inflammation diseases, including hidradenitis suppurativa, atopic dermatitis, macrophage activation syndrome, general pustular psoriasis, and rheumatoid arthritis; IRAKIMiD program to treat MYD88-mutated diffuse large B cell lymphoma; STAT3 program for the treatment of hematologic malignancies and solid tumors, as well as autoimmune diseases and fibrosis; and MDM2 program to treat hematological malignancies and solid tumors. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.

Xenon Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN496, A Kv7 potassium channel opener that is Phase III clinical trials for the treatment of KCNQ2 developmental and epilepsy encephalopathy; and XEN1101, A Kv7 potassium channel opener, which is in Phase II clinical trial for the treatment of epilepsy and other neurological disorders. The company's product candidates also comprise NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase II clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other potential indications, including adult focal epilepsy; and XEN007, A central nervous system-acting calcium channel modulator, which is in Phase II clinical trials. It has a license and collaboration agreement with the Neurocrine Biosciences, Inc. to develop treatments for epilepsy; and with Flexion Therapeutics, Inc. to develop PCRX301 (XEN402, a Nav1.7 inhibitor) for the treatment of post-operative pain. Xenon Pharmaceuticals Inc. was incorporated in 1996 and is headquartered in Burnaby, Canada.

Morphic Holding, Inc., a biopharmaceutical company, discovers and develops oral small-molecule integrin therapeutics for the treatment of autoimmune, cardiovascular, metabolic diseases, as well as fibrosis and cancer. Its lead product candidates are MORF-057, an a4ß7-specific integrin inhibitor affecting inflammation that is in Phase 1 clinical trial for the treatment of inflammatory bowel disease; and preclinical trial to treat idiopathic pulmonary fibrosis and fibrosis diseases. The company is also developing avß6 for the treatment of fibrotic diseases, and avß8 to treat solid tumors, as well as various other products. Morphic Holding, Inc. has a research collaboration with Engitix Ltd for the identification of Morphic integrin technology amenable targets in fibrostenotic IBD; a collaboration agreement with Schrödinger, LLC for integrin targets; and a license agreement with Children's Medical Center Corporation to develop and commercialize products worldwide for any therapeutic or diagnostic use in humans and veterinary applications. It also has collaboration agreements with AbbVie Biotechnology Ltd and Janssen Pharmaceuticals, Inc. to discover or develop integrin-based therapeutics. Morphic Holding, Inc. was incorporated in 2014 and is headquartered in Waltham, Massachusetts.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders. Its portfolio pipeline includes commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focuses on the development of treatments for multiple therapeutic areas, such as rare diseases. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy in the European Economic Area and the United States, as well as to treat nonsense mutation Duchenne muscular dystrophy in Brazil and Russia; commercializes Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean; and markets Evrysdi for the treatment of spinal muscular atrophy in adults and children two months and older in Brazil. The company's splicing platform includes PTC518, which is being developed for the treatment of Huntington's disease. PTC Therapeutics, Inc. has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation to advance drug discovery and development research in regenerative medicine; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

Iovance Biotherapeutics, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing cancer immunotherapy products to harness the power of a patient's immune system to eradicate cancer cells. It has six ongoing phase 2 clinical studies, including C-144-01, of its lead product candidate, lifileucel, for the treatment of metastatic melanoma; C-145-04, of its product candidate lifileucel for recurrent, metastatic, or persistent cervical cancer; and C-145-03, of its product candidate LN-145, for recurrent and/or metastatic head and neck squamous cell carcinoma. Iovance Biotherapeutics, Inc. has collaborations and licensing agreements with H. Lee Moffitt Cancer Center; M.D. Anderson Cancer Center; Ohio State University; Centre hospitalier de l'Université de Montreal; Cellectis S.A.; and Novartis Pharma AG. The company was formerly known as Lion Biotechnologies, Inc. and changed its name to Iovance Biotherapeutics, Inc. in June 2017. Iovance Biotherapeutics, Inc. was incorporated in 2007 and is headquartered in San Carlos, California.

Geron Corporation, a late-stage clinical biopharmaceutical company, focuses on the development and commercialization of therapeutics for myeloid hematologic malignancies. It develops imetelstat, a telomerase inhibitor that is in Phase 3 clinical trials, which inhibits the uncontrolled proliferation of malignant stem and progenitor cells in hematologic myeloid malignancies for the treatment of low or intermediate-1 risk myelodysplastic syndromes and intermediate-2 or high-risk myelofibrosis. The company was incorporated in 1990 and is headquartered in Foster City, California.

Chinook Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead clinical program is atrasentan, a Phase III endothelin receptor antagonist for the treatment of IgA nephropathy and other proteinuric glomerular diseases. Its product candidates also include BION-1301, an anti-APRIL monoclonal antibody is being evaluated in a Phase I/II trial for IgA nephropathy; and CHK-336, an oral small molecule LDHA inhibitor for the treatment of primary hyperoxaluria, as well as research programs for other rare and severe chronic kidney diseases. Chinook Therapeutics, Inc. is headquartered in Seattle, Washington.

IDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, focuses on the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. The company's lead product candidates include IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase I clinical trial for patients with solid tumors having methylthioadenosine phosphorylase deletions; and IDE196, a protein kinase C inhibitor that is in Phase I/II clinical trial for genetically defined cancers having GNAQ or GNA11 gene mutations. Its preclinical pipeline includes various synthetic lethality programs targeting PARG inhibitor in tumors for patients having tumors with a defined biomarker based on genetic mutations and/or molecular signatures; Pol Theta inhibitors in tumors with BRCA or other homologous recombination deficiency mutations; and WRN inhibitors in tumors with high microsatellite instability. The company has a research collaboration agreement with Cancer Research UK and the University of Manchester to develop small molecule inhibitors of Poly (ADP-ribose) glycohydrolase; and a clinical trial collaboration and supply agreement with Pfizer Inc. for Phase I/II study in metastatic uveal melanoma, skin melanoma, and other solid tumors, as well as a strategic partnership with GlaxoSmithKline plc. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

Protagonist Therapeutics, Inc., a biopharmaceutical company, discovers and develops peptide-based therapeutic drugs to address hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing rusfertide (PTG-300), an injectable hepcidin mimetic that is in Phase II clinical trials for the treatment of patients with polycythemia vera and hereditary hemochromatosis, as well as for the treatment of other blood disorders; PN-943, an oral, alpha-4-beta-7 integrin- specific antagonist peptide that is in Phase II clinical trials for treating inflammatory bowel disease (IBD); and PN-235, an orally delivered interleukin-23 receptor specific antagonist for the treatment of IBD and non-IBD indications. The company has a license and collaboration agreement with Janssen Biotech, Inc. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, California.

Veracyte, Inc. operates as a diagnostics company worldwide. The company offers Afirma Genomic Sequencing Classifier and Xpression Atlas, which are used to determine patients with indeterminate results are benign to avoid unnecessary surgery; Decipher Prostate Biopsy and Radical Prostatectomy for prostate cancer diagnosis; Prosigna Breast Cancer Assay for breast cancer diagnosis; Percepta Genomic Sequencing Classifier and Percepta Nasal Swab Test for lung cancer diagnosis; Envisia Genomic Classifier for diagnosing interstitial lung disease, including idiopathic pulmonary fibrosis; and Immunoscore Colon Cancer test for colon cancer diagnosis. It is also developing Percepta Genomic Atlas to help inform lung cancer treatment decisions; Envisia Classifier, the nCounter analysis system; and LymphMark for lymphoma subtyping test. Veracyte, Inc. has technology licensing and collaboration arrangements with Johnson & Johnson; Acerta Pharma; and CareDx. The company was formerly known as Calderome, Inc. and changed its name to Veracyte, Inc. in March 2008. Veracyte, Inc. was incorporated in 2006 and is headquartered in South San Francisco, California.

ImmunityBio, Inc., a clinical-stage biotechnology company, develops therapies and vaccines to treat cancers and infectious diseases. It offers immunotherapy and cell therapy platforms, including antibody cytokine fusion proteins, synthetic immunomodulators, vaccine technologies, natural killer cells, and adaptive (T cell) immune systems. The company also develops therapeutic agents, which are in Phase II or III clinical trial for the treatment of liquid and solid tumors, including bladder, pancreatic, and lung cancers, as well as pathogens as SARS-CoV-2 and HIV. It has collaboration agreements with National Cancer Institute, National Institute of Deafness and Communication Disorders, and Amyris, Inc.; and license agreements with CytRx Corporation, EnGeneIC Pty Limited, GlobeImmune, Inc., and Infectious Disease Research Institute, Sanford Health, Shenzhen Beike Biotechnology Co. Ltd., Sorrento Therapeutics, Inc., and Viracta Therapeutics, Inc. The company was founded in 2014 and is based in San Diego, California.

ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of small molecule drugs that address unmet medical needs in central nervous system disorders. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis. It's pipeline include, pimavanserin, under phase 3 development for the treatment of Alzheimer's disease psychosis, and negative symptoms of schizophrenia; Trofinetide, a novel synthetic analog, under phase 3 development for the treatment of Rett syndrome; ACP-044, a novel first-in-class orally administered non-opioid analgesic, under phase 2 development for treating acute and chronic pain; and ACP-319, a positive allosteric modulator of the muscarinic receptor, under phase 1 development for treating schizophrenia and cognition in Alzheimer's. ACADIA Pharmaceuticals Inc. was founded in 1993 and is headquartered in San Diego, California.

CG Oncology Inc is a clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. Their product candidate cretostimogene, is initially in clinical development for the treatment of patients with high-risk Non-Muscle Invasive Bladder Cancer who are unresponsive to Bacillus Calmette Guerin (BCG) therapy, the current standard-of-care for high-risk NMIBC.

Agios Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of medicines in the field of cellular metabolism and adjacent areas of biology. The company offers PYRUKYND (mitapivat) an activator of both wild-type and a variety of mutant pyruvate kinase, PK, enzymes for the treatment of hemolytic anemias; and AG-946 that is in Phase I clinical study for treating hemolytic anemias and other indications. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Edgewise Therapeutics, Inc., a biopharmaceutical company, develops small molecule therapies for musculoskeletal diseases. The Company's lead product candidate, EDG-5506, is an orally administered small molecule designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy and Becker muscular dystrophy which has completed Phase 1 clinical trial. It develops a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address genetically defined muscle disorders. Edgewise Therapeutics, Inc. was incorporated in 2017 and is headquartered in Boulder, Colorado.

Vera Therapeutics, Inc., a clinical stage biotechnology company, focuses on developing and commercializing treatments for patients with serious immunological diseases in the United States. Its lead product candidate is atacicept, a fusion protein self-administered as a subcutaneous injection that is in Phase IIb clinical trial for patients with immunoglobulin A nephropathy. It is also developing MAU868, a monoclonal antibody for the treatment of BK viremia infections and is under Phase 2 clinical trial. The company was formerly known as Trucode Gene Repair, Inc. and changed its name to Vera Therapeutics, Inc. in April 2020. Vera Therapeutics, Inc. was incorporated in 2016 and is headquartered in Brisbane, California.

Structure Therapeutics Inc., a clinical stage global biopharmaceutical company, develops and delivers novel oral therapeutics to treat a range of chronic diseases with unmet medical needs. Its lead product candidate is GSBR-1290, an oral and biased small molecule agonist of glucagon-like-peptide-1 receptor, a validated G-protein-coupled receptors (GPCRs) drug target for type-2 diabetes mellitus and obesity. The company is also developing oral small molecule therapeutics targeting other GPCRs for the treatment of pulmonary and cardiovascular diseases, including ANPA-0073, a biased agonist, targeting apelin receptor, a GPCR that has been implicated in idiopathic pulmonary fibrosis (IPF) and pulmonary arterial hypertension; and LTSE-2578, an investigational oral small molecule lysophosphatidic acid 1 receptor antagonist for the treatment of IPF. The company was formerly known as ShouTi Inc. Structure Therapeutics Inc. was founded in 2016 and is headquartered in South San Francisco, California.

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes ARO-AAT, a RNA interference (RNAi) therapeutic candidate that is in Phase II clinical trial for the treatment of liver diseases associated with alpha-1 antitrypsin deficiency; ARO-APOC3, which is in phase 2b and one phase 3 clinical trial to treat hypertriglyceridemia; ARO-ANG3 that is in Phase 2b clinical trial to reduce production of angiopoietin-like protein 3; ARO-HSD, which is in Phase 1/2a clinical trial to treat liver diseases; ARO-ENaC, which is in a Phase 1/2a clinical trial to reduce production of the epithelial sodium channel alpha subunit in the airways of the lung; ARO-C3 for the treatment of complement-mediated disease that is in Phase 1/2a clinical trial; ARO-Lung2 for the treatment of chronic obstructive pulmonary disorder; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-XDH to treat uncontrolled gout; ARO-COV for the treatment of COVID-19 and other pulmonary-borne pathogens; and ARO-HIF2, which is in phase 1b clinical trial to treat clear cell renal cell carcinoma. It is also involved in the development of JNJ-3989, a subcutaneously administered RNAi therapeutic candidate to treat chronic hepatitis B virus infection; Olpasiran to reduce the production of apolipoprotein A; and ARO-AMG1 for treating genetically validated cardiovascular targets. Arrowhead Pharmaceuticals, Inc. has a license and research collaboration agreement with Janssen Pharmaceuticals, Inc. to develop ARO-JNJ1, ARO-JNJ2, and ARO-JNJ3 RNAi therapeutics for liver-expressed targets; and license and research collaboration agreement with Takeda Pharmaceuticals U.S.A., Inc. to develop RNAi therapeutic candidate as a treatment for liver disease. Arrowhead Pharmaceuticals, Inc. was incorporated in 1989 and is headquartered in Pasadena, California.

Zai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience primarily in Mainland China and Hong Kong. The company's commercial products include Zejula, a once-daily small-molecule poly polymerase 1/2 inhibitor; Optune, a device that delivers tumor treating fields; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; and Qinlock to treat gastrointestinal stromal tumors. It also develops Odronextamab to treat follicular lymphoma, diffuse large B-cell lymphoma, and other B-celllymphomas; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Margetuximab for the treatment of breast and gastroesophageal cancers; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, pancreatic cancer, and other solid tumors; and Bemarituzumab to treat gastric and gastroesophageal junction cancer. In addition, the company develops CLN-081 for the treatment of patients with EGFR exon 20 insertion NSCLC; Elzovantinib, an orally bioavailable multi-targeted kinase inhibitor; Tebotelimab, a tetravalent IgG4 monoclonal antibody; Retifanlimab that inhibits interactions between PD-1 and its ligands; ZL-2309, an orally active, selective, and ATP-competitive cell division cycle 7 (CDC7) kinase inhibitor; ZL-1201, a humanized IgG4 monoclonal antibody; Efgartigimod to reduce disease-causing immunoglobulin G antibodies; ZL-1102, a human nanobody targeting interleukin- 17A; KarXT for the treatment of psychiatric and neurological conditions; ZL-2313, an investigational inhibitor of triple-mutant EGFR harboring; ZL-2314, an investigational inhibitor of double-mutant EGFR harboring; and Sulbactam/durlobactam for the treatment of serious infections caused by Acinetobacter. Zai Lab Limited was incorporated in 2013 and is headquartered in Shanghai, China.

SpringWorks Therapeutics, Inc. acquires, develops, and commercializes medicines for underserved patient populations suffering from rare diseases and cancer. Its lead product candidate is nirogacestat, an oral small molecule gamma secretase inhibitor that is in Phase III clinical trial for the treatment of desmoid tumors. The company is also developing mirdametinib, an oral small molecule MEK inhibitor that is in Phase 2b clinical trials for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas, as well as Phase 1/2a clinical trial for the treatment of NF1-PN; mirdametinib + lifirafenib, a combination therapy that is in Phase 1b/2 clinical trial in patients with advanced or refractory solid tumors; and mirdametinib in monotherapy and combination approaches to treat solid tumors. In addition, it develops BGB-3245, an oral selective small molecule inhibitor of monomeric and dimeric forms of activating BRAF mutations, which is in Phase I clinical trial. The company has collaborations with BeiGene, Ltd., GlaxoSmithKline LLC, and Allogene to develop combination approaches with nirogacestat and mirdametinib; and license agreements with Pfizer Inc. for nirogacestat and mirdametinib. It also has a license agreement with Katholieke Universiteit Leuven and the Flanders Institute for Biotechnology for a portfolio of novel small molecule inhibitors of the TEA Domain; and Dana-Farber Cancer Institute for a portfolio of novel small molecule inhibitors of Epidermal Growth Factor Receptor. In addition, the company has clinical collaboration agreement with Janssen Biotech, Inc., Precision BioSciences, Inc., Seagen Inc., and AbbVie, Inc. SpringWorks Therapeutics, Inc. was founded in 2017 and is headquartered in Stamford, Connecticut.

Janux Therapeutics, Inc., a biopharmaceutical company, develops therapeutics based on proprietary Tumor Activated T Cell Engager (TRACTr) platform technology to treat patients suffering from cancer. The company's lead TRACTr product candidates that are in preclinical or discovery stage target prostate-specific membrane antigen, epidermal growth factor receptor, and trophoblast cell surface antigen 2. Janux Therapeutics, Inc. is also developing a Tumor Activated Immunomodulator (TRACIr) costimulatory bispecific product candidate against programmed death-ligand 1 and CD28 designed to improve the anti-tumor activity of T cells. In addition, its EGFR-TRACTr is designed to target EGFR in many cancer types with multiple approved monoclonal antibodies. The company was incorporated in 2017 and is headquartered in La Jolla, California.

Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company also develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis, and spinal muscular atrophy type 3, as well as to treat hereditary neuropathy with liability to pressure palsies. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was formerly known as Catalyst Pharmaceutical Partners, Inc. and changed its name to Catalyst Pharmaceuticals, Inc. in May 2015. Catalyst Pharmaceuticals, Inc. was founded in 2002 and is based in Coral Gables, Florida.

Verona Pharma plc, a clinical stage biopharmaceutical company, focuses on development and commercialization of therapies for the treatment of respiratory diseases with unmet medical needs. The company's product candidate is ensifentrine, an inhaled and dual inhibitor of the phosphodiesterase (PDE) 3 and PDE4 enzymes that acts as both a bronchodilator and an anti-inflammatory agent in a single compound, which is in Phase 3 clinical trials for the treatment of chronic obstructive pulmonary disease, asthma, and cystic fibrosis. It is developing ensifentrine in three formulations, including nebulizer, dry powder inhaler, and pressurized metered-dose inhaler. Verona Pharma plc was incorporated in 2005 and is headquartered in London, the United Kingdom.

