Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $207.9b | $438.15 | -0.4% | 1.6M |
 VRTX | Vertex Pharmaceuticals Inc | $119.5b | $545.52 | -1.0% | 556.1K |
 ALNY | Alnylam Pharmaceuticals, Inc. | $27.5b | $238.57 | -0.4% | 861.4K |
 INCY | Incyte Genomics Inc | $24.2b | $129.78 | -1.2% | 540.3K |
 UTHR | United Therapeutics Corp | $21.9b | $516.89 | -0.4% | 214.6K |
 INSM | Insmed, Inc. | $21.4b | $125.08 | +0.4% | 1.1M |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $15.7b | $80.78 | -1.3% | 1.2M |
 CORT | Corcept Therapeutics Inc. | $12.4b | $120.68 | -3.0% | 653.6K |
 ARWR | Arrowhead Research Corporation | $11.9b | $88.93 | +0.2% | 716.5K |
 BMRN | BioMarin Pharmaceuticals Inc | $11.6b | $65.74 | -0.8% | 1.4M |
 CYTK | Cytokinetics Inc. | $10.5b | $79.53 | +2.3% | 1.1M |
 KRYS | Krystal Biotech, Inc. Common Stock | $10.1b | $358.50 | +1.1% | 246.3K |
 CRNX | Crinetics Pharmaceuticals, Inc. | $8.8b | $84.84 | -0.0% | 420.2K |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $8.6b | $63.79 | -0.3% | 1.6M |
 LQDA | Liquidia Corporation Common Stock | $7.5b | $71.17 | +1.2% | 636.7K |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $7.1b | $112.95 | -1.3% | 287.5K |
 COGT | Cogent Biosciences, Inc. Common Stock | $6.7b | $37.10 | -0.2% | 894.5K |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $6.4b | $98.43 | +0.3% | 458.6K |
 PTCT | PTC Therapeutics, Inc. | $5.7b | $72.70 | -1.0% | 395.6K |
 SRRK | Scholar Rock Holding Corporation Common Stock | $5.5b | $60.99 | +3.2% | 2.1M |
 APLS | Apellis Pharmaceuticals, Inc. Common Stock | $5.2b | $41.04 | +0.0% | 68.6M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $5.1b | $67.12 | +0.2% | 920.4K |
 CRSP | CRISPR Therapeutics AG | $4.7b | $59.41 | -2.7% | 837.6K |
 DYN | Dyne Therapeutics, Inc. Common Stock | $4.7b | $26.36 | +0.1% | 1.2M |
 FOLD | Amicus Therapeutics, Inc | $4.5b | $14.49 | +0.0% | 3.5M |
 EWTX | Edgewise Therapeutics, Inc. Common Stock | $4.1b | $44.38 | +0.5% | 650.1K |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 QURE | uniQure N.V. | $3.1b | $50.46 | -0.2% | 823.2K |
 IRON | Disc Medicine, Inc. Common Stock | $3.0b | $82.58 | +0.3% | 375.0K |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $2.7b | $70.65 | -1.2% | 376.1K |
 BEAM | Beam Therapeutics Inc. Common Stock | $2.6b | $30.41 | -2.6% | 1.0M |
 RARE | Ultragenyx Pharmaceutical Inc. | $2.5b | $26.27 | -0.0% | 1.3M |
 BCRX | BioCryst Pharmaceuticals Inc | $2.3b | $9.79 | -0.1% | 1.4M |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $2.3b | $37.47 | -1.1% | 1.7M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.3b | $37.02 | -0.5% | 382.0K |
 SION | Sionna Therapeutics, Inc. Common Stock | $2.2b | $6.17 | -1.2% | 804.0K |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.1b | $24.26 | +0.3% | 630.4K |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.0b | $39.40 | +1.8% | 640.9K |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $2.0b | $154.37 | +4.2% | 162.6K |
 AUPH | Aurinia Pharmaceuticals Inc | $1.9b | $16.61 | -1.3% | 652.0K |
 OCUL | Ocular Therapeutix, Inc. | $1.8b | $10.98 | -1.0% | 1.1M |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.8b | $33.03 | -1.1% | 557.0K |
 ANIP | ANI Pharmaceuticals, Inc. | $1.8b | $76.14 | -1.5% | 357.1K |
 AGIO | Agios Pharmaceuticals, Inc. | $1.8b | $33.98 | +0.3% | 467.9K |
 DMRA | Damora Therapeutics, Inc. Ordinary Shares | $1.8b | $30.37 | -0.7% | 151.0K |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.7b | $19.36 | +2.9% | 2.1M |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.7b | $6.12 | -3.1% | 1.1M |
 FBRX | Forte Biosciences, Inc. Common Stock | $1.6b | $76.98 | +0.0% | 1.4M |
 NTLA | Intellia Therapeutics, Inc | $1.5b | $13.40 | -2.4% | 2.4M |