Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $181.8b | $357.17 | +1.4% | 1.7M |
 VRTX | Vertex Pharmaceuticals Inc | $113.6b | $489.29 | +2.3% | 3.0M |
 ALNY | Alnylam Pharmaceuticals, Inc. | $40.3b | $291.25 | -0.5% | 760.7K |
 UTHR | United Therapeutics Corp | $23.6b | $542.50 | -0.2% | 431.9K |
 INSM | Insmed, Inc. | $23.2b | $104.75 | -0.9% | 3.4M |
 INCY | Incyte Genomics Inc | $19.3b | $112.75 | +5.8% | 2.2M |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $13.0b | $70.87 | +0.9% | 5.3M |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $12.6b | $81.19 | +3.7% | 2.7M |
 BMRN | BioMarin Pharmaceuticals Inc | $11.1b | $59.30 | +2.9% | 1.9M |
 ARWR | Arrowhead Research Corporation | $11.0b | $80.50 | -0.4% | 2.8M |
 CYTK | Cytokinetics Inc. | $10.4b | $82.14 | +1.6% | 3.1M |
 KRYS | Krystal Biotech, Inc. Common Stock | $9.1b | $366.76 | +3.3% | 464.7K |
 CORT | Corcept Therapeutics Inc. | $7.5b | $86.66 | +3.1% | 616.3K |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $6.2b | $123.80 | +5.4% | 1.4M |
 PTCT | PTC Therapeutics, Inc. | $6.1b | $84.40 | -0.2% | 1.5M |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $6.1b | $109.84 | +1.4% | 884.0K |
 COGT | Cogent Biosciences, Inc. Common Stock | $6.0b | $38.20 | +0.0% | 3.1M |
 SRRK | Scholar Rock Holding Corporation Common Stock | $5.9b | $54.19 | +1.7% | 1.8M |
 LQDA | Liquidia Corporation Common Stock | $5.5b | $78.18 | +1.5% | 1.6M |
 CRSP | CRISPR Therapeutics AG | $5.4b | $54.52 | +1.7% | 1.7M |
 APLS | Apellis Pharmaceuticals, Inc. Common Stock | $5.2b | $41.04 | +0.0% | 68.6M |
 FOLD | Amicus Therapeutics, Inc | $4.5b | $14.49 | +0.0% | 3.5M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $4.4b | $60.87 | -0.4% | 1.8M |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.36 | +0.2% | 1.3M |
 CRNX | Crinetics Pharmaceuticals, Inc. | $3.7b | $37.37 | +4.9% | 2.0M |
 EWTX | Edgewise Therapeutics, Inc. Common Stock | $3.7b | $41.51 | -0.8% | 916.0K |
 BEAM | Beam Therapeutics Inc. Common Stock | $3.4b | $35.19 | +2.6% | 2.6M |
 DYN | Dyne Therapeutics, Inc. Common Stock | $3.2b | $20.93 | +1.8% | 2.7M |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 IRON | Disc Medicine, Inc. Common Stock | $2.7b | $77.50 | +6.8% | 1.2M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $2.6b | $62.85 | -2.5% | 1.0M |
 RARE | Ultragenyx Pharmaceutical Inc. | $2.4b | $30.33 | +5.2% | 2.9M |
 BCRX | BioCryst Pharmaceuticals Inc | $2.3b | $9.99 | +0.6% | 5.6M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.1b | $34.01 | +2.1% | 470.5K |
 OCUL | Ocular Therapeutix, Inc. | $2.0b | $9.75 | +0.7% | 1.9M |
 AUPH | Aurinia Pharmaceuticals Inc | $2.0b | $17.20 | -1.8% | 2.0M |
 NTLA | Intellia Therapeutics, Inc | $2.0b | $16.63 | +3.9% | 4.4M |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $1.9b | $19.85 | +2.6% | 1.3M |
 SION | Sionna Therapeutics, Inc. Common Stock | $1.9b | $42.09 | -1.5% | 411.5K |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.9b | $31.98 | +5.1% | 1.2M |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.9b | $16.90 | +5.2% | 3.8M |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $1.8b | $35.34 | +4.0% | 786.0K |
 QURE | uniQure N.V. | $1.8b | $47.66 | -3.1% | 2.8M |
 ANIP | ANI Pharmaceuticals, Inc. | $1.8b | $84.46 | +1.4% | 250.9K |
 AGIO | Agios Pharmaceuticals, Inc. | $1.7b | $37.61 | +4.6% | 1.3M |
 CAPR | Capricor Therapeutics Inc | $1.7b | $26.71 | -13.2% | 3.5M |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $1.7b | $149.33 | +8.9% | 482.9K |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.7b | $6.28 | +2.3% | 3.7M |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $1.6b | $17.79 | +2.0% | 1.3M |
 DMRA | Damora Therapeutics, Inc. Common Stock | $1.5b | $25.43 | +11.9% | 1.4M |