Harmony Biosciences Holdings, Inc., a commercial-stage pharmaceutical company, develops and commercializes therapies for patients with rare neurological disorders in the United States. Its product, WAKIX is a medication for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. The company was formerly known as Harmony Biosciences II, Inc. and changed its name to Harmony Biosciences Holdings, Inc. in February 2020. Harmony Biosciences Holdings, Inc. was incorporated in 2017 and is based in Plymouth Meeting, Pennsylvania.

DICE Therapeutics, Inc., a biopharmaceutical company, builds various oral therapeutic candidates to treat chronic diseases in immunology and other therapeutic areas. Its platform DELSCAPE, is designed to discover selective oral small molecules to modulate protein-protein interactions (PPIs) as effectively as systemic biologics. The company's lead therapeutic candidate is DC-806, an oral antagonist of the pro-inflammatory signaling molecule, interleukin-17, which is a validated drug target implicated in a various immunology indications. It is also developing oral therapeutic candidates targeting a4ß7 integrin for the treatment of inflammatory bowel diseases, as well as targeting aVß1/aVß6 integrin for the treatment of idiopathic pulmonary fibrosis. In addition, the company focuses on immuno-oncology for antibody therapeutics. The company was incorporated in 2013 and is headquartered in South San Francisco, California.

Celldex Therapeutics, Inc., a biopharmaceutical company, engages in developing therapeutic monoclonal and bispecific antibodies for the treatment of various diseases. Its drug candidates include antibody-based therapeutics to treat patients with inflammatory diseases and various forms of cancer. The company's clinical development programs CDX-0159, a Phase I monoclonal antibody that binds the receptor tyrosine kinase KIT and inhibits its activity; CDX-1140, a human agonist monoclonal antibody targeted to CD40, a key activator of immune response, which is found on dendritic cells, macrophages, and B cells, as well as is expressed on various cancer cells; and CDX-527, a bispecific antibody, which uses the company's proprietary active anti-PD-L1 and CD27 human antibodies to couple CD27 costimulation with blockade of the PD-L1/PD-1 pathway to help prime and activate anti-tumor T cell responses through CD27 costimulation. The company has research collaboration and license agreements with University of Southampton to develop human antibodies towards CD27; Amgen Inc. with exclusive rights to CDX-301 and CD40 ligand; and Yale University. Celldex Therapeutics, Inc. was incorporated in 1983 and is headquartered in Hampton, New Jersey.

Keros Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of novel treatments for patients suffering from hematological and musculoskeletal disorders with high unmet medical need. The company's lead protein therapeutic product candidate is KER-050, which is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes, and in patients with myelofibrosis. It is also developing small molecule product candidate KER-047 that is being developed for the treatment of anemia, and is currently in Phase 1 clinical trial; and KER-012, which is in Phase 1 clinical trial to treat disorders associated with bone loss, such as osteoporosis and osteogenesis imperfecta, and for the treatment of pulmonary arterial hypertension. The company was incorporated in 2015 and is headquartered in Lexington, Massachusetts.

Neumora Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutic treatments for brain diseases, neuropsychiatric disorders, and neurodegenerative diseases. The company develops navacaprant (NMRA-140), a novel once-daily oral kappa opioid receptor antagonist, which is in phase 3 clinical trials for the treatment of major depressive disorder. It also develops NMRA-511 that is in phase 1 clinical trials in patients with agitation associated with dementia due to Alzheimer's disease; and NMRA-266, which is in the phase 1 clinical trial for the treatment of schizophrenia and other neuropsychiatric disorders. In addition, its preclinical phase product includes NMRA-NMDA for the treatment of schizophrenia; NMRA-CK1d, a CK1d inhibitor program for the treatment of amyotrophic lateral sclerosis; NMRA-NLRP3 for the treatment of certain neurodegenerative conditions; and NMRA-GCase for the treatment of Parkinson's disease. The company was formerly known as RBNC Therapeutics, Inc. and changed its name to Neumora Therapeutics, Inc. in October 2021. Neumora Therapeutics, Inc. was incorporated in 2019 and is headquartered in Watertown, Massachusetts.

Maravai LifeSciences Holdings, Inc., a life sciences company, provides products to enable the development of drug therapies, diagnostics, novel vaccines, and support research on human diseases in the United States and internationally. The company's products address the key phases of biopharmaceutical development and include nucleic acids for diagnostic and therapeutic applications, antibody-based products to detect impurities during the production of biopharmaceutical products, and products to detect the expression of proteins in tissues of various species. It operates in two segments, Nucleic Acid Production and Biologics Safety Testing. The Nucleic Acid Production segment manufactures and sells products for use in the fields of gene therapy, nucleoside chemistry, oligonucleotide therapy, and molecular diagnostics, including reagents used in the chemical synthesis, modification, labelling, and purification of deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). This segment also offers messenger RNA, oligonucleotides, and oligonucleotide building blocks, as well as plasmid DNA and CleanCap capping technology. The Biologics Safety Testing segment sells analytical products for use in biologic manufacturing process development, including custom product-specific development antibody and assay development services. This segment also provides HCP ELISA kits, other bioprocess impurity and contaminant ELISA kits, ancillary reagents, and custom services. The company serves biopharmaceutical companies, and other biopharmaceutical and life sciences research companies; and academic research institutions and in vitro diagnostics companies. Maravai LifeSciences Holdings, Inc. was incorporated in 2020 and is headquartered in San Diego, California.

Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 for the treatment of hereditary angioedema, as well as other liver-focused programs comprising hemophilia A and hemophilia B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo pipeline includes NTLA-5001 for the treatment of acute myeloid leukemia; and proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune disorders. In addition, it offers tools comprising of Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for the treatment of sickle cell disease; Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; Ospedale San Raffaele; and a strategic collaboration with SparingVision SAS to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. The company was formerly known as AZRN, Inc. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

Vericel Corporation, a commercial-stage biopharmaceutical company, engages in the research, development, manufacture, and distribution of cellular therapies for sports medicine and severe burn care markets in the United States. The company markets autologous cell therapy products comprising MACI, an autologous cellularized scaffold product for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; and Epicel, a permanent skin replacement humanitarian use device for the treatment of adult and pediatric patients with deep-dermal or full-thickness burns. Its preapproval stage product is NexoBrid, a registration-stage biological orphan product for eschar removal in adults with deep partial-thickness and/or full-thickness thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was incorporated in 1989 and is headquartered in Cambridge, Massachusetts.

Novavax, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of vaccines to prevent serious infectious diseases and address health needs. The company's vaccine candidates include NVX-CoV2373, a coronavirus vaccine candidate that is in two Phase III trials, one Phase IIb trial, and one Phase I/II trial; NanoFlu, a nanoparticle seasonal quadrivalent influenza vaccine candidate that is in Phase 3 clinical trial; and ResVax, a respiratory syncytial virus (RSV) fusion (F) protein nanoparticle vaccine candidate. It is also developing RSV F vaccine that is in Phase II clinical trial for older adults (60 years and older), as well as that is in Phase I clinical trial for pediatrics. It has a collaboration agreement with Takeda Pharmaceutical Company Limited for the development, manufacturing, and commercialization of NVX-CoV2373, a COVID-19 vaccine candidate. Novavax, Inc. was incorporated in 1987 and is headquartered in Gaithersburg, Maryland.

Beam Therapeutics Inc., a biotechnology company, develops precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing BEAM-101 for the treatment of sickle cell disease and beta thalassemia; BEAM-102 for the treatment of sickle cell disease; and BEAM-201, an allogeneic chimeric antigen receptor T cell for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia; and BEAM-301, a liver-targeted development candidate for the treatment of patients with Glycogen Storage Disease Type Ia. It also develops therapies for alpha-1 antitrypsin deficiency; ocular diseases; and other liver, muscle, and central nervous system disorders. The company has an alliance with Boston Children's Hospital; a research and clinical trial collaboration agreement with Magenta Therapeutics, Inc.; license agreement with Sana Biotechnology, Inc.; and a research collaboration with the Institute of Molecular and Clinical Ophthalmology Basel. It also has a research collaboration agreement with Pfizer Inc. and Apellis Pharmaceuticals, Inc.; and collaboration and license agreement with Verve Therapeutics, Inc. The company was incorporated in 2017 and is based in Cambridge, Massachusetts.

Akero Therapeutics, Inc., a cardio-metabolic nonalcoholic steatohepatitis (NASH) company, engages in the development of medicines designed to restore metabolic balance and improve overall health. The company's lead product candidate is efruxifermin (EFX), an analog of fibroblast growth factor 21, which protects against cellular stress and regulates metabolism of lipids, carbohydrates, and proteins throughout the body. It also conducts a Phase 2a clinical trial, the BALANCED study, to evaluate EFX in the treatment of biopsy-confirmed NASH patients. The company was formerly known as Pippin Pharmaceuticals, Inc. and changed its name to Akero Therapeutics, Inc. in May 2018. Akero Therapeutics, Inc. was incorporated in 2017 and is headquartered in South San Francisco, California.

Soleno Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of novel therapeutics for the treatment of rare diseases. Its lead candidate is Diazoxide Choline Controlled-Release, a once-daily oral tablet for the treatment of Prader-Willi Syndrome, which is being evaluated in an ongoing Phase III clinical development program. The company was formerly known as Capnia, Inc. and changed its name to Soleno Therapeutics, Inc. in May 2017. Soleno Therapeutics, Inc. was incorporated in 1999 and is based in Redwood City, California.

Galapagos NV, an integrated biopharmaceutical company, engages in the discovery, development, and commercialization of various medicines for high unmet medical need. Its pipeline products include filgotinib, a JAK1 inhibitor that is in various phases of clinical trials for the treatment of rheumatoid arthritis, Crohn's disease, ulcerative colitis, small bowel CD, fistulizing CD, ankylosing spondylitis, psoriatic arthritis, and uveitis. The company's pipeline products also comprise GLPG1972 that has completed Phase 2b trial for treating osteoarthritis; Toledo molecules, including GLPG3970, GLPG4399, and GLPG4876 for inflammation; and GLPG4716 and Ziritaxestat to treat idiopathic pulmonary fibrosis. In addition, its other pipeline products include GLPG2737, a cystic fibrosis transmembrane conductance regulator that is in Phase 2 clinical trials to treat patients with autosomal dominant polycystic kidney disease; and GLPG0555, a JAK1 inhibitor, which is in Phase 1b for treatment of patients with osteoarthritis. The company has collaboration agreements with Gilead Sciences, Inc.; AbbVie S.à r.l.; and Novartis Pharma AG. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.

BELLUS Health Inc., a clinical stage biopharmaceutical company, develops therapeutics for the treatment of refractory chronic cough (RCC) and other cough hypersensitivity indications. Its lead product candidate includes BLU-5937, an antagonist of the P2X3 receptor, which is in Phase II clinical trial for treatment of RCC and chronic pruritus. The company was incorporated in 1993 and is based in Laval, Canada.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. The company's lead product candidate is LIVMARLI, an investigational oral drug for the treatment of progressive familial intrahepatic cholestasis disease, as well as for the treatment of Alagille syndrome and biliary atresia disease. It also develops Volixibat drug for treatment of intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Mirum Pharmaceuticals, Inc. was incorporated in 2018 and is headquartered in Foster City, California.

Recursion Pharmaceuticals, Inc. operates as a clinical-stage biotechnology company, engages in the decoding biology by integrating technological innovations across biology, chemistry, automation, data science, and engineering to industrialize drug discovery. The company develops REC-994, which is in Phase IIa clinical trial to treat cerebral cavernous malformation; REC-2282 for the treatment of neurofibromatosis type 2; REC-4881 to treat familial adenomatous polyposis; and REC-3599, which is in Phase I clinical trial to treat GM2 gangliosidosis. Its preclinical stage product includes REC-3964 to treat Clostridium difficile colitis; REC-64917 for the treat of neural or systemic inflammation; REC-65029 to treat HRD-negative ovarian cancer; REC-648918 to enhance anti-tumor immune; REC-2029 for the treatment of wnt-mutant hepatocellular carcinoma; REC-14221 to treat solid and hematological malignancies; and REC-64151 for the treatment of immune checkpoint resistance in KRAS/STK11 mutant non-small cell lung cancer. The company has collaboration and agreement with Bayer AG; the University of Utah Research Foundation; Ohio State Innovation Foundation; Chromaderm, Inc.; and Takeda Pharmaceutical Company Limited. Recursion Pharmaceuticals, Inc. was incorporated in 2013 and is headquartered in Salt Lake City, Utah.

Fusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines. The company has developed Targeted Alpha Therapies platform together with its proprietary Fast-Clear linker technology to enable us to connect alpha particle emitting isotopes to various targeting molecules in order to selectively deliver the alpha particle payloads to tumors. Its lead product candidate is FPI-1434 that is in Phase 1 clinical trials as a monotherapy in patients with solid tumors expressing insulin-like growth factor 1 receptor. The company is also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, it is progressing its earlier-stage product candidate, FPI-1966 into clinical development for the treatment of head and neck, and bladder cancers expressing fibroblast growth factor receptor. The company has a strategic collaboration agreement with AstraZeneca UK Limited to jointly discover, develop, and commercialize alpha-emitting radiopharmaceuticals and combination therapies for the treatment of cancer. Fusion Pharmaceuticals Inc. was incorporated in 2014 and is headquartered in Hamilton, Canada.

Ligand Pharmaceuticals Incorporated, a biopharmaceutical company, focuses on developing or acquiring technologies that help pharmaceutical companies to discover and develop medicines worldwide. Its commercial programs include Kyprolis and Evomela, which are used to treat multiple myeloma; Veklury for the treatment of moderate or severe COVID-19; Teriparatide injection product for osteoporosis; Vaxneuvance for the prevention of invasive disease caused by Streptococcus pneumoniae; and Pneumosil, a pneumococcal conjugate vaccine to help fight against pneumococcal pneumonia among children. The company also offers Rylaze, a recombinant erwinia asparaginase for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma in adult and pediatric patients; and Nexterone, a captisol-enabled formulation of amiodarone; and Zulresso, a captisol-enabled formulation of brexanolone for the treatment of postpartum depression. In addition, it provides Noxafil-IV, a captisol-enabled formulation of posaconazole for IV use; Duavee for the treatment of postmenopausal osteoporosis; Aziyo portfolio of commercial pericardial repair and CanGaroo envelope extracellular matrix products; Exemptia for autoimmune diseases; Vivitra for breast cancer; Bryxta and Zybev for various indications; and Minnebro for the treatment of hypertension. The company's partners and licenses programs, which are in clinical development used for the treatment of cancer, seizure, diabetes, cardiovascular disease, muscle wasting, liver and kidney disease, and other diseases. Further, it sells Captisol materials. The company was incorporated in 1987 and is headquartered in Emeryville, California.

Centessa Pharmaceuticals plc, a clinical-stage pharmaceutical company, discovers, develops, and delivers medicines to patients. Its registrational pipeline products include Lixivaptan, a vasopressin V2 receptor small molecule inhibitor that is in Phase III clinical development for the treatment of autosomal dominant polycystic kidney disease; and SerpinPC, an activated protein C inhibitor, which is in Phase IIa clinical development for the treatment of hemophilia A and B. The company also has emerging pipeline products under clinical proof of concept stage comprising LB101, a PD-L1xCD47 LockBody, as well as LB201, a PD-L1xCD3 LockBody, which are designed to selectively drive potent CD47 and CD3 effector function activity while avoiding systemic toxicity; ZF874, a small molecule pharmacological chaperone folding corrector of the Z variant of alpha-1-antitrypsin in Phase I clinical development for the treatment of alpha-1-antitrypsin deficiency; MGX292, a recombinant modified BMP9 replacement protein designed to overcome the deficiency in BMP9 signaling in Pulmonary Arterial Hypertension; and OX2R Agonists that are oral and intranasal selective orexin receptor 2 agonists designed to leverage structural insights and to directly target the underlying pathophysiology of orexin neuron loss in Narcolepsy Type 1. In addition, the company has exploratory pipeline products under clinical proof of concept stage containing CBS001, an anti-LIGHT antibody, which preferentially binds the inflammatory membrane form of LIGHT in inflammatory/fibrotic diseases; and CBS004, a humanized mAb specific to BDCA2, expressed exclusively on plasmacytoid dendritic cells in systemic sclerosis, systemic lupus erythematosus, and other autoimmune diseases. Centessa Pharmaceuticals plc was incorporated in 2020 and is based in Altrincham, the United Kingdom.

Fortrea Holdings Inc., a contract research organization, primarily engages in the provision of biopharmaceutical product and medical device development services worldwide. It operates through two segments: Clinical Services and Enabling Services. The Clinical Services segment provides across the clinical pharmacology and clinical development spectrum. The Enabling Services segment provides patient access and clinical trial technology solutions to customers that streamline complex randomization and optimize the trial drug supply process. The company offers delivery models that include full service, functional service provider, and hybrid service structures. It also offers phase I-IV clinical trial management, differentiated technology enabled trial solutions, and post approval services. The company serves pharmaceutical, biotechnology, and medical device organizations. Fortrea Holdings Inc. was incorporated in 2023 and is based in Durham, North Carolina.

Kiniksa Pharmaceuticals, Ltd., a biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical needs worldwide. Its product candidates include ARCALYST, an interleukin-1alpha and interleukin-1beta, for the treatment of recurrent pericarditis, which is an inflammatory cardiovascular disease; Mavrilimumab, a monoclonal antibody inhibitor that completed Phase II clinical trials for the treatment of giant cell arteritis; Vixarelimab, a monoclonal antibody, which is in Phase 2a clinical trial for the treatment of prurigo nodularis, a chronic inflammatory skin condition; and KPL-404, a monoclonal antibody inhibitor of the CD40- CD154 interaction, a T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching, and type 1 immune response. Kiniksa Pharmaceuticals, Ltd. was incorporated in 2015 and is based in Hamilton, Bermuda.

Viridian Therapeutics, Inc., a biotechnology company, develops treatments for patients suffering from serious diseases. It develops VRDN-001, a humanized monoclonal anti-IGF-1R antibody that is in Phase 1/2 clinical trial for the treatment of thyroid eye disease (TED); VRDN-002, an IGF-1R antibody, which is in Phase 1 clinical trial; and VRDN-003, a therapeutic antibody targeting IGF-1R for the treatment of TED. The company was formerly known as Miragen Therapeutics, Inc. and changed its name to Viridian Therapeutics, Inc. in January 2021. Viridian Therapeutics, Inc. was founded in 2006 and is headquartered in Waltham, Massachusetts.

MannKind Corporation, a biopharmaceutical company, focuses on the development and commercialization of inhaled therapeutic products for endocrine and orphan lung diseases in the United States. It offers Afrezza, an inhaled insulin used to improve glycemic control in adults with diabetes. It also promotes Thyquidity to adult and pediatric endocrinologists, and other healthcare providers for the treatment of hypothyroidism. The company has a license and collaboration agreement with United Therapeutics Corporation. It also has an agreement with NRx Pharmaceuticals to develop a dry powder formulation of ZYESAMI (aviptadil), a synthetic form of human vasoactive intestinal peptide to help protect cells against inflammatory conditions. MannKind Corporation was incorporated in 1991 and is headquartered in Westlake Village, California.

