| ALNY | Alnylam Pharmaceuticals |
| ASND | Ascendis Pharma |
| IONS | Ionis Pharmaceuticals |
| TVTX | Travere Therapeutics |
| RARE | Ultragenyx Pharmaceutical |
| PTCT | PTC Therapeutics |
| MAZE | Maze Therapeutics |
| DNLI | Denali Therapeutics |
| SRRK | Scholar Rock |
| BMRN | BioMarin Pharmaceutical |