Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $217.9b | $443.01 | +1.3% | 1.5M |
 VRTX | Vertex Pharmaceuticals Inc | $127.9b | $517.46 | -1.2% | 940.8K |
 ALNY | Alnylam Pharmaceuticals, Inc. | $29.4b | $226.50 | +1.6% | 1.2M |
 INSM | Insmed, Inc. | $24.3b | $94.06 | -0.0% | 3.0M |
 INCY | Incyte Genomics Inc | $23.4b | $112.43 | -2.1% | 1.8M |
 UTHR | United Therapeutics Corp | $23.2b | $592.92 | +0.9% | 461.2K |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $13.1b | $68.18 | -0.6% | 1.3M |
 CORT | Corcept Therapeutics Inc. | $12.6b | $86.50 | +1.7% | 1.7M |
 BMRN | BioMarin Pharmaceuticals Inc | $11.1b | $56.00 | -0.4% | 1.9M |
 KRYS | Krystal Biotech, Inc. Common Stock | $9.5b | $312.94 | +0.7% | 182.3K |
 CYTK | Cytokinetics Inc. | $9.1b | $60.53 | -0.8% | 540.0K |
 ARWR | Arrowhead Research Corporation | $9.1b | $58.16 | +1.6% | 777.3K |
 CRNX | Crinetics Pharmaceuticals, Inc. | $8.8b | $84.97 | 0.0% | N/A |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $7.2b | $43.08 | +1.1% | 1.6M |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $6.4b | $89.50 | +0.4% | 196.9K |
 SRRK | Scholar Rock Holding Corporation Common Stock | $5.7b | $41.42 | +0.6% | 746.1K |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $5.4b | $83.33 | +3.8% | 345.4K |
 COGT | Cogent Biosciences, Inc. Common Stock | $5.4b | $31.52 | -1.0% | 1.8M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $5.4b | $49.87 | -0.2% | 1.9M |
 CRSP | CRISPR Therapeutics AG | $5.4b | $48.98 | -0.8% | 850.2K |
 PTCT | PTC Therapeutics, Inc. | $5.3b | $60.52 | +0.7% | 535.1K |
 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 | $39.18 | -0.3% | 316.4K |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $3.5b | $27.07 | -0.4% | 1.2M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $3.4b | $70.02 | +2.7% | 351.8K |
 DYN | Dyne Therapeutics, Inc. Common Stock | $2.8b | $15.72 | +0.6% | 1.4M |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 LQDA | Liquidia Corporation Common Stock | $2.6b | $43.47 | +2.6% | 3.9M |
 BEAM | Beam Therapeutics Inc. Common Stock | $2.5b | $23.78 | -1.0% | 746.9K |
 IRON | Disc Medicine, Inc. Common Stock | $2.5b | $77.60 | +4.4% | 623.9K |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.3b | $36.90 | +2.0% | 537.4K |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.2b | $22.78 | +4.3% | 1.3M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.2b | $31.54 | +0.5% | 251.7K |
 AUPH | Aurinia Pharmaceuticals Inc | $2.1b | $17.05 | +1.0% | 670.2K |
 BCRX | BioCryst Pharmaceuticals Inc | $2.1b | $7.84 | +3.1% | 2.9M |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $1.9b | $127.19 | +1.1% | 95.8K |
 AGIO | Agios Pharmaceuticals, Inc. | $1.9b | $36.20 | -0.5% | 706.6K |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.8b | $17.75 | +1.5% | 1.0M |
 NTLA | Intellia Therapeutics, Inc | $1.7b | $11.28 | +0.4% | 1.6M |
 ANIP | ANI Pharmaceuticals, Inc. | $1.7b | $73.86 | -0.1% | 157.3K |
 XERS | Xeris Biopharma Holdings, Inc. Common Stock | $1.7b | $10.19 | +0.1% | 1.5M |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.7b | $25.45 | +1.6% | 762.6K |
 QURE | uniQure N.V. | $1.6b | $21.83 | -3.5% | 894.2K |
 ETON | Eton Pharmaceutcials, Inc. Common Stock | $1.6b | $49.69 | +0.3% | 348.3K |
 FBRX | Forte Biosciences, Inc. Common Stock | $1.6b | $76.98 | 0.0% | N/A |
 OCUL | Ocular Therapeutix, Inc. | $1.5b | $6.48 | -0.5% | 1.4M |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.5b | $4.05 | +1.0% | 1.4M |
 RARE | Ultragenyx Pharmaceutical Inc. | $1.5b | $16.08 | +2.7% | 2.6M |