Arvinas, Inc., a clinical-stage biopharmaceutical company, engages in the discovery, development, and commercialization of therapies to degrade disease-causing proteins. Its lead product candidates include Bavdegalutamide, a proteolysis targeting chimera (PROTAC) protein degrader that is in phase I clinical trial targeting the androgen receptor (AR) protein for the treatment of men with metastatic castration-resistant prostate cancer (mCRPC); ARV-471, a PROTAC protein degrader targeting the estrogen receptor protein for the treatment of patients with metastatic ER positive/HER2 negative breast cancer; and ARV-766 an investigational orally bioavailable PROTAC protein degrader for the treatment of men with mCRPC. The company has collaborations with Pfizer Inc., Genentech, Inc., F. Hoffman-La Roche Ltd., and Bayer AG. Arvinas, Inc. was founded in 2013 and is based in New Haven, Connecticut.

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. It has license agreements with Fred Hutchinson Cancer Research Center; Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; CIEMAT and UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. is headquartered in Cranbury, New Jersey.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are SNDX-5613, which is in phase 1/2 clinical trial targeting the binding interaction of Menin with the mixed lineage leukemia 1 protein for the treatment of MLL-rearranged (MLLr) and nucleophosmin 1 mutant acute myeloid leukemia (NPM1c AML); and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD). The company is also developing Entinostat. It also has collaborative research and development agreement with National Cancer Institute; a clinical trial agreement with Eastern Cooperative Oncology Group; and a license agreement with Kyowa Hakko Kirin Co., Ltd. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, Massachusetts.Massachusetts.

Ardelyx, Inc., a biopharmaceutical company, discovers, develops, and commercializes medicines to treat gastrointestinal and cardiorenal therapeutic areas in the United States and internationally. The company's lead product candidate is tenapanor, which has completed Phase III clinical trial for the treatment of patients with irritable bowel syndrome with constipation, as well as in Phase III clinical trial to control serum phosphorus in adult patients with chronic kidney disease (CKD)on dialysis, or hyperphosphatemia. It is also developing RDX013, a potassium secretagogue, for the treatment of elevated serum potassium, or hyperkalemia, a problem among certain patients with kidney and/or heart disease; and RDX020, an early-stage program in metabolic acidosis, a serious electrolyte disorder in patients with CKD. The company has agreements with Kyowa Kirin in Japan, Fosun Pharmaceutical Industrial Development Co. Ltd. in China, and Knight Therapeutics, Inc. in Canada for the development and commercialization of tenapanor in their respective territories. The company was formerly known as Nteryx, Inc. and changed its name to Ardelyx, Inc. in June 2008. Ardelyx, Inc. was incorporated in 2007 and is headquartered in Waltham, Massachusetts.

BioCryst Pharmaceuticals, Inc., a biotechnology company, discovers novel, oral, and small-molecule medicines. The company markets peramivir injection, an intravenous neuraminidase inhibitor for the treatment of acute uncomplicated influenza under the RAPIVAB, RAPIACTA, and PERAMIFLU names; and ORLADEYO, an oral serine protease inhibitor to treat hereditary angioedema. It is also developing BCX9930, an oral factor D inhibitor, which is in Phase II clinical trial for complement-mediated diseases; BCX9250, an oral activin receptor-like kinase-2 inhibitor that is in Phase I clinical trial to treat fibrodysplasia ossificans progressiva; and Galidesivir, a RNA dependent-RNA polymerase inhibitor, which is in Phase I clinical trial to treat various RNA viruses, including Marburg, Yellow Fever, Ebola, and Zika. The company has collaborations and in-license relationships with the Torii Pharmaceutical Co., Ltd.; Seqirus UK Limited; Shionogi & Co., Ltd.; Green Cross Corporation; Mundipharma International Holdings Limited; National Institute of Allergy and Infectious Diseases; Biomedical Advanced Research and Development Authority; the U.S. Department of Health and Human Services; and The University of Alabama at Birmingham, as well as Albert Einstein College of Medicine of Yeshiva University and Industrial Research, Ltd. BioCryst Pharmaceuticals, Inc. was founded in 1986 and is headquartered in Durham, North Carolina.

Bicycle Therapeutics plc, a clinical-stage biopharmaceutical company, develops a class of medicines for diseases that are underserved by existing therapeutics. Its lead product candidate is BT1718, a bicycle toxin conjugate (BTC), which is in Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. The company's oncology product candidates also comprise BT5528, a BTC that is in a Phase I/II clinical trial targeting EphA2; and BT8009, which is in Phase I/II clinical trial targeting Nectin-4. In addition, it is developing THR-149, a plasma kallikrein inhibitor that completed Phase II clinical trials for the treatment of diabetic macular edema; BT7480, a Bicycle tumor-targeted immune cell agonist (TICA) targeting Nectin-4 and agonizing; BT7455, an EphA2/CD137 Bicycle TICA, which is in preclinical studies; and BT1718, a BTC that target tumors, that is in Phase I/IIa clinical trial that express Membrane Type 1 matrix metalloproteinase. Further, the company collaborates with biopharmaceutical companies and organizations to develop programs in therapeutic areas, such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. It has a clinical trial and license agreement with the Cancer Research Technology Limited and Cancer Research UK; research collaboration agreements with AstraZeneca, Sanofi, Oxurion, and the Dementia Discovery Fund; and discovery collaboration and license agreement with Genentech for the discovery and development of Bicycle peptides for multiple immuno-oncology targets. Bicycle Therapeutics plc was incorporated in 2009 and is headquartered in Cambridge, the United Kingdom.

Immunocore Holdings plc, a commercial-stage biotechnology company, engages in the development of immunotherapies for the treatment of cancer, infectious, and autoimmune diseases. The company offers KIMMTRAK for the treatment of patients with unresectable or metastatic uveal melanoma. Its other programs for oncology comprise IMC-C103C that is in Phase I/II dose escalation trial in patients with solid tumor cancers, including non-small-cell lung (NSCLC), gastric, head and neck, ovarian, and synovial sarcoma cancers; IMC-F106C, which is in a Phase I/II dose escalation trial in patients with multiple solid tumor cancers comprising NSCLC, small-cell lung, endometrial, ovarian, cutaneous melanoma, and breast cancers. In addition, the company's programs for infectious diseases include IMC-I109V, which is in a Phase I/II clinical trial in patients with chronic hepatitis B virus; and IMC-M113V that is in pre-clinical development stage for patients with human immunosuppression virus. Further, it develops product candidates to provide precision targeted immunosuppression for the treatment of autoimmune diseases. Immunocore Holdings plc was founded in 1999 and is headquartered in Abingdon, the United Kingdom.

Spyre Therapeutics, Inc., a biotechnology company, focuses on advancing a pipeline of antibody therapeutics for the treatment of inflammatory bowel disease by combining antibody engineering, rational therapeutic combinations, and precision medicine approaches for patient selection. The company's pipeline includes extended half-life antibodies targeting a4ß7, TL1A, and IL-23. The company was formerly known as Aeglea BioTherapeutics, Inc. and changed its name to Spyre Therapeutics, Inc. in November 2023. Spyre Therapeutics, Inc. was founded in 2013 and is based in Waltham, Massachusetts.

Kura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer in the United States. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, a small molecule inhibitor of the menin-Lysine K-specific Methyltransferase 2A protein-protein interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; and tipifarnib, an orally bioavailable inhibitor of farnesyl transferase that is in Phase II clinical trials for the treatment of solid tumors and hematologic indications. The company has a clinical collaboration with Novartis to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression or PIK3CA mutation and/or amplification. Kura Oncology, Inc. was founded in 2014 and is headquartered in San Diego, California.

NewAmsterdam Pharma Company N.V., a clinical-stage biopharmaceutical company, focuses on improving patient care in populations with metabolic diseases. Its lead investigational candidate, obicetrapib, is a novel, selective inhibitor that targets the Cholesteryl Ester Transfer Protein (CETP), which has been clinically shown to reduce low-density lipoprotein cholesterol (LDL-C) while at the same time substantially increase high-density lipoprotein cholesterol (HDL-C). The company was founded in 2019 and is based in Naarden, the Netherlands.

Disc Medicine, Inc., a clinical-stage biotechnology company, engages in discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. It builds a portfolio of therapeutic candidates that address a spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, primarily heme biosynthesis and iron homeostasis. The company is based in Watertown, Massachusetts.

Nurix Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule therapies for the treatment of cancer and immune disorders. The company develops NX-2127, an orally available Bruton's tyrosine kinase (BTK) degrader for the treatment of relapsed or refractory B-cell malignancies; NX-5948, an orally bioavailable BTK degrader for the treatment of relapsed or refractory B-cell malignancies and autoimmune diseases; and NX-1607, an orally available Casitas B-lineage lymphoma proto-oncogene-B (CBL-B) inhibitor for immuno-oncology indications. It also develops NX-0255, a CBL-B inhibitor for ex vivo use to enhance adoptive T-cell therapy; and DeTIL-0255 that is in the Phase 1 clinical trial for the treatment of gynecologic cancers, including ovarian, endometrial, and cervical cancer. The company has a strategic collaboration agreement with Gilead Sciences, Inc. for cancer and other challenging diseases patients; and Sanofi S.A. The company was formerly known as Nurix Inc. and changed its name to Nurix Therapeutics, Inc. in October 2018. The company was incorporated in 2009 and is headquartered in San Francisco, California.

Belite Bio, Inc, a clinical-stage biopharmaceutical drug development company, engages in the research and development of novel therapeutics targeting atrophic age-related macular degeneration and autosomal recessive stargardt diseases. Its lead product candidate is LBS-008, an oral once-a-day treatment that can reduce and maintain the delivery of vitamin A to the eye to reduce the accumulation of toxic vitamin A by-products in ocular tissue that is in phase 3 clinical trial. The company is also developing LBS-009, an anti-retinol binding protein 4 oral therapy targeting liver disease, including non-alcoholic fatty liver disease, nonalcoholic steatohepatitis, and type 2 diabetes, which is in the preclinical development phase. Belite Bio, Inc was founded in 2016 and is based in San Diego, California. Belite Bio, Inc operates as a subsidiary of Lin Bioscience International Ltd.

Cassava Sciences, Inc., a clinical stage biotechnology company, develops drugs for neurodegenerative diseases. Its lead therapeutic product candidate is simufilam, a small molecule drug, which is completed Phase 2b clinical trial; and investigational diagnostic product candidate is SavaDx, a blood-based biomarker/diagnostic to detect Alzheimer's disease. The company was formerly known as Pain Therapeutics, Inc. and changed its name to Cassava Sciences, Inc. in March 2019. Cassava Sciences, Inc. was incorporated in 1998 and is based in Austin, Texas.

Xencor, Inc., a clinical stage biopharmaceutical company, focuses on the discovery and development of engineered monoclonal antibody and cytokine therapeutics to treat patients with cancer and autoimmune diseases. The company provides Sotrovimab that targets the SARS-CoV-2 virus; Ultomiris for the treatment of patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome; and Monjuvi for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma. It develops Plamotamab, a tumor-targeted antibody, which is in Phase I clinical trial to treat non-Hodgkin lymphoma; Vudalimab, a bispecific antibody, which is in Phase II clinical trial to treat metastatic castration-resistant prostate cancer and other solid tumor types; and Tidutamab that is in Phase II clinical trial to treat neuroendocrine tumors. The company is also developing XmAb306, which is in Phase I clinical trial to treat solid tumors; XmAb104 and XmAb841, which are in Phase I clinical trial to treat patients with selected solid tumors; XmAb564 that is in Phase I clinical trial to treat autoimmune diseases; AMG 509, which is in Phase I clinical trial to treat prostate cancer; XmAb819 for patients with renal cell carcinoma; and Novartis XmAb. It develops VIR-3434, which is in Phase II clinical trial for patients with hepatitis B virus infection; VIR-2482 that is in Phase 1/2 clinical trial to trat influenza A; VIR-7832, which is in Phase 1b/2a trial to treat mild-to-moderate COVID-19; and BMS-986414 + BMS-986413 is in Phase 2/3 NIH ACTIV-2 trial in treating COVID-19. The company develops AIMab7195 to reduce blood serum levels of IgE that mediates allergic responses and allergic disease; Obexelimab to treat autoimmune disease; and Xpro1595 to treat patients with Alzheimer's disease, mild cognitive impairment, and depression. It has a license agreement with Caris Life Sciences. Xencor, Inc. was incorporated in 1997 and is headquartered in Monrovia, California.

ARS Pharmaceuticals, Inc. develops ARS-1, a novel intranasal epinephrine spray with absorption technology for patients and their families at-risk of severe allergic reactions to food, medications, and insect bites. Its product includes Neffy, a low-dose intranasal epinephrine nasal spray. The company was incorporated in 2015 and is based in San Diego, California.

Arcus Biosciences, Inc., a clinical-stage biopharmaceutical company, develops and commercializes cancer therapies in the United States. Its product pipeline includes, Etrumadenant, a dual A2a/A2b adenosine receptor antagonist, which is in a Phase 1b/2 clinical trial; and Zimberelimab, an anti-PD-1 antibody that is in Phase 1b clinical trial for monotherapy. The company also develops Domvanalimab, an anti-TIGIT monoclonal antibody, which is in Phase 2 development for the treatment of first-line metastatic non-small cell lung cancer in combination with Zimberelimab; Quemliclustat, a small-molecule CD73 inhibitor is in a Phase 1/1b study for the treatment of first-line metastatic pancreatic cancer; and AB521, an oral and small molecule HIF-2a inhibitor that is in Phase 1 study for the treatment of patients with von Hippel- Lindau disease. It has a clinical development collaboration agreement with Strata Oncology, Inc. to evaluate Zimberelimab; a collaboration with AstraZeneca, BVF Partners L.P to evaluate domvanalimab, its investigational anti-TIGIT antibody, in combination with Imfinzi (durvalumab) in a registrational Phase 3 clinical trial in patients with unresectable Stage III non-small cell lung cancer; and license agreements with Taiho Pharmaceutical Co., Ltd, Abmuno Therapeutics LLC, and WuXi Biologics to develop anti-CD39 antibody for the treatment of cancer. The company was incorporated in 2015 and is headquartered in Hayward, California.

Ocular Therapeutix, Inc., a biopharmaceutical company, focuses on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its bioresorbable hydrogel-based formulation technology. The company markets ReSure Sealant, an ophthalmic device to prevent wound leaks in corneal incisions following cataract surgery; and DEXTENZA, a dexamethasone ophthalmic insert to treat post-surgical ocular inflammation and pain following ophthalmic surgery, as well as allergic conjunctivitis. It is also developing OTX-TKI, an axitinib intravitreal implant that is in phase 1 clinical trials for the treatment of wet age-related macular degeneration and other retinal diseases; OTX-TIC, a travoprost intracameral implant, which is in phase 2 clinical trials for the treatment of open-angle glaucoma or ocular hypertension; OTX-CSI, a cyclosporine intracanalicular insert that has completed phase 2 clinical trials for the treatment of dry eye disease; and OTX-DED, a dexamethasone intracanalicular insert, which is in phase 2 clinical trials for the short-term treatment of the signs and symptoms of dry eye disease. The company has a strategic collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) for the development and commercialization of products using the Company's sustained-release hydrogel in combination with Regeneron's large molecule VEGF-targeting compounds for the treatment of retinal diseases; and AffaMed Therapeutics Limited for the development and commercialization of DEXTENZA and OTX-TIC, as well as a discovery collaboration with Mosaic Biosciences to identify new targets and therapeutic agents for the treatment of dry age-related macular degeneration (dMAD). Ocular Therapeutix, Inc. was incorporated in 2006 and is headquartered in Bedford, Massachusetts.

Longboard Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing transformative medicines for neurological diseases. The company's lead product candidate is LP352, which is in a Phase 1b/2a clinical trial for the treatment of seizures associated with developmental and epileptic encephalopathies. Its preclinical product candidates include LP659 and LP143, which focuses on developing therapies for multiple neurological diseases. The company was formerly known as Arena Neuroscience, Inc. and changed its name to Longboard Pharmaceuticals, Inc. in October 2020. Longboard Pharmaceuticals, Inc. was incorporated in 2020 and is based in La Jolla, California.

Tarsus Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of novel therapeutic candidates for ophthalmic conditions. Its lead product candidate is TP-03, a novel therapeutic that is in Phase III for the treatment of blepharitis caused by the infestation of Demodex mites, as well as to treat meibomian gland disease. The company is also developing TP-04 for the treatment of rosacea; and TP-05 for Lyme prophylaxis and community malaria reduction. In addition, the company develops lotilaner to address diseases across therapeutic categories in human medicine, including eye care, dermatology, and other diseases. Tarsus Pharmaceuticals, Inc. was incorporated in 2016 and is headquartered in Irvine, California.

Tyra Biosciences, Inc., a preclinical-stage biopharmaceutical company, focuses on developing therapies to overcome tumor resistance and enhance outcomes for patients with cancer. Its lead product candidate is TYRA-300, a selective inhibitor of fibroblast growth factor receptor (FGFR)3 for the treatment of muscle invasive bladder cancer. The company is also developing programs targeting FGFR2- intrahepatic cholangiocarcinoma,FGFR3-related achondroplasia, REarranged during transfection kinase, and FGFR4-related cancers. In addition, the company offers SNAP platform which enable rapid structural design through iterative molecular SNAPshots. Tyra Biosciences, Inc. was incorporated in 2018 and is based in Carlsbad, California.

Phathom Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada to vonoprazan, a potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach. It is also developing vonoprazan, which is in Phase III clinical trials for the treatment of erosive gastroesophageal reflux disease; and in combination with antibiotics for the treatment of Helicobacter pylori infection. Phathom Pharmaceuticals, Inc. was incorporated in 2018 and is headquartered in Florham Park, New Jersey.

