Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $195.4b | $364.85 | -1.5% | 2.1M |
 VRTX | Vertex Pharmaceuticals Inc | $126.1b | $486.51 | -0.1% | 776.5K |
 ALNY | Alnylam Pharmaceuticals, Inc. | $40.2b | $268.18 | -5.6% | 1.1M |
 INSM | Insmed, Inc. | $23.1b | $109.04 | +0.8% | 1.7M |
 UTHR | United Therapeutics Corp | $23.0b | $535.63 | +1.3% | 310.2K |
 INCY | Incyte Genomics Inc | $22.6b | $117.64 | +0.5% | 1.6M |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $14.6b | $79.99 | +1.4% | 1.1M |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $13.1b | $54.71 | -0.5% | 2.2M |
 CYTK | Cytokinetics Inc. | $11.6b | $80.80 | -0.3% | 1.4M |
 ARWR | Arrowhead Research Corporation | $11.5b | $74.72 | +4.2% | 2.1M |
 BMRN | BioMarin Pharmaceuticals Inc | $11.1b | $58.50 | -0.7% | 1.0M |
 KRYS | Krystal Biotech, Inc. Common Stock | $11.0b | $354.52 | +3.8% | 263.6K |
 CORT | Corcept Therapeutics Inc. | $9.3b | $90.55 | +0.8% | 855.8K |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $7.6b | $109.57 | +0.6% | 684.1K |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $7.1b | $118.02 | +1.2% | 314.4K |
 LQDA | Liquidia Corporation Common Stock | $7.1b | $79.15 | +6.2% | 1.0M |
 PTCT | PTC Therapeutics, Inc. | $6.8b | $78.56 | 0.0% | 537.1K |
 COGT | Cogent Biosciences, Inc. Common Stock | $6.6b | $38.45 | +3.0% | 1.6M |
 SRRK | Scholar Rock Holding Corporation Common Stock | $6.6b | $54.93 | +2.1% | 619.9K |
 CRSP | CRISPR Therapeutics AG | $5.4b | $47.87 | -1.6% | 1.4M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $5.3b | $56.20 | +2.5% | 1.3M |
 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 |
 EWTX | Edgewise Therapeutics, Inc. Common Stock | $4.4b | $38.93 | +4.2% | 493.8K |
 CRNX | Crinetics Pharmaceuticals, Inc. | $3.9b | $83.92 | -0.1% | 4.5M |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 DYN | Dyne Therapeutics, Inc. Common Stock | $3.7b | $24.20 | +4.5% | 1.4M |
 BEAM | Beam Therapeutics Inc. Common Stock | $3.5b | $28.14 | -3.0% | 1.6M |
 RARE | Ultragenyx Pharmaceutical Inc. | $3.3b | $29.28 | -3.1% | 1.2M |
 QURE | uniQure N.V. | $3.2b | $40.65 | +2.3% | 853.4K |
 IRON | Disc Medicine, Inc. Common Stock | $2.8b | $76.15 | +3.7% | 198.3K |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $2.7b | $59.92 | +3.7% | 841.3K |
 BCRX | BioCryst Pharmaceuticals Inc | $2.5b | $9.28 | +2.0% | 2.8M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.4b | $34.98 | +3.7% | 214.2K |
 NTLA | Intellia Therapeutics, Inc | $2.4b | $11.51 | -4.1% | 3.8M |
 AGIO | Agios Pharmaceuticals, Inc. | $2.2b | $39.60 | +2.2% | 647.9K |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $2.2b | $156.96 | +6.6% | 236.3K |
 AUPH | Aurinia Pharmaceuticals Inc | $2.2b | $16.07 | +0.1% | 621.8K |
 OCUL | Ocular Therapeutix, Inc. | $2.2b | $8.99 | -2.2% | 1.6M |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.1b | $33.77 | +0.6% | 564.7K |
 STOK | Stoke Therapeutics, Inc. Common Stock | $2.0b | $29.08 | -0.3% | 313.3K |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.0b | $18.38 | -0.1% | 1.9M |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $2.0b | $17.74 | +4.2% | 1.1M |
 SION | Sionna Therapeutics, Inc. Common Stock | $2.0b | $50.31 | +8.9% | 1.1M |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $2.0b | $6.12 | -0.3% | 2.4M |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.9b | $17.30 | -0.2% | 2.3M |
 ANIP | ANI Pharmaceuticals, Inc. | $1.9b | $79.23 | -3.2% | 262.5K |
 DMRA | Damora Therapeutics, Inc. Common Stock | $1.6b | $31.46 | +4.7% | 437.3K |
 KALV | KalVista Pharmaceuticals, Inc. Common Stock | $1.4b | $27.00 | 0.0% | N/A |