Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $239.4b | $376.63 | 0.0% | 596 |
 VRTX | Vertex Pharmaceuticals Inc | $141.4b | $515.28 | 0.0% | 19 |
 ALNY | Alnylam Pharmaceuticals, Inc. | $35.8b | $239.14 | 0.0% | 5 |
 INSM | Insmed, Inc. | $27.7b | $120.98 | 0.0% | 263 |
 INCY | Incyte Genomics Inc | $26.1b | $124.11 | 0.0% | 41 |
 UTHR | United Therapeutics Corp | $21.0b | $500.00 | 0.0% | N/A |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $15.0b | $68.30 | 0.0% | 393 |
 BMRN | BioMarin Pharmaceuticals Inc | $13.0b | $64.57 | 0.0% | 117 |
 ARWR | Arrowhead Research Corporation | $12.1b | $71.08 | 0.0% | 136 |
 CORT | Corcept Therapeutics Inc. | $11.9b | $110.39 | 0.0% | 58 |
 KRYS | Krystal Biotech, Inc. Common Stock | $10.9b | $336.60 | 0.0% | 357 |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $10.2b | $47.34 | 0.0% | 1.4K |
 CYTK | Cytokinetics Inc. | $10.0b | $69.50 | 0.0% | 4.7K |
 CRNX | Crinetics Pharmaceuticals, Inc. | $8.8b | $84.97 | 0.0% | N/A |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $7.2b | $97.75 | 0.0% | 3.9K |
 SRRK | Scholar Rock Holding Corporation Common Stock | $6.8b | $50.00 | 0.0% | 53 |
 TVTX | Travere Therapeutics, Inc. Common Stock | $6.3b | $62.50 | 0.0% | 9.0K |
 LQDA | Liquidia Corporation Common Stock | $6.2b | $64.23 | 0.0% | 102 |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $6.1b | $94.38 | 0.0% | 2.4K |
 COGT | Cogent Biosciences, Inc. Common Stock | $6.0b | $33.08 | 0.0% | 60 |
 PTCT | PTC Therapeutics, Inc. | $6.0b | $65.51 | 0.0% | 167 |
 CRSP | CRISPR Therapeutics AG | $5.5b | $52.42 | 0.0% | 3.8K |
 APLS | Apellis Pharmaceuticals, Inc. Common Stock | $5.2b | $41.04 | +0.0% | 68.6M |
 DYN | Dyne Therapeutics, Inc. Common Stock | $4.7b | $17.81 | 0.0% | 540 |
 EWTX | Edgewise Therapeutics, Inc. Common Stock | $4.6b | $43.77 | 0.0% | 332 |
 FOLD | Amicus Therapeutics, Inc | $4.5b | $14.49 | +0.0% | 3.5M |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $3.8b | $33.37 | 0.0% | 1 |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $3.7b | $78.56 | 0.0% | 305 |
 QURE | uniQure N.V. | $3.1b | $43.24 | 0.0% | 55.7K |
 IRON | Disc Medicine, Inc. Common Stock | $3.1b | $67.50 | 0.0% | 10 |
 BEAM | Beam Therapeutics Inc. Common Stock | $3.0b | $23.38 | 0.0% | 2.8K |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.6b | $21.85 | 0.0% | 142 |
 BCRX | BioCryst Pharmaceuticals Inc | $2.6b | $8.31 | 0.0% | 305 |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.5b | $40.81 | 0.0% | 6 |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.5b | $41.90 | 0.0% | 1 |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $2.4b | $21.36 | 0.0% | 78 |
 OCUL | Ocular Therapeutix, Inc. | $2.3b | $10.19 | 0.0% | 232 |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $2.2b | $151.98 | 0.0% | 2.0K |
 AUPH | Aurinia Pharmaceuticals Inc | $2.2b | $16.19 | 0.0% | 135 |
 AGIO | Agios Pharmaceuticals, Inc. | $2.0b | $32.88 | 0.0% | 806 |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.9b | $28.16 | 0.0% | N/A |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.8b | $5.04 | 0.0% | N/A |
 NTLA | Intellia Therapeutics, Inc | $1.8b | $11.43 | 0.0% | 4.2K |
 ETON | Eton Pharmaceutcials, Inc. Common Stock | $1.8b | $55.22 | 0.0% | 219 |
 DMRA | Damora Therapeutics, Inc. Ordinary Shares | $1.7b | $25.25 | 0.0% | 249 |
 ANIP | ANI Pharmaceuticals, Inc. | $1.7b | $70.28 | 0.0% | 344 |
 FBRX | Forte Biosciences, Inc. Common Stock | $1.6b | $76.98 | 0.0% | N/A |
 XERS | Xeris Biopharma Holdings, Inc. Common Stock | $1.6b | $7.52 | 0.0% | N/A |