Day One Biopharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops and commercializes targeted therapies for patients with genetically defined cancers. Its lead product candidate is DAY101, an oral brain-penetrant type II pan-rapidly accelerated fibrosarcoma kinase inhibitor that is in Phase II clinical trial for pediatric patients with relapsed/progressive low-grade glioma. The company is also developing Pimasertib, an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2. Day One Biopharmaceuticals, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

Innoviva, Inc. engages in the development and commercialization of pharmaceuticals in the United States and internationally. Its products include RELVAR/BREO ELLIPTA, a once-daily combination medicine consisting of a LABA, vilanterol (VI), an inhaled corticosteroid (ICS), and fluticasone furoate; ANORO ELLIPTA, a once-daily medicine combining a long-acting muscarinic antagonist (LAMA), umeclidinium bromide (UMEC), with a LABA, and VI; and TRELEGY ELLIPTA, a once-daily combination medicine consisting of an ICS, LAMA, and LABA. Innoviva, Inc. has a strategic partnership with Sarissa Capital Management LP. The company has long-acting beta2 agonist (LABA) collaboration agreement with Glaxo Group Limited to develop and commercialize once-daily products for the treatment of chronic obstructive pulmonary disease and asthma. The company was formerly known as Theravance, Inc. and changed its name to Innoviva, Inc. in January 2016. Innoviva, Inc. was incorporated in 1996 and is headquartered in Burlingame, California.

Enliven Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery and development of small molecule inhibitors to help patients with cancer. Its pipeline of small molecule kinase inhibitors include ELVN-001, which is being evaluated in a Phase 1 clinical trial in adults with chronic myeloid leukemia; and ELVN-002, a Phase 1 clinical trial has been activated to evaluate people with cancers harboring an abnormal HER2 gene. The company is based in Boulder, Colorado.

Cogent Biosciences, Inc., a biotechnology company, focuses on developing precision therapies for genetically defined diseases. Its lead product candidate includes CGT9486, a selective tyrosine kinase inhibitor designed to inhibit the KIT D816V mutation that drives systemic mastocytosis, as well as other mutations in KIT exon 17, which are found in patients with advanced gastrointestinal stromal tumors. It has a licensing agreement with Plexxikon Inc. for the research, development, and commercialization of bezuclastinib. The company was formerly known as Unum Therapeutics Inc. and changed its name to Cogent Biosciences, Inc. in October 2020. Cogent Biosciences, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

Immatics N.V., a clinical-stage biopharmaceutical company, focuses on the discovery and development of T cell receptor (TCR) based immunotherapies for the treatment of cancer in the United States. The company is developing targeted immunotherapies with a focus on treating solid tumors through two distinct treatment modalities, such as adoptive cell therapies (ACT) and antibody-like TCR Bispecifics. Its ACTengine product candidates are in Phase I clinical trials, which include IMA201 that targets melanoma-associated antigen 4 or 8 in patients with solid tumors; IMA202 that targets melanoma-associated antigen 1 in patients with various solid tumors, including squamous non-small cell lung carcinoma and hepatocellular carcinoma; and IMA203 that targets preferentially expressed antigen in melanoma in adult patients with relapsed and/or refractory solid tumors, as well as IMA204, which is in preclinical studies that targets tumor stroma cell. The company's TCR Bispecifics product candidates, which are in preclinical studies include IMA401, a cancer testis antigen for the treatment of solid tumors; and IMA402 for the treatment of solid tumors. It also develops IMA101 for the treatment of cancer; and IMA301, an allogenic cellular therapy product candidate. The company has a strategic collaboration agreement with GlaxoSmithKline Intellectual Property Development Limited to develop novel adoptive cell therapies targeting multiple cancer indications; MD Anderson Cancer Center to develop multiple T cell and TCR-based adoptive cellular therapies; Celgene Switzerland LLC to develop novel adoptive cell therapies targeting multiple cancers; and Genmab A/S to develop T cell engaging bispecific immunotherapies targeting multiple cancer indications. Immatics N.V. is headquartered in Tübingen, Germany.

Gyre Therapeutics Inc is a biopharmaceutical company headquartered in San Diego, CA, that is primarily focused on the development and commercialization of Hydronidone (F351) for the treatment of Metabolic Dysfunction Associated Steatohepatitis (MASH-associated liver fibrosis (MASH fibrosis), formerly known as Nonalcoholic Steatohepatitis (NASH)) in the United States. In the United States, Hydronidone is currently being evaluated for the treatment of liver fibrosis across a broad spectrum of chronic liver diseases under an active Investigational New Drug (IND) application. Gyre is also advancing a diverse pipeline in China through a controlling interest in the Beijing Continent, including pirfenidone, F573, F528, and F230.

Relay Therapeutics, Inc. operates as a clinical-stage precision medicines company. It engages in transforming the drug discovery process with an initial focus on enhancing small molecule therapeutic discovery in targeted oncology and genetic disease indications. The company's lead product candidates include RLY-4008, an oral small molecule inhibitor of fibroblast growth factor receptor 2 (FGFR2), which is in a first-in-human clinical trial for patients with advanced or metastatic FGFR2-altered solid tumors; RLY-2608, a lead mutant-PI3Ka inhibitor program that targets phosphoinostide 3 kinase alpha; and RLY-1971, an oral small molecule inhibitor of protein tyrosine phosphatase Src homology region 2 domain-containing phosphatase-2 that is in Phase 1 trial in patients with advanced solid tumors. It has collaboration and license agreements with D. E. Shaw Research, LLC to research certain biological targets through the use of D. E. Shaw Research computational modeling capabilities focused on analysis of protein motion to develop and commercialize compounds and products directed to such targets; and Genentech, Inc. for the development and commercialization of RLY-1971. The company was formerly known as Allostery, Inc. and changed its name to Relay Therapeutics, Inc. in December 2015. Relay Therapeutics, Inc. was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Arcutis Biotherapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing treatments for dermatological diseases. Its lead product candidate is ARQ-151, a topical roflumilast cream that has completed Phase III clinical trials for the treatment of plaque psoriasis and atopic dermatitis. The company is also developing ARQ-154, a topical foam formulation of roflumilast for the treatment of seborrheic dermatitis and scalp psoriasis; ARQ-252, a selective topical janus kinase type 1 inhibitor for hand eczema and vitiligo; and ARQ-255, a topical formulation of ARQ-252 designed to reach deeper into the skin in order to treat alopecia areata. The company was formerly known as Arcutis, Inc. and changed its name to Arcutis Biotherapeutics, Inc. in October 2019. Arcutis Biotherapeutics, Inc. was incorporated in 2016 and is headquartered in Westlake Village, California.

Travere Therapeutics, Inc., a biopharmaceutical company, focuses on the identification, development, commercialization, and delivering of therapies for the treatment of rare diseases. Its marketed products include Chenodal, a synthetic oral form of chenodeoxycholic acid for the treatment of radiolucent stones in gallbladders; Cholbam, a cholic acid capsule to treat pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, as well as for adjunctive treatment of patients with peroxisomal disorders; and Thiola and Thiola EC, a tiopronin tablet for the treatment of homozygous cystinuria. The company's product candidates also consist of Sparsentan, which is in Phase III clinical trial for the treatment of focal segmental glomerulosclerosis and immunoglobulin A nephropathy; and TVT-058, a novel investigational human enzyme replacement candidate, which is in Phase I/II clinical trials for the treatment of classical homocystinuria. It has a cooperative research and development agreement with National Institutes of Health's National Center for Advancing Translational Sciences and patient advocacy organizations, CDG Care, and Alagille Syndrome Alliance for the identification of potential small molecule therapeutics for NGLY1 deficiency and Alagille syndrome. The company was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in November 2020. Travere Therapeutics, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

Wave Life Sciences Ltd., a clinical stage genetic medicine company, designs, optimizes, and produces novel stereopure oligonucleotides through PRISM, a discovery and drug developing platform. It is developing oligonucleotides target ribonucleic acid to reduce the expression of disease-promoting proteins or restore the production of functional proteins, or modulate protein expression. The company also develops WVE-004, a C9orf72 molecule for the treatment of amyotrophic lateral sclerosis and frontotemporal dementia; WVE-003, a mutant huntingtin SNP3 molecule for the treatment of Huntington's disease; WVE-N531, an Exon 53 molecule for the treatment of Duchenne muscular dystrophy; and ATXN3, a discovery stage program for the treatment of spinocerebellar ataxia 3, as well as multiple preclinical programs for CNS disorders. In addition, it focuses on developing GalNAc-conjugated AIMers to treat hepatic indications comprising Alpha-1 antitrypsin deficiency (AATD); and two preclinical programs, such as Usher syndrome type 2A (USH2A) and retinitis pigmentosa due to a P23H mutation in the RHO gene (RhoP23H) for the treatment of retinal diseases. It has collaboration agreements with Pfizer Inc., Takeda Pharmaceutical Company Limited, University of Oxford, University of Massachusetts, Western Washington University, Grenoble Institute of Neurosciences, IRBM S.p.A, University of Louisville, and University College London. The company was incorporated in 2012 and is based in Singapore.

Aurinia Pharmaceuticals Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies to treat various diseases with unmet medical need in the United States and internationally. The company offers LUPKYNIS for the treatment of adult patients with active lupus nephritis. It has a collaboration and license agreement with Otsuka Pharmaceutical Co., Ltd. The company is headquartered in Victoria, Canada.

Vir Biotechnology, Inc., a commercial-stage immunology company, develops therapeutic products to treat and prevent serious infectious diseases. It develops Sotrovimab (VIR-7832), a SARS-CoV-2-neutralizing mAbs to treat and prevent COVID-19 infection under the Xevudy brand; VIR-2218 and VIR-3434 for the treatment of hepatitis B virus; VIR-2482 for the prevention of influenza A virus; and VIR-1111 for the prevention of human immunodeficiency virus. The company has grant agreements with Bill & Melinda Gates Foundation and National Institutes of Health; an option and license agreement with Brii Biosciences Limited and Brii Biosciences Offshore Limited; a collaboration and license agreement with Alnylam Pharmaceuticals, Inc.; license agreements with The Rockefeller University and MedImmune, Inc.; collaboration with WuXi Biologics and Glaxo Wellcome UK Ltd.; and a collaborative research agreement with GlaxoSmithKline Biologicals SA. It also has a manufacturing agreement with Samsung Biologics Co.,Ltd. for the manufacture of SARS-COV-2 antibodies; and clinical collaboration with Gilead Sciences, Inc. for chronic hepatitis B virus. Vir Biotechnology, Inc. was incorporated in 2016 and is headquartered in San Francisco, California.

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, develops therapies for central nervous system disorders characterized by neuronal imbalance. Its lead product candidates include PRAX-114, an extrasynaptic-preferring GABAA receptor positive allosteric modulator that is in Phase IIa clinical trial for the treatment of major depressive disorder and perimenopausal depression; and PRAX-944, a selective small molecule inhibitor of T-type calcium channels, which is in Phase IIa clinical trial for the treatment of essential tremor. The company is also developing PRAX-562, a persistent sodium current blocker that is in Phase I clinical trial to treat severe pediatric epilepsy and adult cephalgia; PRAX-222, an antisense oligonucleotide (ASO) for patients with gain-of-function (GOF) SCN2A epilepsy; and KCNT1 program for the treatment of KCNT1 GOF epilepsy. It has a cooperation and license agreement with RogCon Inc.; a license agreement with Purdue Neuroscience Company; a research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and collaboration with The Florey Institute to develop three novel ASOs. The company was incorporated in 2015 and is based in Boston, Massachusetts.

AnaptysBio, Inc., a clinical stage biotechnology company, engages in developing therapeutic product candidates for inflammation and immuno-oncology indications. Its products include Imsidolimab, an antibody that inhibits the interleukin-36 receptor (IL-36R) for the treatment of various dermatological inflammatory diseases; Rosnilimab, an anti-PD-1 agonist antibody program designed to augment PD-1 signaling through rosnilimab treatment to suppress T-cell driven human inflammatory diseases; and ANB032, an anti-BTLA modulator antibody applicable to human inflammatory diseases associated with lymphoid and myeloid immune cell dysregulation. The company also focuses on developing various antibody programs that are advanced to preclinical and clinical milestones under its collaborations. It has a collaboration and license agreement with GlaxoSmithKline, Inc. and Bristol-Myers Squibb; and license agreements with United Kingdom Research and Innovation, as well as Millipore Corporation. The company was formerly known as Anaptys Biosciences, Inc. and changed its name to AnaptysBio, Inc. in July 2006. AnaptysBio, Inc. was incorporated in 2005 and is based in San Diego, California.

Pharvaris N.V., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for rare diseases. The company develops PHA121, a small molecule bradykinin B2-receptor antagonist that is in Phase II clinical trial for the treatment of hereditary angioedema (HAE). It also develops PHVS416, an on-demand, rapid exposure soft capsule for patients suffering from acute HAE attacks and is under Phase 2 clinical trial; and PHVS719, a prophylactic extended-release tablet for HAE patients and is under Phase 1 clinical trial. It operates in the Netherlands, Switzerland, and the United States. The company was incorporated in 2015 and is based in Leiden, the Netherlands.

Gracell Biotechnologies Inc., a clinical-stage biopharmaceutical company, primarily discovers and develops cell therapies for the treatment of cancer in the People's Republic of China. Its lead product candidates include GC012F, a FasTCAR-enabled dual BCMA- and CD19-directed autologous CAR-T product candidate that is in Phase I trial for the treatment of multiple myeloma; GC019F, a FasTCAR-enabled CD19-directed autologous CAR-T product candidate, which is in Phase I clinical trial for the treatment of adult B cell acute lymphoblastic leukemia (B-ALL), as well as in preclinical stage for the treatment of relapsed or refractory (r/r) B cell non-Hodgkin's lymphoma (B-NHL); and GC027, a TruUCAR-enabled CD7-directed allogeneic CAR-T product candidate, which is in Phase I clinical trial for the treatment of adult T cell acute lymphoblastic leukemia. The company's product candidates also comprise GC007g, a donor-derived CD19-directed allogeneic CAR-T cell therapy that is in Phase 1/2 registrational trial clinical trial for the treatment of r/r B-ALL; and GC502, a TruUCAR-enabled dual CD19- and CD7 -directed, off-the-shelf allogeneic CAR-T product candidate, which is in Phase I trial for the treatment of B-cell malignancies. In addition, it has a portfolio of earlier stage product candidates targeting various cancer indications, such as ovarian cancer, breast cancer, peripheral T cell lymphoma, a subtype of NHL, and T cell lymphoblastic leukemia. The company was founded in 2017 and is headquartered in Suzhou, China.

IGM Biosciences, Inc., a biotechnology company, develops Immunoglobulin M (IgM) antibodies for the treatment of cancer, infectious diseases, and autoimmune and inflammatory diseases. The company's lead product candidate is IGM-2323, a bispecific IgM antibody that is in Phase 2 clinical trials to treat patients with relapsed/refractory B cell Non-Hodgkin's lymphoma (NHL). It is also developing IGM-8444, an IgM antibody, which is in Phase 1 clinical trials for the treatment of patients with solid cancers; and IGM-7354, an anti-PD-L1 IgM antibody for the treatment of patients with solid and hematologic malignancies; IGM-2644, a bispecific T cell engaging IgM antibody targeting CD38 and CD3 proteins for the treatment of patients with multiple myeloma; and IGM-2537, a bispecific T cell engaging IgM antibody targeting CD123 and CD3 proteins for the treatment of patients with acute myeloid leukemia, myelodysplastic syndromes, and acute lymphoblastic leukemia. It has a collaboration and license agreement with Genzyme Corporation to generate, develop, manufacture, and commercialize IgM antibodies. The company was formerly known as Palingen, Inc. and changed its name to IGM Biosciences, Inc. in 2010. IGM Biosciences, Inc. was incorporated in 1993 and is headquartered in Mountain View, California.

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel products to target cardiovascular disease, galactosemia, and diabetic complications. Its lead product candidate is AT-007 that has completed phase 1/2 for the treatment of galactosemia in healthy volunteers and adults, as well as is in pediatric clinical study for the treatment of galactosemia in kids; for treating sorbitol dehydrogenase deficiency; and for the treatment of phosphomannomutase enzyme-CDG. The company also develops AT-001 that is in phase III clinical trials for treating diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy. Its preclinical stage products include AT-003 for the treatment diabetic retinopathy; AT-104, a PI3K inhibitor for treating orphan hematological oncology T Cell Acute Lymphoblastic Leukemia. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

Cullinan Oncology Inc., a biopharmaceutical company, focuses on developing a pipeline of targeted oncology and immuno-oncology therapies for cancer patients in the United States. The company's lead candidate is the CLN-081, an orally available small molecule that is in a Phase I/IIa dose escalation and expansion trial for treating patients with non-small cell lung cancer. Its preclinical products include CLN-049, a humanized bispecific antibody for the treatment of acute myeloid leukemia; CLN-619, a monoclonal antibody for the treatment of solid tumors; CLN-617, a fusion protein for the treatment of solid tumors; CLN-978, a T cell engaging antibody for B-cell malignancies; Opal, a bispecific fusion protein to block the PD-1 axis, and to activate the 4-IBB/CD137 pathway on T cells in tumors; and Jade, a cell therapy to target a novel senescence and cancer-related protein. The company has collaboration agreements with Cullinan Pearl Corp. to develop, manufacture, and commercialize CLN-081 and products containing CLN-081; and Adimab, LLC to discover and/or optimize antibodies. Cullinan Oncology, Inc. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Autolus Therapeutics plc, a clinical-stage biopharmaceutical company, develops T cell therapies for the treatment of cancer. The company's clinical-stage programs include obecabtagene autoleucel (AUTO1), a CD19-targeting programmed T cell investigational therapy that is in Phase 1b/2 clinical trial for the treatment of adult ALL; AUTO1/22, which is in a Phase 1 clinical trial in pediatric patients with relapsed or refractory ALL; AUTO4, a programmed T cell investigational therapy for the treatment of peripheral T-cell lymphoma targeting TRBC1; AUTO6NG, a programmed T cell investigational therapy, which is in preclinical trail targeting GD2 in development for the treatment of neuroblastoma; and AUTO8, a product candidate that is in a Phase I clinical trial for multiple myeloma. It also focuses on developing AUTO5, a hematological product candidate, which is in preclinical development. The company was incorporated in 2014 and is headquartered in London, the United Kingdom.

Sana Biotechnology, Inc., a biotechnology company, focuses on utilizing engineered cells as medicines. The company develops ex vivo and in vivo cell engineering platforms for various therapeutic areas with unmet treatment needs, including oncology, diabetes, central nervous system disorders, cardiovascular diseases, genetic disorders, and others. Its product candidates include SG295 and SG242 that target CD19+ cancer cells, including non-Hodgkin Lymphoma, chronic lymphocytic leukemia, and acute lymphoblastic leukemia; SG221 and SG239 for the treatment of multiple myeloma; and SG328 for ornithine transcarbamylase deficiency. It also develops SG418 for sickle cell disease and beta-thalassemia; SC291, a CD19 allogeneic T cell therapy; SC255 for multiple myeloma; SC451 for type I diabetes mellitus; and SC379 for secondary progressive multiple sclerosis, Pelizaeus-Merzbacher disease, and Huntington's disease. Sana Biotechnology, Inc. was formerly known as FD Therapeutics, Inc. and changed its name to Sana Biotechnology, Inc. in September 2018. The company was incorporated in 2018 and is headquartered in Seattle, Washington.

