Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $217.9b | $401.95 | -0.1% | 1.4M |
 VRTX | Vertex Pharmaceuticals Inc | $127.9b | $501.99 | -0.2% | 475.7K |
 ALNY | Alnylam Pharmaceuticals, Inc. | $29.4b | $227.15 | +1.8% | 1.2M |
 INSM | Insmed, Inc. | $24.3b | $104.95 | +1.3% | 3.1M |
 INCY | Incyte Genomics Inc | $23.4b | $114.28 | -1.2% | 2.1M |
 UTHR | United Therapeutics Corp | $23.2b | $541.72 | +1.0% | 518.2K |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $13.1b | $67.03 | +1.3% | 3.4M |
 CORT | Corcept Therapeutics Inc. | $12.6b | $121.17 | -2.0% | 595.9K |
 BMRN | BioMarin Pharmaceuticals Inc | $11.1b | $56.23 | -2.3% | 2.5M |
 KRYS | Krystal Biotech, Inc. Common Stock | $9.5b | $330.83 | -2.3% | 299.9K |
 CYTK | Cytokinetics Inc. | $9.1b | $58.22 | -2.1% | 2.7M |
 ARWR | Arrowhead Research Corporation | $9.1b | $61.54 | -1.3% | 1.4M |
 CRNX | Crinetics Pharmaceuticals, Inc. | $8.8b | $84.97 | 0.0% | N/A |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $7.2b | $44.10 | +1.2% | 1.9M |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $6.4b | $86.05 | -1.7% | 791.4K |
 SRRK | Scholar Rock Holding Corporation Common Stock | $5.7b | $42.39 | -2.2% | 1.5M |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $5.4b | $79.22 | -2.7% | 921.1K |
 COGT | Cogent Biosciences, Inc. Common Stock | $5.4b | $30.45 | -1.7% | 2.0M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $5.4b | $56.96 | -2.9% | 1.6M |
 CRSP | CRISPR Therapeutics AG | $5.4b | $54.16 | -5.9% | 2.4M |
 PTCT | PTC Therapeutics, Inc. | $5.3b | $63.66 | +0.0% | 781.5K |
 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 | $4.5b | $39.48 | -0.6% | 963.6K |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $3.5b | $28.04 | +1.0% | 1.1M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $3.4b | $74.17 | -0.5% | 340.8K |
 DYN | Dyne Therapeutics, Inc. Common Stock | $2.8b | $15.83 | -0.2% | 3.6M |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 LQDA | Liquidia Corporation Common Stock | $2.6b | $25.93 | -6.3% | 4.7M |
 BEAM | Beam Therapeutics Inc. Common Stock | $2.5b | $25.52 | -1.7% | 2.2M |
 IRON | Disc Medicine, Inc. Common Stock | $2.5b | $61.65 | -2.7% | 519.2K |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.3b | $39.35 | -1.4% | 372.5K |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.2b | $18.22 | -4.1% | 2.0M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.2b | $30.97 | +2.2% | 682.8K |
 AUPH | Aurinia Pharmaceuticals Inc | $2.1b | $15.71 | -0.2% | 591.7K |
 BCRX | BioCryst Pharmaceuticals Inc | $2.1b | $7.96 | -1.7% | 2.7M |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $1.9b | $128.50 | -2.7% | 245.6K |
 AGIO | Agios Pharmaceuticals, Inc. | $1.9b | $31.73 | +1.6% | 771.4K |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.8b | $17.32 | -1.0% | 1.9M |
 NTLA | Intellia Therapeutics, Inc | $1.7b | $12.40 | -3.0% | 7.3M |
 ANIP | ANI Pharmaceuticals, Inc. | $1.7b | $71.53 | -1.4% | 225.3K |
 XERS | Xeris Biopharma Holdings, Inc. Common Stock | $1.7b | $10.18 | -0.5% | 1.9M |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.7b | $24.36 | -3.8% | 591.6K |
 QURE | uniQure N.V. | $1.6b | $23.14 | +1.1% | 2.5M |
 ETON | Eton Pharmaceutcials, Inc. Common Stock | $1.6b | $54.72 | -1.4% | 408.6K |
 FBRX | Forte Biosciences, Inc. Common Stock | $1.6b | $76.98 | 0.0% | N/A |
 OCUL | Ocular Therapeutix, Inc. | $1.5b | $7.35 | +1.3% | 2.4M |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.5b | $4.58 | -2.2% | 2.7M |
 RARE | Ultragenyx Pharmaceutical Inc. | $1.5b | $14.48 | -0.2% | 3.4M |