| SRPT | Sarepta Therapeutics |
| KRYS | Krystal Biotech |
| DYN | Dyne Therapeutics |
| CAPR | Capricor Therapeutics |
| SLDB | Solid Biosciences |
| IONS | Ionis Pharmaceuticals |
| RARE | Ultragenyx Pharmaceutical |
| BBIO | BridgeBio Pharma |
| BMRN | BioMarin Pharmaceutical |
| WVE | Wave Life Sciences |