Prothena Corporation plc, a late-stage clinical company, focuses on discovery and development of novel therapies for life-threatening diseases in the United States. The company is involved in developing Birtamimab, an investigational humanized antibody that is in Phase III clinical trial for the treatment of AL amyloidosis; Prasinezumab, a humanized monoclonal antibody, which is in Phase IIb clinical trial for the treatment of Parkinson's disease; PRX004 that completed Phase I clinical trial for the treatment of Transthyretin amyloidosis; and PRX005, which is in Phase I clinical trial for the treatment of Alzheimer's disease. Its discovery and preclinical programs include PRX012 for the treatment of Alzheimer's disease; and dual Aß-Tau vaccine for the treatment and prevention of Alzheimer's disease. Prothena Corporation plc has a license, development, and commercialization agreement with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to develop and commercialize antibodies that target alpha-synuclein; and a collaboration agreement with Bristol-Myers Squibb to develop antibodies. The company was founded in 2012 and is based in Dublin, Ireland.

Zymeworks Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes biotherapeutics for the treatment of cancer. The company's lead product candidates include zanidatamab, a novel bispecific antibody that is in Phase 1 and Phase 2 clinical trials for the treatment of biliary tract, gastroesophageal adenocarcinomas, breast, and colorectal cancer; and ZW49, a biparatopic anti-human epidermal growth factor receptor 2 (HER2) antibody-drug conjugate that is in Phase 1 clinical trial for the treatment of advanced or metastatic HER2-expressing tumors. The company has strategic partnerships with Merck Sharp & Dohme Research Ltd.; Eli Lilly and Company; Bristol-Myers Squibb company; GlaxoSmithKline Intellectual Property Development Ltd.; Daiichi Sankyo Co., Ltd.; Janssen Biotech, Inc.; BeiGene, Ltd.; and Exelixis, Inc. It also has licensing and research collaboration with LEO Pharma A/S to research, develop, and commercialize bispecific antibodies; and Iconic Therapeutics, Inc. Zymeworks Inc. was incorporated in 2003 and is headquartered in Vancouver, Canada.

Mesoblast Limited, a biopharmaceutical company, develops and commercializes allogeneic cellular medicines in the United States, Australia, Singapore, the United Kingdom, and Switzerland. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage cells. The company's products under the Phase III clinical trials include remestemcel-L for the treatment of steroid refractory acute graft versus host disease, as well as acute respiratory distress syndrome due to COVID-19 infection; Rexlemestrocel-L to treat advanced chronic heart failure; and MPC-06-ID for chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV for the treatment of biologic refractory rheumatoid arthritis diabetic nephropathy. The company has strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. for the treatment of wound healing in patients with epidermolysis bullosa; and Grünenthal to develops and commercializes cell therapy for the treatment of chronic low back pain. Mesoblast Limited was incorporated in 2004 and is headquartered in Melbourne, Australia.

Immunome, Inc., a biopharmaceutical company, discovers and develops antibody therapeutics for oncology and infectious disease. The company's lead oncology program includes IMM-ONC-01, which targets IL-38 tumor-derived immune checkpoint capable of promoting evasion of the immune system. It also develops IMM-BCP-01, an antibody cocktail product candidate for the treatment of SARS-CoV-2 infections and COVID-19. The company was incorporated in 2006 and is headquartered in Exton, Pennsylvania.

MiMedx Group, Inc. develops and distributes placental tissue allografts for various sectors of healthcare. It processes the human placental tissues utilizing its patented and proprietary PURION process to produce allografts that retains the tissue's inherent biological properties and regulatory proteins. The company's patented and proprietary processing method employs aseptic processing techniques in addition to terminal sterilization. Its products include EpiFix, a semi-permeable protective barrier membrane product used for the treatment of chronic wounds, including diabetic foot ulcers, venous leg ulcers, and pressure ulcers; AmnioFix, a semi-permeable protective barrier allograft, which comprises dehydrated human amnion/chorion membrane for use in surgical recovery applications; EpiCord and AmnioCord are dehydrated human umbilical cord allografts that are used to provide a protective environment for the healing process, as well as used in the advanced wound care and surgical recovery applications; and AMNIOBURN a semi-permeable protective barrier allograft used in the treatment of partial-thickness and full-thickness burns, as well as lead product includes mdHACM, a micronized form of AMNIOFIX, supplied in powder form. The company's products have applications primarily in the areas of wound care, burn, surgical, and non-operative sports medicine sectors of healthcare. It also sells allografts for dental applications on an original equipment manufacturer basis. The company sells its products through direct sales force and independent sales agents, as well as through independent distributors primarily in the United States. MiMedx Group, Inc. is headquartered in Marietta, Georgia.

Silence Therapeutics plc, a biotechnology company, focuses on the discovery and development of novel ribonucleic acid (RNA) therapeutics in hematology, cardiovascular, and other rare and metabolic indications. The company's platform includes mRNAi GalNAc Oligonucleotide Discovery platform designed to accurately target specific disease-associated genes in the liver. It designs short interfering RNA molecules to harness the body's natural mechanism of RNA interference, and degrading messenger RNA molecules that encode specific targeted disease-associated proteins in a cell. The company is developing various product candidates, including SLN360, which is Phase I clinical trials for the treatment of cardiovascular disease with high lipoprotein; SLN124 that is in Phase I clinical trials for the treatment of non-transfusion dependent thalassemia, and Phase I clinical trials for the treatment of myelodysplastic syndrome; and SLN124 for the treatment of polycythemia vera. It has collaboration agreements with AstraZeneca PLC to discover, develop, and commercialize small interfering RNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases; and Mallinckrodt Pharma IP Trading DAC to develop and commercialize RNAi drug targets designed to silence the complement cascade in complement-mediated disorders. The company also has a collaboration with Hansoh Pharmaceutical Group Company Limited to develop siRNAs for three undisclosed targets leveraging Silence's mRNAi GOLD platform. The company was formerly known as SR Pharma plc and changed its name to Silence Therapeutics plc in May 2007. Silence Therapeutics plc is headquartered in London, the United Kingdom.

CARGO Therapeutics, Inc., a clinical-stage biotechnology company, develops chimeric antigen receptor (CAR) T-cell therapies for cancer patients. The company's lead program is CRG-022, an autologous CD22 CAR T-cell product candidate designed to address resistance mechanisms by targeting CD22, an alternate tumor antigen that is expressed in B-cell malignancies. It also develops CRG-023, a tri-specific CAR T product candidate that targets tumor cells with three B-cell antigen targets. The company was formerly known as Syncopation Life Sciences, Inc. and changed its name to CARGO Therapeutics, Inc. in September 2022. CARGO Therapeutics, Inc. was incorporated in 2019 and is headquartered in San Mateo, California.

Liquidia Corporation, a biopharmaceutical company, develops, manufactures, and commercializes various products for unmet patient needs in the United States. Its product candidates include YUTREPIA, an inhaled dry powder formulation of treprostinil for the treatment of pulmonary arterial hypertension. It also distributes generic treprostinil injection in the United States. Liquidia Corporation was founded in 2004 and is headquartered in Morrisville, North Carolina.

Tango Therapeutics, Inc., a biotechnology company, discovers and develops drugs for the treatment of cancer. Its lead program is TNG908, a synthetic lethal small molecule inhibitor of protein arginine methyltransferase 5 that is being developed as a treatment for cancers with methylthioadenosine phosphorylase deletions. The company also develops Ubiquitin-specific protease 1, an inhibitor to treat patients with BRCA1 or BRCA2-mutant cancers; and Target 3 for STK11-mutant cancers. Tango Therapeutics, Inc. has a strategic collaboration with Gilead Sciences, Inc. for the discovery, development, and commercialization of a pipeline of therapies for patients with cancer. The company was founded in 2017 and is based in Cambridge, Massachusetts.

Dianthus Therapeutics, Inc. operates as a clinical-stage biotechnology company that designs, develops, and delivers novel monoclonal antibodies for people living with severe autoimmune and inflammatory diseases. It develops DNTH103 that is in phase 1 clinical trails in patients with generalized myasthenia gravis, multifocal motor neuropathy, and chronic inflammatory demyelinating polyneuropathy. Dianthus Therapeutics, Inc. was incorporated in 2015 and is based in New York, New York.

ArriVent BioPharma, Inc. operates as a clinical-stage biopharmaceutical company that engages in the identification, development, and commercialization of medicines for the unmet medical needs of patients with cancers. It also engages in the development and commercialization of targeted cancer therapies for non-small-cell lung cancer (NSCLC) and other solid tumors. The company develops Furmonertinib, an epidermal growth factor receptor mutant-selective tyrosine kinase inhibitor that is in phase 3 clinical trial for the treatment of NSCLC patients; and ARR-002. It has strategic collaborations with Aarvik Therapeutics Inc. The company was incorporated in 2021 and is based in Newtown Square, Pennsylvania.

89bio, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for the treatment of liver and cardio-metabolic diseases. Its lead product candidate is pegozafermin, a glycoPEGylated analog of fibroblast growth factor 21 for the treatment of nonalcoholic steatohepatitis. The company also intends to develop pegozafermin for the treatment of severe hypertriglyceridemia. 89bio, Inc. was incorporated in 2018 and is headquartered in San Francisco, California.

Erasca, Inc., a clinical-stage biopharmaceutical company, focuses on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers. The company's lead candidates include ERAS-007, an oral inhibitor of ERK1/2 for the treatment of non-small cell lung cancer, colorectal cancer, and acute myeloid leukemia; and ERAS-601, an oral SHP2 inhibitor for patients with advanced or metastatic solid tumors. It is also developing ERAS-801, a central nervous system-penetrant EGFR inhibitor for the treatment of patients with recurrent glioblastoma multiforme. The company was incorporated in 2018 and is headquartered in San Diego, California.

AbCellera Biologics Inc. develops antibody discovery platform. Its full-stack, artificial intelligence-powered antibody discovery platform searches and analyzes the database of natural immune systems to find antibodies that could be developed as drugs. As of December 31, 2021, the company had 156 discovery programs that are either completed, in progress, or under contract with 36 partners. AbCellera Biologics Inc. has a research collaboration and license agreement with Eli Lilly and Company. The company was incorporated in 2012 and is headquartered in Vancouver, Canada.

Nuvation Bio Inc., a clinical-stage biopharmaceutical company, focuses on the development of therapeutic candidates for oncology. The company's lead product candidate is NUV-422, a small molecule inhibitor targeting cyclin-dependent kinase (CDK)2, CDK4, and CDK6. It is also developing NUV-868, a selective oral small molecule BET inhibitor that epigenetically regulates proteins that control tumor growth and differentiation; NUV-569, a differentiated oral small molecule selective inhibitor of the Wee1 kinase for DNA damage repair; NUV-1182, an adenosine receptor inhibitor; and drug-drug conjugate (DDC) platform that focuses on targeting an inhibitor of poly ADP ribose polymerase (PARP) to anti-cancer warheads of existing drugs, as well as PARP inhibitor to address ER+ breast and ovarian cancer. The company was formerly known as RePharmation Inc. and changed its name to Nuvation Bio Inc. in April 2019. Nuvation Bio Inc. was founded in 2018 and is headquartered in New York, New York.

Adaptive Biotechnologies Corporation, a commercial-stage company, develops an immune medicine platform for the diagnosis and treatment of various diseases. The company offers immunoSEQ, a platform and core immunosequencing product that is used to answer translational research questions, as well as to discover new prognostic and diagnostic signals; and T-Detect COVID for the confirmation of past COVID-19 infection. It also provides clonoSEQ, a clinical diagnostic product for the detection and monitoring of minimal residual disease in patients with multiple myeloma, B cell acute lymphoblastic leukemia, and chronic lymphocytic leukemia, as well as available as a CLIA-validated laboratory developed test for patients with other lymphoid cancers; and immunoSEQ T-MAP COVID for vaccine developers and researchers to measure the T-cell immune response to vaccines. In addition, the company offers a pipeline of clinical products and services that are used for the diagnosing, monitoring, and treatment of diseases, such as cancer, autoimmune conditions, and infectious diseases. It serves the life sciences research, clinical diagnostics, and drug discovery applications. Adaptive Biotechnologies Corporation has strategic collaborations with Genentech, Inc. for the development, manufacture, and commercialization of neoantigen directed T cell therapies for the treatment of a range of cancers; and Microsoft Corporation to develop diagnostic tests for the early detection of various diseases from a single blood test. The company was formerly known as Adaptive TCR Corporation and changed its name to Adaptive Biotechnologies Corporation in December 2011. Adaptive Biotechnologies Corporation was incorporated in 2009 and is headquartered in Seattle, Washington.

Replimune Group, Inc., a biotechnology company, develops oncolytic immuno-gene therapies to treat cancer. It uses its proprietary Immunotherapy platform to design and develop product candidates that are intended to activate the immune system against cancer. The company's lead product candidate is RP1, a selectively replicating version of herpes simplex virus 1, which is in Phase I/II clinical trials for a range of solid tumors; and that is in Phase II clinical trials for patients with cutaneous squamous cell carcinoma. It is also developing RP2, which is in Phase I clinical trials for an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 that is in Phase I clinical trials to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

ABIVAX Société Anonyme, a clinical-stage biotechnology company, focuses on developing therapeutics that harness the body's natural regulatory mechanisms to modulate the immune response in patients with chronic inflammatory diseases. The company is evaluating its lead drug candidate, obefazimod, in Phase 3 clinical trials for the treatment of moderately to severely active ulcerative colitis. ABIVAX Société Anonyme was incorporated in 2013 and is headquartered in Paris, France.

Arbutus Biopharma Corporation, a biopharmaceutical company, develops novel therapeutics for chronic Hepatitis B virus (HBV) infection, SARS-CoV-2, and other coronaviruses in the United States. Its HBV product pipeline consists of AB-729, a proprietary subcutaneously delivered RNA interference product candidate, which in Phase Ia/Ib clinical trial targeted to hepatocytes that inhibits viral replication and reduces various HBV antigens using novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology; and AB-836, an oral capsid inhibitor that suppresses HBV DNA replication. The company's research and development programs include AB-161, an oral HBV RNA destabilizer to destabilize HBV RNA, which leads in the reduction of HBsAg and other viral proteins; AB-101, an oral PD-L1 inhibitor to reawaken patients' HBV-specific immune response; and small molecule antiviral medicines to treat coronaviruses, including COVID-19. It has strategic alliance, licensing, and research collaboration agreements with Talon Therapeutics, Inc.; Gritstone Oncology, Inc.; Alnylam Pharmaceuticals, Inc.; Qilu Pharmaceuticals Co, Ltd.; Assembly Biosciences, Inc.; Acuitas Therapeutics, Inc.; and Antios Therapeutics, Inc. Arbutus Biopharma Corporation also has a clinical collaboration agreement with Vaccitech plc to evaluate a triple combination of AB-729 for the treatment of chronic HBV infection. The company was formerly known as Tekmira Pharmaceuticals Corporation and changed its name to Arbutus Biopharma Corporation in July 2015. Arbutus Biopharma Corporation is headquartered in Warminster, Pennsylvania.

Avid Bioservices, Inc., a contract development and manufacturing organization, provides process development and current good manufacturing practices (CGMP) clinical and commercial manufacturing services focused on biopharmaceutical drug substances derived from mammalian cell culture. The company produces monoclonal antibodies and recombinant proteins; and offers services, including CGMP clinical and commercial drug substance manufacturing, bulk packaging, release and stability testing, and regulatory submission and support. It also provides various process development services, such as upstream and downstream development and optimization, analytical methods development, testing, and characterization. The company serves biotechnology and biopharmaceutical industries. The company was formerly known as Peregrine Pharmaceuticals, Inc. and changed its name to Avid Bioservices, Inc. in January 2018. Avid Bioservices, Inc. was incorporated in 1981 and is headquartered in Tustin, California.

ORIC Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapies for treatment of cancers in the United States. Its clinical stage product candidates include ORIC-533, an orally bioavailable small molecule inhibitor of CD73 being developed for resistance to chemotherapy- and immunotherapy-based treatment regimens; ORIC-944, an allosteric inhibitor of the polycomb repressive complex 2 for prostate cancer; and ORIC-114, a brain penetrant orally bioavailable irreversible inhibitor designed to selectively target epidermal growth factor receptor and human epidermal growth factor receptor 2 with high potency towards exon 20 insertion mutations. The company is also developing multiple discovery stage precision medicines targeting other cancer resistance mechanisms. It has a license and collaboration agreement with Voronoi Inc.; and a license agreement with Mirati Therapeutics, Inc. The company was incorporated in 2014 and is headquartered in South San Francisco, California.

Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom and internationally. Its lead product candidate, etigilimab (OMP-313M32), an antibody T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1b clinical trial for the treatment of tumor. The company is also developing Navicixizumab (OMP-305B83), which has completed Phase 1b clinical trials for the treatment of the late line ovarian cancer; Acumapimod (BCT-197), a p38 MAP kinase inhibitor that is in Phase II clinical trials to treat acute exacerbations of chronic obstructive pulmonary disease; and Leflutrozole (BGS-649), an oral aromatase inhibitor for the treatment of hypogonadotropic hypogonadism. In addition, it develops rare disease product candidates, including Setrusumab (BPS-804), a novel antibody for the treatment of osteogenesis imperfecta; and Alvelestat (MPH-966), an oral small molecule that is in Phase II clinical trial to treat Alpha-1 antitrypsin deficiency. The company has a collaboration with The University of Texas MD Anderson Cancer Center to evaluate anti-TIGIT therapeutic antibody candidate, etigilimab. Mereo BioPharma Group plc was incorporated in 2015 and is based in London, the United Kingdom.

Terns Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and obesity. The company develops TERN-101, a liver-distributed and non-bile acid farnesoid X receptor agonist, which is in Phase IIa clinical trial for the treatment of NASH; and TERN-201, a vascular adhesion protein-1 inhibitor that is in Phase Ib clinical trial for the treatment of NASH. It also develops TERN-501, a thyroid hormone receptor beta agonist with enhanced liver distribution and metabolic stability that is in Phase I clinical trial for the treatment of NASH; and TERN-601, a small-molecule Glucagon-Like Peptide-1 receptor agonist program that is intended to orally be administered for NASH and metabolic diseases, such as obesity. The company was incorporated in 2016 and is headquartered in Foster City, California.

Savara Inc., a clinical stage biopharmaceutical company, focuses on rare respiratory diseases. Its lead product candidate is molgramostim, an inhaled granulocyte-macrophage colony-stimulating factor, which is in Phase III development stage for the treatment of autoimmune pulmonary alveolar proteinosis. The company is headquartered in Austin, Texas.

Olema Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies for women's cancers. Its lead product candidate is OP-1250, an estrogen receptor (ER) antagonist and a selective ER degrader, which is in Phase 1/2 clinical trial for the treatment of recurrent, locally advanced, or metastatic estrogen receptor-positive, human epidermal growth factor receptor 2-negative breast cancer. The company was formerly known as CombiThera, Inc. and changed its name to Olema Pharmaceuticals, Inc. in March 2009. Olema Pharmaceuticals, Inc. was incorporated in 2006 and is headquartered in San Francisco, California.

Pliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States. Its lead product candidate is PLN-74809, an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins, which is in three Phase 2a trials. The company also develops PLN-1474, a small-molecule selective inhibitor of avß1, which completed Phase 1 clinical trial for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis. In addition, it is developing two additional preclinical integrin-based programs, which include an oncology program, as well as a program for an allosteric agonistic monoclonal antibody against an undisclosed integrin receptor for treatment of muscular dystrophies, including duchenne muscular dystrophy. Pliant Therapeutics, Inc. was incorporated in 2015 and is based in South San Francisco, California.

Opthea Limited, a clinical stage biopharmaceutical company, develops and commercializes therapies primarily for eye disease in Australia. The company's development activities are based on the intellectual property portfolio covering Vascular Endothelial Growth Factors (VEGF) VEGF-C, VEGF-D, and VEGF Receptor-3 for the treatment of diseases associated with blood and lymphatic vessel growth, as well as vascular leakage. Its lead asset is OPT 302, a soluble form of VEGFR 3 in clinical development as a novel therapy for wet neovascular age related macular degeneration and diabetic macular edema DME, as well as a first in class VEGF C/D inhibitors for treatment with VEGF A inhibitors for the treatment of wet neovascular AMD and other retinal diseases. The company was formerly known as Circadian Technologies Limited and changed its name to Opthea Limited in December 2015. Opthea Limited was incorporated in 1984 and is based in South Yarra, Australia.

Zenas BioPharma, Inc., a clinical-stage biopharmaceutical company, engages in the development and commercialization of transformative immunology-based therapies. Its lead product candidate is obexelimab, a bifunctional monoclonal antibody for various indications, including immunoglobulin G4-related disease, multiple sclerosis, systemic lupus erythematosus, and warm autoimmune hemolytic anemia. The company also develops ZB002, an anti-TNFa monoclonal antibody; ZB004, a cytotoxic T-lymphocyte-associated antigen 4-immunoglobulin fusion; ZB001, an anti-insulin-like growth factor-1 receptor monoclonal antibody; and ZB005, an anti-active complement component 1s monoclonal antibody. Zenas BioPharma, Inc. was formerly known as Zenas BioPharma (Cayman) Limited and changed its name to Zenas BioPharma, Inc. in August 2023. The company was incorporated in 2019 and is based in Waltham, Massachusetts.

CureVac N.V., a clinical-stage biopharmaceutical company, focuses on developing various transformative medicines based on messenger ribonucleic acid (mRNA). It is developing prophylactic vaccines, such as mRNA-based vaccine candidates CV2CoV, which is in Phase 1 clinical trial against SARS-CoV-2; CV7202, a prophylactic mRNA-based vaccine, which has completed Phase 1 clinical trial for rabies virus glycoprotein; and CVSQIV, which is in Phase 1 clinical trial against influenza, as well as vaccine for lassa fever, yellow fever, respiratiory syncytial virus, rotavirus, malaria, and universal influenza. The company also develops RNA-based cancer immunotherapies, including CV8102, which is in Phase 1 clinical trial for treating cutaneous melanoma and adenoidcystic carcinoma, as well as squamous cell cancer of skin, head, and neck. The company was founded in 2000 and is headquartered in Tübingen, Germany.

Tourmaline Bio, Inc. operates as a clinical biotechnology company that develops medicines for patients with life-altering immune diseases. It develops medicines and therapies for the Atherosclerotic cardiovascular disease (ASCVD) and Thyroid Eye Disease (TED). The company was incorporated in 2021 and is based in New York, New York.

Humacyte, Inc. engages in the development and manufacture of off-the-shelf, implantable, and bioengineered human tissues for the treatment of diseases and conditions across a range of anatomic locations in multiple therapeutic areas. The company using its proprietary and scientific technology platform to engineer and manufacture human acellular vessels (HAVs). Its investigational HAVs are designed to be easily implanted into any patient without inducing a foreign body response or leading to immune rejection. The company is developing a portfolio of HAVs, which would target the vascular repair, reconstruction, and replacement market, including vascular trauma; arteriovenous access for hemodialysis; peripheral arterial disease; and coronary artery bypass grafting, as well as developing its HAVs for pediatric heart surgery and cellular therapy delivery, including pancreatic islet cell transplantation to treat Type 1 diabetes. The company was founded in 2004 and is headquartered in Durham, North Carolina.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide (ASO) medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output to design ASOs to precisely upregulate protein expression. Its lead clinical candidate is STK-001, which is in phase I/IIa clinical trial to treat Dravet syndrome, a severe and progressive genetic epilepsy; and STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. It had entered into a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.

Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on the discovery and development of medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company develops Apitegromab, an inhibitor of the activation of latent myostatin that has completed the Phase 3 clinical trials for the treatment of spinal muscular atrophy; and SRK-181, which is in Phase 1 clinical trials for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies. It is also developing a pipeline of novel product candidates with potential to transform the lives of patients suffering from a range of serious diseases, including neuromuscular disorders, cancer, and fibrosis. The company has a collaboration agreement with Gilead Sciences, Inc. to discover and develop specific inhibitors of transforming growth factor beta activation for the treatment of fibrotic diseases. Scholar Rock Holding Corporation was founded in 2012 and is headquartered in Cambridge, Massachusetts.

Arcturus Therapeutics Holdings Inc., an RNA medicines company, focuses on the development of vaccines for infectious, and liver and respiratory rare diseases in the United States. The company's development programs comprise LUNAR-OTC development program for ornithine transcarbamylase (OTC) deficiency; and LUNAR-CF program for cystic fibrosis lung disease caused by mutations in cystic fibrosis transmembrane conductance regulator (CFTR) gene, as well as vaccine programs include LUNAR-COV19 and LUNAR-FLU. It has collaboration partnerships with Vinbiocare Biotechnology Joint Stock Company for the manufacture of COVID-19 vaccines; Janssen Pharmaceuticals, Inc. to develop nucleic acid-based therapeutic candidates for the treatment of hepatitis B virus; Ultragenyx Pharmaceutical, Inc. to develop mRNA therapeutic candidates for rare disease targets; CureVac AG to develop mRNA therapeutic and vaccine candidates for various indications; Singapore Economic Development Board and Duke-NUS Medical School to develop LUNAR-COV19 vaccine; and Millennium Pharmaceuticals, Inc. to discover siRNA medicines for the treatment of non-alcoholic steatohepatitis. The company was founded in 2013 and is headquartered in San Diego, California.

Annexon, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapeutics for autoimmune, neurodegenerative, and ophthalmic disorders. The company's C1q is an initiating molecule of the classical complement pathway that targets distinct disease processes, such as antibody-mediated autoimmune disease and complement-mediated neurodegeneration. Its product candidates include ANX005, a monoclonal antibody, which is in Phase II/III clinical trials to treat patients with guillain- barré syndrome; Phase II trial in patients with warm autoimmune hemolytic anemia; and Phase II clinical trial for Huntington's disease and amyotrophic lateral sclerosis. The company is also developing ANX009 that is in Phase Ib trial in patients with lupus nephritis; and ANX007, which is in Phase II clinical trials to treat patients with geographic atrophy. In addition, it develops ANX105, an investigational monoclonal antibody targeting neurodegenerative indications; and ANX1502, an investigational oral small molecule for the treatment of certain autoimmune indications. The company was incorporated in 2011 and is headquartered in Brisbane, California.

Astria Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutics for rare and niche allergic, and immunological diseases in the United States. Its lead product candidate is STAR-0215, a monoclonal antibody inhibitor of plasma kallikrein, which is in preclinical development stage for the treatment of hereditary angioedema. The company was formerly known as Catabasis Pharmaceuticals, Inc. and changed its name to Astria Therapeutics, Inc. in September 2021. Astria Therapeutics, Inc. was incorporated in 2008 and is based in Boston, Massachusetts.

Exscientia plc, an artificial intelligence-driven pharmatech company, engages in discovering, designing, and developing drugs. The company offers end-to-end solution of artificial intelligence (AI) and technologies for target identification, drug candidate design, translational models, and patient selection. In addition, the company focuses on small molecule drug candidates. Its platform enables to design candidate drug molecules, as well as to provide patients with drug therapies through AI guided assessment. The company was founded in 2012 and is headquartered in Oxford, the United Kingdom.

Mineralys Therapeutics, Inc., a clinical-stage biopharmaceutical company that develops therapies for the treatment of hypertension and associated cardiovascular diseases. It clinical-stage product candidate is lorundrostat, a proprietary, orally administered, highly selective aldosterone synthase inhibitor for the treatment of patients with uncontrolled or resistant hypertension. The company was incorporated in 2019 and is headquartered in Radnor, Pennsylvania.

Entrada Therapeutics, Inc., a biotechnology company, develops endosomal escape vehicle (EEV) therapeutics for the treatment of multiple neuromuscular diseases. Its endosomal escape vehicle platform develops a portfolio of oligonucleotide, antibody, and enzyme-based programs. The company's lead product candidate is ENTR-601-44, which is in preclinical trail for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1. It also engages in the development of EEV-PMO-CAG for the treatment of myotonic dystrophy type 1. The company was formerly known as CycloPorters, Inc. and changed its name to Entrada Therapeutics, Inc. in October 2017. Entrada Therapeutics, Inc. was incorporated in 2016 and is headquartered in Boston, Massachusetts.

Y-mAbs Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of novel antibody based therapeutic products for the treatment of cancer in the United States. It offers DANYELZA, a monoclonal antibody in combination with granulocyte-macrophage colony-stimulating factor for the treatment of pediatric patients with relapsed or refractory high-risk neuroblastoma. The company is also developing DANYELZA that is in Phase II clinical studies for the treatment of patients with first-line neuroblastoma, third-line neuroblastoma, and in relapsed osteosarcoma; GD2-GD3 vaccine that is in Phase II clinical trial for the treatment of Stage 4 high-risk neuroblastoma; and Omburtamab, a murine monoclonal antibody that is in clinical trials for the treatment of central nervous system/ leptomeningeal metastases from neuroblastoma, desmoplastic small round cell tumor, diffuse intrinsic pontine glioma, and other B7-H3 positive tumors. In addition, it is developing Nivatrotamab for the treatment of small cell lung cancer, as well as is in Phase I/II trial for the treatment of refractory GD2 positive adult and pediatric solid tumors; and Lu-omburtamab-DTPA that is in Phase I clinical development for the treatment of medulloblastoma and B7-H3 positive leptomeningeal metastases from solid tumors. The company has a license agreement with Memorial Sloan Kettering Cancer Center and Massachusetts Institute of Technology to develop and commercialize licensed products. Y-mAbs Therapeutics, Inc. was incorporated in 2015 and is headquartered in New York, New York.

Allogene Therapeutics, Inc., a clinical stage immuno-oncology company, develops and commercializes genetically engineered allogeneic T cell therapies for the treatment of cancer. It develops, manufactures, and commercializes UCART19, an allogeneic chimeric antigen receptor (CAR) T cell product candidate for the treatment of pediatric and adult patients with R/R CD19 positive B-cell ALL. The company also develops ALLO-501, an anti-CD19 allogeneic CAR T cell product candidate that is in Phase I clinical trial for the treatment of R/R non-Hodgkin lymphoma; and ALLO-501A, which is in Phase I/II clinical trial for the treatment R/R large B-cell lymphoma or transformed follicular lymphoma. In addition, it is developing ALLO-715, an allogeneic CAR T cell product candidate that is in a Phase I clinical trial for treating R/R multiple myeloma; ALLO-605, an allogeneic CAR T cell product candidate for the treatment of multiple myeloma; ALLO-647, an anti-CD52 monoclonal antibody; CD70 to treat renal cell cancer; ALLO-819, an allogeneic CAR T cell product candidates for the treatment of acute myeloid leukemia; and DLL3 for the treatment of small cell lung cancer and other aggressive neuroendocrine tumors. The company has license and collaboration agreements with Pfizer Inc.; Servier; Cellectis S.A.; and Notch Therapeutics Inc., as well as clinical trial collaboration agreement with SpringWorks Therapeutics, Inc. It also has a strategic collaboration agreement with The University of Texas MD Anderson Cancer Center for the preclinical and clinical investigation of allogeneic CAR T cell product candidates. The company was incorporated in 2017 and is headquartered in South San Francisco, California.

Lexicon Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of pharmaceutical products. Its orally-delivered small molecule drug candidates under development comprise Sotagliflozin that completed Phase III clinical trials for the for the treatment of heart failure and type 1 diabetes; and LX9211, which is in Phase II clinical development for the treatment of neuropathic pain. The company has strategic collaboration and license agreements with Bristol-Myers Squibb Company, and Genentech, Inc. Lexicon Pharmaceuticals, Inc. was incorporated in 1995 and is headquartered in The Woodlands, Texas.

4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.

Pharming Group N.V., a biopharmaceutical company, develops and commercialize protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs in the United States, Europe, and internationally. The company's lead product is Ruconest, a recombinant human C1 esterase inhibitor that is used for the treatment of acute hereditary angioedema. It also engages in the development of rhC1INH for the treatment of pre-eclampsia, acute kidney injury, and COVID-19; leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) to treat patients with activated PI3K delta syndrome; and alpha-glucosidase therapy for the treatment of pompe and fabry diseases. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema. Pharming Group N.V. is headquartered in Leiden, the Netherlands.

Third Harmonic Bio, Inc., a clinical-stage biopharmaceutical company, focuses on the development of the medicine for the treatment of allergic and inflammatory diseases. It develops THB001, an oral small-molecule KIT inhibitor for the treatment of chronic urticaria, a dermatologic disease driven by mast cell activation that results in red, itchy, painful welts, or hives, as well as for airway and gastrointestinal tract indications. The company was formerly known as Project IGE, Inc. and changed its name to Third Harmonic Bio, Inc. in June 2019. Third Harmonic Bio, Inc. was founded in 2019 and is headquartered in Cambridge, Massachusetts.

ProKidney Corp., a clinical-stage biotechnology, engages in developing cellular therapy candidates. It is developing Renal Autologous Cell Therapy, an autologous homologous cell admixture that is in a Phase III development program, as well as Phase II clinical trials for the treatment of moderate to severe diabetic kidney disease; and Phase I clinical trial for patients with congenital anomalies of the kidney and urinary tract. The company was founded in 2015 and is headquartered in Winston-Salem, North Carolina.

Celcuity Inc., a clinical stage biotechnology company, focuses on the development of molecularly targeted therapies for cancer patients in the United States. The company's CELsignia diagnostic platform uses a patient's living tumor cells to identify the specific abnormal cellular process driving a patient's cancer and the related targeted therapy for the treatment. Its drug candidate includes Gedatolisib, which selectively targets various class I isoforms of PI3K and mammalian target of rapamycin and focus on the treatment of patients with hormone receptor positive, HER2-negative, and advanced or metastatic breast cancer. The company is also developing CELsignia MP test, a qualitative laboratory developed test that measures HER2, c-Met, and PI3K signaling activity in breast and ovarian tumor cells. It had a license agreement with Pfizer, Inc. for the development and commercialization rights to Gedatolisib. Celcuity Inc. was founded in 2011 and is headquartered in Minneapolis, Minnesota.

Neurogene Inc. develops life-changing genetic medicines for patients and their families affected by neurological diseases. Its product candidate includes NGN-401, an investigational AAV9 gene therapy for the treatment of Rett syndrome; and NGN-101 to treat neuronal ceroid lipofuscinosis subtype 5 batten disease. The company is headquartered in New York, New York.

Revance Therapeutics, Inc., a biotechnology company, engages in the development, manufacture, and commercialization of neuromodulators for various aesthetic and therapeutic indications in the United States and internationally. The company's lead drug candidate is DaxibotulinumtoxinA for injection, which has completed phase III clinical trials for the treatment of glabellar (frown) lines and cervical dystonia; is in phase II clinical trials to treat upper facial lines, moderate or severe dynamic forehead lines, and moderate or severe lateral canthal lines; and has completed Phase II clinical trials for the treatment of adult upper limb spasticity and plantar fasciitis. It is also developing DaxibotulinumtoxinA in preclinical trial for the treatment of migraine, as well as a topical program for various indications; and OnabotulinumtoxinA, a biosimilar to BOTOX. The company has a collaboration and license agreement with Viatris Inc. to develop, manufacture, and commercialize onabotulinumtoxinA. The company was formerly known as Essentia Biosystems, Inc. and changed its name to Revance Therapeutics, Inc. in April 2005. Revance Therapeutics, Inc. was incorporated in 1999 and is headquartered in Nashville, Tennessee.

Q32 Bio Inc is a clinical-stage biotechnology company focused on developing novel biologics to effectively and safely restore healthy immune balance in patients with autoimmune & inflammatory diseases driven by pathological immune dysfunction. Q32 is advancing antibody-based therapeutic candidates designed to target two central pathways of adaptive & innate immunity. The adaptive immune system is largely composed of T- & B-cell mediated cellular & antibody responses, while the innate immune system is the body’s first line of defense employing leukocytes that are responsible for clearing pathogens and cellular debris and modulating T- & B-cell function. ADX-097, the lead product candidate from Q32’s complement inhibitor platform, is a humanized anti-C3d monoclonal antibody fusion protein.

UroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization novel solutions for specialty cancers and urothelial diseases. It offers RTGel, a polymeric biocompatible and reverse thermal gelation hydrogel to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102, which is in Phase III clinical trials for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial carcinoma and low-grade non-muscle invasive bladder cancer. It is also developing UGN-301 for the treatment of high-grade non-muscle invasive bladder cancer. The company has a license agreement with Allergan Pharmaceuticals International Limited for developing and commercializing pharmaceutical products that contain RTGel and clostridial toxins; Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; and strategic research collaboration with MD Anderson to advance investigational treatment for high-grade bladder cancer. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase III clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II;RGX-111, which is in Phase I/II clinical trial for treating mucopolysaccharidosis type I;RGX-181 which is in pre clinic stage for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease;RGX-202, to treat Duchenne muscular dystrophy which is in phase I/II clinical trial; and RGX-381, to treat the ocular manifestations of CLN2 disease which is in preclinical stage. REGENXBIO Inc. also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop novel gene therapies. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.

Foghorn Therapeutics Inc., a clinical-stage biopharmaceutical company, discovers and develops medicines targeting genetically determined dependencies within the chromatin regulatory system. The company uses its proprietary Gene Traffic Control platform to identify, validate, and potentially drug targets within the system. It is developing FHD-286, a small molecule inhibitor of the enzymatic activity of BRG1 and BRM for the treatment of metastatic uveal melanoma and relapsed and/or refractory acute myeloid leukemia and myelodysplastic syndrome; and FHD-609, a small molecule protein degrader for BRD9 to treat patients with synovial sarcoma. The company is also developing an enzymatic inhibitor and a protein degrader as selective modulators of BRM; and ARID1B selective modulators for the treatment of ovarian, endometrial, colorectal, bladder, and gastric cancers. It has a research collaboration and license agreement with Merck Sharp & Dohme Corp. to discover and develop novel oncology therapeutics against a transcription factor target; and with Loxo Oncology to create novel oncology medicines. Foghorn Therapeutics Inc. was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Oculis Holding AG, a clinical-stage biopharmaceutical company, develops novel topical treatments for ophthalmic diseases for both back- and front-of-the-eye. The company's lead candidate is OCS-01, a topical dexamethasone formulation, which is in Phase 3 clinical trials for the treatment of diabetic macular edema; OCS-02, a topical biologic candidate that is in Phase 2b clinical trials for the treatment for keratoconjunctivitis sicca, or dry eye disease; and OCS-05, a novel neuroprotective agent for acute optic neuritis and other neuro-ophtha disorders, such as glaucoma, diabetic retinopathy, geographic atrophy, and neurotrophic keratitis. The company is based in Zug, Switzerland.

KalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, discovers, develops, and commercializes small molecule protease inhibitors for diseases with unmet needs. The company's product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include KVD001, a plasma kallikrein inhibitor that completed a Phase II clinical trial for the treatment of DME; sebetralstat, which is initiation of the Phase 3 KONFIDENT trial as a potential oral, on-demand therapy for HAE attacks; KVD824, an oral product candidate for the treatment of HAE; and Factor XIIa, an oral inhibitor program which is in preclinical stage targets an enzyme in HAE. The company is headquartered in Cambridge, Massachusetts.

OmniAb, Inc., a biotechnology company, provides therapeutic antibody discovery technologies in the United States. The company's discovery platform provides industry partners access to the diverse antibody repertoires and screening technologies to enable discovery of next-generation therapeutics. Its OmniAb platform is the biological intelligence of proprietary transgenic animals, including OmniRat, OmniChicken, and OmniMouse that have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. The company's OmniFlic (transgenic rat) and OmniClic (transgenic chicken) address industry needs for bispecific antibody applications though a common light chain approach, and OmniTaur that features unique structural attributes of cow antibodies for complex targets. The company was founded in 2012 and is headquartered in Emeryville, California.

Capricor Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of spectrum of diseases and disorders. Its lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase III clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD); and CAP-1002, which is in Phase II clinical trial for the treatment of cytokine storm associated with SARS-CoV-2. The company also develops CAP-2003 that is in pre-clinical development for the treatment of trauma related injuries and conditions; and two vaccine candidates, which are in development stage for the potential prevention of COVID-19. It collaborates with Lonza Houston, Inc. for the clinical manufacturing of CAP-1002, its cell therapy candidate for the treatment of DMD and other indications. The company was founded in 2005 and is headquartered in San Diego, California.

PureTech Health plc, a clinical-stage biotherapeutics company, discovers, develops, and commercializes medicines for inflammatory, fibrotic and immunological, intractable cancers, lymphatic and gastrointestinal, neurological and neuropsychological, and other diseases in the United States. The company offers KarXT targeting muscarinic acetylcholine receptors to treat schizophrenia and psychosis in Alzheimer's disease; a regenerative biology platform for androgenetic alopecia, epithelial ageing, and other medical conditions; an immunomodulation platform to treat chronic and acute inflammatory disorders; oral therapies based on defined consortia of bacteria is isolated from the human microbiome; and therapies to treat cognitive dysfunction associated with depression, multiple sclerosis, post COVID and ICU, and cancer related conditions. It also provides hematopoietic stem cell based therapies for patients with blood cancer; a voice-based technology platform to detect voice changes linked to health conditions; and a technology platform for the oral delivery of biologics, vaccines, and other drugs. In addition, the company is developing LYT-100 to treat lymphedema, and other lymphatic flow disorders; LYT-200, a IgG4 monoclonal antibody to target galectin-9; LYT-210 to treat solid tumors; Glyph, a synthetic lymphatic targeting chemistry platform; Orasome technology to enable the oral administration of macromolecule therapeutic payloads; meningeal lymphatics platform to treat Alzheimer's and Parkinson's diseases; and Alivio technology platform for inflammation-targeted disease immunomodulation. PureTech Health plc has collaboration and license agreements with Boehringer Ingelheim International GMBH; Eli Lilly and Company; Imbrium Therapeutics L.P.; and Shionogi & Co., Ltd. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Contineum Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on discovering and developing novel oral small molecule therapies for neuroscience, inflammation, and immunology indications with high unmet need. Its lead asset is PIPE-791, a novel, brain penetrant, small molecule inhibitor of the lysophosphatidic acid 1 receptor (LPA1R) for the treatment of idiopathic pulmonary fibrosis and progressive multiple sclerosis (MS). The company also develops PIPE-307, a novel, small molecule selective inhibitor of the muscarinic type 1 M1 receptor to treat depression and relapse remitting MS; and CTX-343, a peripherally-restricted LPA1R antagonist. Contineum Therapeutics, Inc. was formerly known as Pipeline Therapeutics, Inc. and changed its name to Contineum Therapeutics, Inc. in November 2023. The company was incorporated in 2009 and is headquartered in San Diego, California.

Acelyrin, Inc., a clinical biopharma company, focuses on identifying, acquiring, and accelerating the development and commercialization of transformative medicines. The company's lead product candidate is izokibep, a small protein therapeutic designed to inhibit IL-17A with high potency, which is in Phase 3 clinical trials for use in the treatment of Hidradenitis Suppurativa, Psoriatic Arthritis, and uveitis, as well as in Phase 2 clinical trials for use in the treatment of Axial Spondyloarthritis. It is also developing lonigutamab, a humanized immunoglobulin G1 (IgG1) monoclonal antibody against insulin-like growth factor 1 receptor, which is in Phase 1 clinical trials for use in the treatment of thyroid eye disease; and SLRN-517, a fully human IgG1 monoclonal antibody targeting c-KIT, which is in preclinical stage for use in the treatment of chronic urticaria. The company was incorporated in 2020 and is headquartered in Agoura Hills, California.

Valneva SE, a specialty vaccine company, focuses on the development and commercialization of prophylactic vaccines for infectious diseases with unmet needs. Its commercial vaccines for travelers include IXIARO, an inactivated Vero cell culture-derived Japanese encephalitis vaccine indicated for active immunization against Japanese encephalitis; DUKORAL, an oral vaccine for the prevention of diarrhea caused by Vibrio cholera and/or heat-labile toxin producing Enterotoxigenic Escherichia coli bacterium; and VLA2001, a vaccine candidate against SARS-CoV-2. The company also develops VLA15, a vaccine candidate that has completed Phase II clinical trial against Borrelia; and VLA1553, a vaccine candidate, which is in Phase III clinical trial against the chikungunya virus. It sells its products in the United States, Canada, Germany, Austria, Nordics, the United Kingdom, other European countries, and internationally. Valneva SE has collaborations with Pfizer, Inc. to co-develop and commercialize its Lyme disease vaccine; and Instituto Butantan for the development, manufacturing, and marketing of single-shot chikungunya vaccine. The company was founded in 1998 and is headquartered in Saint-Herblain, France.

Anavex Life Sciences Corp., a clinical stage biopharmaceutical company, engages in the development of drug candidates for the treatment of central nervous system (CNS) diseases. Its lead drug candidate is ANAVEX 2-73, which is in Phase III clinical trial for the treatment of Alzheimer's disease; Phase III clinical trial to treat pediatric patients with Rett syndrome; Phase II clinical trial for the treatment of Parkinson's disease; and preclinical clinical trials to treat epilepsy, infantile spasms, Fragile X syndrome, Angelman syndrome, multiple sclerosis, and tuberous sclerosis complex. The company's drug candidate also comprises ANAVEX 3-71, which is in Phase I clinical trial for the treatment of frontotemporal dementia and other dementia indications; and preclinical clinical trials for the treatment of neurodegenerative diseases, such as Alzheimer's and Parkinson's diseases. Its preclinical drug candidates include ANAVEX 1-41, a sigma-1 receptor agonist for the treatment of depression, stroke, Parkinson's, and Alzheimer's diseases; ANAVEX 1066, a mixed sigma-1/sigma-2 ligand for the potential treatment of neuropathic and visceral pain; and ANAVEX 1037 to treat prostate and pancreatic cancer. The company was incorporated in 2004 and is headquartered in New York, New York.

Prime Medicine, Inc., a biotechnology company, delivers genetic therapies to address diseases by deploying gene editing technology. It offers Prime Editors with a Prime Editor protein, comprising a fusion between a Cas protein and a reverse transcriptase enzyme; and a pegRNA, which targets the Prime Editor to a specific genomic location and provides a template for making the desired edit to the target DNA sequence. The company was incorporated in 2019 and is headquartered in Cambridge, Massachusetts.

Mind Medicine (MindMed) Inc., a clinical stage biopharmaceutical company, develops novel products to treat brain health disorders related to psychiatry, addiction, pain, and neurology. The company develops MM-120, which is in phase 2 for the treatment of generalized anxiety disorder and attention deficit hyperactivity disorder, as well as for the treatment of chronic pain; and MM-110, an a3ß4 nicotinic cholinergic receptor antagonist that has completed phase 1 for the treatment of opioid withdrawal. It also develops MM-402, a R-enantiomer of 3,4-methylenedioxymethamphetamine for the treatment of core symptoms of autism spectrum disorder. The company is headquartered in Vancouver, Canada.

Alector, Inc., a clinical stage biopharmaceutical company, develops therapies for the treatment of neurodegeneration diseases. Its products include AL001, a humanized recombinant monoclonal antibody, which is in Phase III clinical trial for the treatment of frontotemporal dementia, Alzheimer's, Parkinson's, and amyotrophic lateral sclerosis diseases; and AL101 that is in Phase I clinical trial for the treatment of neurodegenerative diseases, including Alzheimer's and Parkinson's diseases. The company also offers AL002, a product candidate that is in Phase II clinical trial for the treatment of Alzheimer's disease; and AL003, which is in Phase I clinical trial for the treatment of Alzheimer's disease. In addition, its products in development stage include AL044 that targets MS4A4A, a risk gene for Alzheimer's disease. Alector, Inc. has a collaboration agreement with Adimab, LLC for the research and development of antibodies; and a strategic collaboration agreement with GlaxoSmithKline plc for the development and commercialization of monoclonal antibodies, such as AL001 and AL101 to treat neurodegenerative diseases. The company was founded in 2013 and is headquartered in South San Francisco, California.

Ginkgo Bioworks Holdings, Inc., together with its subsidiaries, develops platform for cell programming. Its platform is used to program cells to enable biological production of products, such as novel therapeutics, food ingredients, and chemicals derived from petroleum. The company serves various end markets, including specialty chemicals, agriculture, food, consumer products, and pharmaceuticals. Ginkgo Bioworks has a partnership with Selecta Biosciences, Inc. to develop ImmTOR technology platform. Ginkgo Bioworks Holdings, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.

CorMedix Inc., a biopharmaceutical company, focuses on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory diseases in the United States and internationally. Its lead product candidate is DefenCath/Neutrolin, a novel anti-infective solution for the reduction and prevention of catheter-related infections and thrombosis in patients requiring central venous catheters in clinical settings, such as hemodialysis, total parenteral nutrition, and oncology. The company was formerly known as Picton Holding Company, Inc. and changed its name to CorMedix, Inc. in January 2007. CorMedix Inc. was incorporated in 2006 and is based in Berkeley Heights, New Jersey.

Aura Biosciences, Inc. operates as a biotechnology company that develops therapies to treat cancer. The company develops virus-like drug conjugates (VDC) technology platform for the treat tumors of high unmet need in ocular and urologic oncology. It develops AU-011, a VDC candidate for the treatment of primary choroidal melanoma. It also develops AU-011 in additional ocular oncology indications, including choroidal metastases. The company was incorporated in 2009 and is headquartered in Cambridge, Massachusetts.

Sage Therapeutics, Inc., a biopharmaceutical company, develops and commercializes medicines to treat central nervous system disorders. Its lead product candidate is ZULRESSO, an intravenous formulation of brexanolone for the treatment of postpartum depression (PPD) in adults. The company's product pipeline also includes zuranolone, a neuroactive steroid, which is in Phase III clinical trials for treating PPD, major depressive disorders, treatment resistant disorders, generalized anxiety disorders, and bipolar depression; and SAGE-324, a compound that is in Phase II clinical trial to treat essential tremors, as well as has completed Phase I clinical trial for epilepsy and Parkinson's diseases. In addition, its product pipeline comprises SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor, which has completed Phase I clinical trial for the treatment of depression, Huntington's disease, Alzheimer's disease, attention deficit hyperactivity disorder, schizophrenia, and neuropathic pain. Sage Therapeutics, Inc. has a strategic collaboration with Shionogi & Co., Ltd. for the development and commercialization of zuranolone in Japan, Taiwan, and South Korea; and a collaboration and license agreement with Biogen MA Inc. to jointly develop and commercialize SAGE-217 and SAGE-324 products. The company was formerly known as Sterogen Biopharma, Inc. and changed its name to Sage Therapeutics, Inc. in September 2011. Sage Therapeutics, Inc. was incorporated in 2010 and is headquartered in Cambridge, Massachusetts.

Instil Bio, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of patients with cancer. The company develops cell therapy of autologous tumor infiltrating lymphocyte (TIL). Its TIL product candidates in pipeline include ITIL-168 for indications, such as melanoma, cutaneous squamous cell carcinoma, non-small cell lung cancer, head and neck squamous cell carcinoma, and cervical cancer; and ITIL-306 to recognize folate receptor alpha (FOLR1) with indications, including gynecological, non-small cell lung cancer, renal caner, and others. The company was incorporated in 2018 and is headquartered in Dallas, Texas.

Altimmune, Inc., a clinical stage biopharmaceutical company, focuses on developing treatments for obesity and liver diseases. The company's lead product candidate, pemvidutide (proposed INN, formerly known as ALT-801), is a GLP-1/glucagon dual receptor agonist that is in Phase 1b trial for the treatment of obesity and non-alcoholic steatohepatitis. It is also developing HepTcell, an immunotherapeutic product candidate, which is in Phase 2 clinical trial for patients chronically infected with the hepatitis B virus. The company was formerly known as Vaxin Inc. and changed its name to Altimmune, Inc. in September 2015. Altimmune, Inc. was founded in 1997 is headquartered in Gaithersburg, Maryland.

Absci Corporation, a drug and target discovery company, provides biologic drug candidates and production cell lines using integrated drug creation platform for partners in the United States. Its integrated drug creation platform enables the creation of biologics by unifying the drug discovery and cell line development processes into one process. Absci Corporation was founded in 2011 and is headquartered in Vancouver, Washington.

EyePoint Pharmaceuticals, Inc., a pharmaceutical company, develops and commercializes ophthalmic products for the treatment of eye diseases in the United States, China, and the United Kingdom. The company provides ILUVIEN, an injectable sustained-release micro-insert for treatment of diabetic macular edema; YUTIQ, a fluocinolone acetonide intravitreal implant for intravitreal injection for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye; and DEXYCU, a dexamethasone intraocular suspension, for the treatment of post-operative ocular inflammation, including treatment following cataract surgery. It is also developing EYP-1901, a twice-yearly bioerodible formulation of tyrosine kinase inhibitor for the treatment of wet age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion; and YUTIQ50 for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye. The company has strategic collaborations with Alimera Sciences, Inc., Bausch & Lomb, OncoSil Medical UK Limited, Ocumension Therapeutics, and Equinox Science, LLC. It also has a commercial alliance with ImprimisRx PA, Inc. for the joint promotion of DEXYCU for the treatment of post-operative inflammation following ocular surgery. The company was formerly known as pSivida Corp. and changed its name to EyePoint Pharmaceuticals, Inc. in March 2018. EyePoint Pharmaceuticals, Inc. was incorporated in 1987 and is headquartered in Watertown, Massachusetts.

Xeris Biopharma Holdings, Inc., a biopharmaceutical company, engages in developing and commercializing therapies for patient populations in endocrinology, neurology, and gastroenterology. The company markets Gvoke, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia; and Keveyis, a therapy for the treatment of hyperkalemic, hypokalemic, and related variants of primary periodic paralysis; and Recorlev, a cortisol synthesis inhibitor proved for the treatment of endogenous hypercortisolemia in adult patients with Cushing's syndrome. It also has a pipeline of development programs to extend the marketed products into new indications and uses and bring new products using its proprietary formulation technology platforms, XeriSol and XeriJect. The company was incorporated in 2005 and is headquartered in Chicago, Illinois.

Larimar Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing treatments for rare diseases using its novel cell penetrating peptide technology platform. Its lead product candidate is CTI-1601, which is in Phase 1 clinical trial for the treatment of Friedreich's ataxia, a rare, progressive, and fatal genetic disease. The company is based in Bala Cynwyd, Pennsylvania.

Taysha Gene Therapies, Inc., a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN1 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 foe the treatment of for SLC13A5 Deficiency; and TSHA-101 for the treatment of GM2 gangliosidosis. Taysha Gene Therapies, Inc. has a strategic partnership with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments. The company was incorporated in 2019 and is based in Dallas, Texas.

Verve Therapeutics, Inc., a genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases. Its lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver. The company also engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.

Fate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops programmed cellular immunotherapies for cancer and immune disorders worldwide. Its NK- and T-cell immuno-oncology programs under development include FT516 for the treatment of acute myeloid leukemia (AML) B-cell lymphoma, and advanced solid tumor; FT596 to treat B-cell lymphoma and chronic lymphocytic leukemia; FT538 to treat AML and multiple myeloma; FT576 to treat multiple myeloma; FT819 to treat hematologic malignancies and solid tumors; FT536 to treat solid tumors; and FT500 for the treatment of advanced solid tumors. The company has a collaboration and option agreement with Ono Pharmaceutical Co. Ltd. for the development and commercialization of two off-the-shelf iPSC-derived CAR T-cell product candidates; strategic research collaboration and license agreement with Juno Therapeutics, Inc. to screen for and identify small molecule modulators that enhance the therapeutic properties of genetically-engineered T-cell immunotherapies; and a collaboration and option agreement with Janssen Biotech, Inc. Fate Therapeutics, Inc. was incorporated in 2007 and is headquartered in San Diego, California.

C4 Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutic candidates to degrade disease-causing proteins for the treatment of cancer, neurodegenerative conditions, and other diseases. Its lead product candidate is CFT7455, an orally bioavailable MonoDAC degrader of protein that is in Phase 1/2 trial targeting IKZF1 and IKZF3 for multiple myeloma and non-Hodgkin lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma. The company is also developing CFT8634, an orally bioavailable BiDAC degrader of BRD9, a protein target for synovial sarcoma and SMARCB1-deleted solid tumors; CFT1946, an orally bioavailable BiDAC degrader targeting V600X mutant BRAF to treat melanoma, non-small cell lung cancer (NSCLC), colorectal cancer, and other solid malignancies; CFT8919, an orally bioavailable, allosteric, and mutant-selective BiDAC degrader of epidermal growth factor receptor, or EGFR, with an L858R mutation in NSCLC; and earlier stage programs comprising RET degraders for the treatment of various cancers. C4 Therapeutics, Inc. has strategic collaborations with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; Biogen MA, Inc.; and Calico Life Sciences LLC. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.

Theravance Biopharma, Inc., a biopharmaceutical company, discovers, develops, and commercializes respiratory medicines in the United States, Europe, and Asia. The company offers YUPELRI, a once-daily, nebulized long-acting muscarinic antagonist used for the treatment of chronic obstructive pulmonary disease (COPD). Its product portfolio also include Izencitinib, a gut-selective pan-janus kinase (JAK) inhibitor that is in Phase IIb/III clinical trials for the treatment of rheumatoid arthritis, myelofibrosis, and ulcerative colitis, as well as for a range of inflammatory intestinal diseases, including ulcerative colitis and Crohn's disease. In addition, the company's product portfolio comprise Ampreloxetine, an investigational norepinephrine reuptake inhibitor that has completed Phase III study for neurogenic orthostatic hypotension; Nezulcitinib, a lung-selective, nebulized JAK inhibitor, which is in Phase II clinical development for the potential treatment of hospitalized patients with acute lung injury caused by COVID-19; Inhaled ALK5i, a potential inhaled anti-fibrotic agent that is in Phase I for the treatment of idiopathic pulmonary fibrosis; and TD-5202, an investigational, orally administered, gut-selective, irreversible JAK3 inhibitor that is in Phase I clinical study for treatment of inflammatory intestinal diseases. Further, it offers TRELEGY for the treatment of COPD and asthma; Velusetrag, an oral and investigational medicine for gastrointestinal motility disorders; and Selective 5-HT4 Agonist for treatment of gastrointestinal motility disorders. It has a licensing and collaboration agreements with Pfizer Inc., Viatris Inc., Janssen Biotech, Inc., Alfasigma S.p.A, and Takeda Pharmaceutical Company Limited. Theravance Biopharma, Inc. was incorporated in 2013 and is based in George Town, the Cayman Islands.

G1 Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the discovery, development, and commercialization of small molecule therapeutics for the treatment of patients with cancer. The company offers COSELA, which helps to decrease chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive stage small cell lung cancer. It is also developing trilaciclib, a (CDK) 4/6 inhibitor that is in Phase III clinical trials for patients with first line colorectal cancer; Phase III clinical trial for the treatment of first line triple negative breast cancer; Phase II clinical trial for patients with first line bladder cancer; Phase II clinical trial for patients with combination with the antibody-drug conjugate; and Phase II clinical trial for the treatment of neoadjuvant breast cancer. In addition, the company develops lerociclib, an oral CDK4/6 inhibitor for multiple oncology indications; and rintodestrant, an oral selective estrogen receptor degrader, which is in Phase IIa clinical trials for the treatment of estrogen receptor-positive and HER2-negative breast cancer. The company has a license agreement with EQRx, Inc. and Genor Biopharma Co. Inc. for the development and commercialization of lerociclib using an oral dosage form to treat any indication in humans, as well as Nanjing Simcere Dongyuan Pharmaceutical Co., LTD. for the development and commercialization of trilaciclib for any indication in humans through parenteral delivery, and ARC Therapeutics for the development and commercialization of a CDK2 inhibitor for all human and veterinary uses. G1 Therapeutics, Inc. was incorporated in 2008 and is headquartered in Research Triangle Park, North Carolina.

AC Immune SA, a clinical stage biopharmaceutical company, discovers, designs, and develops medicines and diagnostic products for the prevention and treatment of neurodegenerative diseases associated with protein misfolding. Its SupraAntigen and Morphomer platforms are designed to generate vaccines, antibodies, and small molecules, which selectively interact with misfolded proteins that are common in a range of neurodegenerative diseases. The company is developing Crenezumab, a humanized, conformation-specific monoclonal antibody, which is in Phase II clinical prevention trial for the treatment of Alzheimer's disease (AD). It is also developing ACI-24, an anti-Abeta vaccine candidate that is in Phase II clinical study for AD, as well as completed Phase Ib clinical study for Down syndrome; ACI-35, an anti-Tau vaccine candidate that has completed Phase Ib clinical study; and Tau- positron emission tomography (PET) imaging tracer, which is in Phase II clinical study. In addition, the company is researching and developing small molecule Tau aggregation inhibitors for AD and NeuroOrphan indications. Further, it has discovery and preclinical stage molecules targeting range of neurodegenerative diseases, which include diagnostics targeting TDP-43, alpha-synuclein, and NLRP3. AC Immune SA has license agreements and collaborations with Genentech, Inc.; Biogen International GmbH; Janssen Pharmaceuticals, Inc.; Life Molecular Imaging SA; Eli Lilly and Company; and WuXi Biologics. The company was incorporated in 2003 and is headquartered in Lausanne, Switzerland.

Iteos Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the discovery and development of immuno-oncology therapeutics for patients. The company's product pipeline includes inupadenant, a small molecule antagonist of the adenosine A2AR antagonists that is in Phase 2 clinical trials; and EOS-448, an antagonist of TIGIT or T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1/2 clinical trial, as well as used to engage the Fc gamma receptor, or Fc?R to activate dendritic cells and macrophages and to promote antibody-dependent cellular cytotoxicity, or ADCC activity. Iteos Therapeutics, Inc. was founded in 2011 and is headquartered in Watertown, Massachusetts.

Biomea Fusion, Inc., a biopharmaceutical company, focuses on the discovery and development of covalent small molecule drugs to treat patients with genetically defined cancers and metabolic diseases. Its lead product candidate is BMF-219, an orally bioavailable, potent, and selective covalent inhibitor of menin, a transcriptional regulator in oncogenic signaling in multiple cancers. The company was incorporated in 2017 and is headquartered in Redwood City, California.

Zevra Therapeutics, Inc., a rare disease company melding science, discovers and develops various proprietary prodrugs to treat serious medical conditions in the United States. The company utilizes its Ligand Activated Therapy technology to generate improved prodrug versions of FDA-approved drugs, as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. Its prodrug product candidate pipeline is focused on the high need areas of attention deficit hyperactivity disorder, stimulant use disorder, and CNS rare diseases, including idiopathic hypersomnia (IH). The company's lead product candidate KP1077, which is under Phase II clinical trial for the treatment of IH and narcolepsy, is based on its prodrug of d-methylphenidate, known as serdexmethylphenidate. It is also developing KP879, a prodrug product candidate for the treatment of stimulant use disorder and is under Phase II clinical trial. In addition, the company has received FDA approval for AZSTARYS, a once-daily treatment for attention deficit hyperactivity disorder in patents age six years and older, and for APADAZ, an immediate-release combination product containing benzhydrocodone, a prodrug of hydrocodone, and acetaminophen. The company's product pipeline include, arimoclomol It has collaboration and license agreement with KVK-Tech, Inc. and Commave Therapeutics SA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.

Nautilus Biotechnology, Inc., a development stage life sciences company, engages in creating a platform technology for quantifying and unlocking the complexity of the proteome. It develops Nautilus Platform, a proteomics platform that includes end-to-end solution comprised of instruments, consumables, and software analysis. The company was founded in 2016 and is headquartered in Seattle, Washington.

Lyell Immunopharma, Inc., a T cell reprogramming company, engages in developing T cell therapies for patients with solid tumors. The company develops therapies using technology platforms, such as Gen-R, an ex vivo genetic reprogramming technology to overcome T cell exhaustion; and Epi-R, an ex vivo epigenetic reprogramming technology to generate population of T cells with durable stemness. Its pipeline includes LYL797, a T cell product candidate for the treatment of non-small cell lung cancer and triple negative breast cancers; LYL845, that targets multiple solid tumors; and NY-ESO-1 for synovial sarcoma and other solid tumor indications. The company entered into research and development collaboration and license agreement with GlaxoSmithKline for NY-ESO-1 program. Lyell Immunopharma, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

GH Research PLC, a clinical-stage biopharmaceutical company, engages in developing various therapies for the treatment of psychiatric and neurological disorders. The company focuses on developing 5-Methoxy-N,N-Dimethyltryptamine (5-MeO-DMT) therapies for the treatment of patients with treatment-resistant depression (TRD). Its lead program is GH001, an inhalable 5-MeO-DMT product candidate that has completed two Phase 1 clinical trials and Phase 1/2 clinical trial in patients with TRD. The company also develops GH002, an injectable 5-MeO-DMT product candidate; and GH003, an intranasal 5-MeO-DMT product candidate, which are in preclinical development trials with a focus on psychiatric and neurological disorders. GH Research PLC was incorporated in 2018 and is based in Dublin, Ireland.

Selecta Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the research and development of nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's lead therapeutic gene therapy program is SEL-302 that is in Phase I clinical trial to enhance the treatment of methylmalonic acidemia. It is also developing biologic therapies, such as SEL-212 that is in Phase III clinical trials for the treatment of chronic refractory gout; and product candidates to treat IgA-mediated diseases, including IgA nephropathy, linear IgA bullous dermatitis, IgA pemphigus, and Henoch-Schonlein purpura. In addition, the company is developing gene therapies comprising for the treatment of pompe disease, duchenne muscular dystrophy, limb-girdle muscular dystrophy, lysosomal storage disorder, and other autoimmune diseases. Further, it develops tolerogenic therapies to treat autoimmune diseases. The company has license and collaboration agreements with Ginkgo Bioworks Holdings, Inc.; Genovis AB (publ.); Cyrus Biotechnology, Inc.; IGAN Biosciences, Inc.; Astellas Therapeutics, Inc.; Takeda Pharmaceuticals USA, Inc.; Swedish Orphan Biovitrum AB (publ.); Sarepta Therapeutics, Inc.; Asklepios Biopharmaceutical, Inc.; Massachusetts Institute of Technology; and Shenyang Sunshine Pharmaceutical Co., Ltd. Selecta Biosciences, Inc. was incorporated in 2007 and is headquartered in Watertown, Massachusetts.

Eliem Therapeutics, Inc., a clinical-stage biotechnology company, focuses on developing therapies for neuronal excitability disorders to address unmet needs in chronic pain, neuropsychiatry, epilepsy, and other disorders of the peripheral and central nervous systems. Its two lead drug candidates are ETX-810 for chronic pain, which is in Phase IIa clinical trial; and ETX-155 for the treatment of major depressive disorder, perimenopausal depression, and focal onset seizures that is in Phase I clinical trial. The company also develops preclinical pipeline programs, such as Kv7 Program for pain, epilepsy, and depression; and Anxiolytic for the treatment of generalized anxiety disorder. Eliem Therapeutics, Inc. was incorporated in 2018 and is headquartered in Redmond, Washington.

Immutep Limited, a biotechnology company, engages in the research and development of pharmaceutical product candidates. The company develops immunotherapeutic products for the treatment of cancer and autoimmune diseases. Its lead product candidate is eftilagimod alpha (efti or IMP321), a recombinant protein that is in Phase IIb clinical trial as a chemoimmunotherapy combination for metastatic breast cancer. The company also develops TACTI-002, which is in a Phase II clinical trial for the treatment of head and neck squamous cell carcinoma (HNSCC) and non-small cell lung cancer; TACTI-003 that is in Phase IIb clinical trial to treat HNSCC; and INSIGHT-004 and INSIGHT-003, which is in a Phase I clinical trial for the treatment of solid tumors, as well as INSIGHT-005 that is in Phase I/IIa clinical trial to treat solid tumors. Its other products include IMP761, an agonist of lymphocyte activation gene 3 for autoimmune disease; IMP701, an antagonist antibody that acts to stimulate T cell proliferation in cancer patients; and IMP731, a depleting antibody that removes T cells involved in autoimmunity. Immutep Limited has collaboration agreements with GlaxoSmithKline, Novartis, CYTLIMIC Inc., Merck & Co., Inc., Institute of Clinical Cancer Research, Merck KGaA, and EOC Pharma. The company was formerly known as Prima BioMed Ltd and changed its name to Immutep Limited in November 2017. Immutep Limited was incorporated in 1987 and is based in Sydney, Australia.

Corvus Pharmaceuticals, Inc., a clinical stage biopharmaceutical company, focuses on the development and commercialization of immuno-oncology therapies. Its lead product candidate is Mupadolimab (CPI-006), an anti-CD73 monoclonal antibody, which is in Phase Ib/II clinical trial for non-small cell lung cancer and head and neck cancers. The company also develops CPI-818, a covalent inhibitor of ITK, which is in Phase I/Ib clinical trial to treat patients with various malignant T-cell lymphomas, as well as designed to inhibit the proliferation of certain malignant T-cells; and Ciforadenant (CPI-444), an oral, small molecule antagonist of the A2A receptor that is in Phase II clinical trial for patients with either advanced or refractory renal cell cancer. Its preclinical stage products include CPI-182, an antibody designed to block inflammation and myeloid suppression; and CPI-935, an adenosine A2B receptor antagonist to prevent fibrosis. Corvus Pharmaceuticals, Inc. has a strategic collaboration with Angel Pharmaceuticals for the development its pipeline of targeted investigational medicines. The company was incorporated in 2014 and is based in Burlingame, California.

Inozyme Pharma, Inc., a rare disease biopharmaceutical company, engages in developing therapeutics for treating abnormal mineralization impacting the vasculature, soft tissue, and skeleton diseases. The company's lead product candidate is INZ-701, a soluble, recombinant protein to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies, as well as for the treatment of calciphylaxis. It has a license agreement with Yale University for specified therapeutic and prophylactic products. Inozyme Pharma, Inc. was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Monte Rosa Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel small molecule precision medicines that employ the body's natural mechanisms to selectively degrade therapeutically relevant proteins. It develops an oral molecular glue degrader for GSPT1, a translational termination factor and degron-containing protein for the treatment of Myc-driven cancers. The company also develops CDK2 to treat ovarian, uterine, and breast cancers; NEK7 for the treatment of inflammatory diseases, such as Crohn's disease, neurodegenerative disease, diabetes, and liver disease; VAV1, a target protein for autoimmune diseases; and BCL11A, a therapeutically-relevant protein in hemoglobinopathies. Monte Rosa Therapeutics, Inc. was incorporated in 2019 and is headquartered in Boston, Massachusetts.

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited blindness, as well as Xerostomia following radiation treatment for head and neck cancers; degenerative diseases; neurodegenerative diseases, such as amyotrophic lateral sclerosis; and Parkinson's diseases. Its programs in clinical development include Phase 1/2 clinical stage programs in Achromatopsia, X-Linked Retinitis Pigmentosa, RPE65-deficiency, and radiation-induced Xerostomia, as well as Parkinson's program. The company also focuses on initiating a clinical program in xerostomia related to Sjogren's syndrome and have preclinical programs in neurodegenerative diseases. It has a research collaboration agreement with Janssen Pharmaceuticals, Inc. to develop regulatable gene therapy treatment using the company's proprietary riboswitch technology. MeiraGTx Holdings plc was incorporated in 2015 and is based in New York, New York.

Aldeyra Therapeutics, Inc., a biotechnology company, develops and commercializes medicines for immune-mediated ocular and systemic diseases. The company's lead product candidate is reproxalap, a reactive aldehyde species (RASP)modulator, which is in Phase III clinical trial for the treatment of dry eye diseases and allergic conjunctivitis. It also develops ADX-629, a first-in-class orally administered RASP modulator that is Phase II clinical trial for psoriasis, asthma, and COVID-19; and ADX-2191, a dihydrofolate reductase inhibitor which is in phase 3 for the prevention of proliferative vitreoretinopathy, and phase II clinical trial for the treatment of retinitis pigmentosa, as well as for treating primary vitreoretinal lymphoma. The company has a license agreement with Madrigal Pharmaceuticals, Inc. for developing ADX-1612, which inhibits the protein chaperome for the treatment of inflammatory diseases. The company was formerly known as Aldexa Therapeutics, Inc. and changed its name to Aldeyra Therapeutics, Inc. in March 2014. Aldeyra Therapeutics, Inc. was incorporated in 2004 and is based in Lexington, Massachusetts.

Voyager Therapeutics, Inc., a gene therapy company, focuses on the development of treatments and next-generation platform technologies. The company's lead clinical candidate is the VY-AADC, which is in open-label Phase 1 clinical trial for the treatment of Parkinson's disease. Its preclinical programs comprise VY-SOD102 for the treatment of amyotrophic lateral sclerosis; VY-HTT01 for Huntington's disease; VY-FXN01 for Friedreich's ataxia; and Tau program for the treatment of tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, as well as for spinal muscular atrophy. The company has collaboration and license agreements with Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. for the research, development, and commercialization of adeno-associated virus gene therapy products. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Cambridge, Massachusetts.

Nkarta, Inc., a a clinical-stage biopharmaceutical company, develops and commercializes cell therapies for cancer treatment. The company's approach for cellular immunotherapy involves chimeric antigen receptors on the surface of a natural killer (NK) cell that enable the cell to recognize specific proteins or antigens that are present on the surface of tumor cells. Its two co-lead product candidates are NKX101, which is in Phase I clinical trials for the treatment of relapsed/refractory acute myeloid leukemia or higher risk myelodysplastic syndromes; and NKX019, a pre-clinical product, which is based on the ability to treat various B cell malignancies by targeting the CD19 antigen found on these types of cancerous cells. The company has a research collaboration agreement with CRISPR Therapeutics AG. Nkarta, Inc. was incorporated in 2015 and is based in South San Francisco, California.

Korro Bio, Inc., a biopharmaceutical company, discovers, develops, and commercializes genetic medicines based on editing RNA for the treatment of rare and highly prevalent diseases. Korro Bio, Inc. is based in Cambridge, Massachusetts.

XOMA Corporation operates as a biotechnology royalty aggregator in Europe, the United States, and the Asia Pacific. The company engages in helping biotech companies for enhancing human health. It acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. The company focuses on early to mid-stage clinical assets primarily in Phase 1 and 2 with commercial sales potential that are licensed to partners. It has a portfolio with approximately 70 assets. XOMA Corporation was incorporated in 1981 and is headquartered in Emeryville, California.