Rare Disease Therapeutics
Part of Biotech - Drug Development
 AMGN | Amgen Inc | $207.9b | $405.69 | -0.7% | 1.9M |
 VRTX | Vertex Pharmaceuticals Inc | $119.5b | $486.56 | -0.2% | 731.4K |
 ALNY | Alnylam Pharmaceuticals, Inc. | $27.5b | $217.61 | -5.5% | 2.0M |
 INCY | Incyte Genomics Inc | $24.2b | $119.00 | -1.2% | 2.0M |
 UTHR | United Therapeutics Corp | $21.9b | $543.50 | +2.7% | 926.9K |
 INSM | Insmed, Inc. | $21.4b | $131.92 | +33.9% | 11.3M |
 BBIO | BridgeBio Pharma, Inc. Common Stock | $15.7b | $82.82 | +1.0% | 960.3K |
 CORT | Corcept Therapeutics Inc. | $12.4b | $106.93 | -2.5% | 616.0K |
 ARWR | Arrowhead Research Corporation | $11.9b | $85.86 | -1.3% | 784.8K |
 BMRN | BioMarin Pharmaceuticals Inc | $11.6b | $64.42 | +2.6% | 2.8M |
 CYTK | Cytokinetics Inc. | $10.5b | $80.21 | +3.5% | 2.2M |
 KRYS | Krystal Biotech, Inc. Common Stock | $10.1b | $322.25 | -0.9% | 140.1K |
 CRNX | Crinetics Pharmaceuticals, Inc. | $8.8b | $83.69 | -0.1% | 1.7M |
 IONS | Ionis Pharmaceuticals, Inc. Common Stock | $8.6b | $54.48 | +0.5% | 1.9M |
 LQDA | Liquidia Corporation Common Stock | $7.5b | $89.65 | +0.2% | 500.8K |
 RYTM | Rhythm Pharmaceuticals, Inc. Common Stock | $7.1b | $112.63 | +1.3% | 623.4K |
 COGT | Cogent Biosciences, Inc. Common Stock | $6.7b | $42.13 | -1.1% | 803.7K |
 MIRM | Mirum Pharmaceuticals, Inc. Common Stock | $6.4b | $95.42 | -11.0% | 2.8M |
 PTCT | PTC Therapeutics, Inc. | $5.7b | $70.86 | +2.3% | 753.4K |
 SRRK | Scholar Rock Holding Corporation Common Stock | $5.5b | $53.59 | +10.1% | 1.5M |
 APLS | Apellis Pharmaceuticals, Inc. Common Stock | $5.2b | $41.04 | +0.0% | 68.6M |
 TVTX | Travere Therapeutics, Inc. Common Stock | $5.1b | $62.54 | -0.5% | 2.3M |
 CRSP | CRISPR Therapeutics AG | $4.7b | $51.64 | -1.6% | 836.3K |
 DYN | Dyne Therapeutics, Inc. Common Stock | $4.7b | $26.02 | -1.1% | 945.8K |
 FOLD | Amicus Therapeutics, Inc | $4.5b | $14.49 | +0.0% | 3.5M |
 EWTX | Edgewise Therapeutics, Inc. Common Stock | $4.1b | $44.38 | -1.3% | 1.1M |
 CPRX | Catalyst Pharmaceutical Inc. | $3.8b | $31.51 | 0.0% | N/A |
 QURE | uniQure N.V. | $3.1b | $46.79 | +2.7% | 1.1M |
 IRON | Disc Medicine, Inc. Common Stock | $3.0b | $77.44 | -1.7% | 268.4K |
 SLNO | Soleno Therapeutics, Inc. Common Stock | $2.7b | $53.02 | +0.0% | 1.6M |
 TARS | Tarsus Pharmaceuticals, Inc. Common Stock | $2.7b | $65.24 | +6.7% | 1.5M |
 BEAM | Beam Therapeutics Inc. Common Stock | $2.6b | $27.45 | +0.6% | 964.0K |
 RARE | Ultragenyx Pharmaceutical Inc. | $2.5b | $26.11 | +3.6% | 1.5M |
 BCRX | BioCryst Pharmaceuticals Inc | $2.3b | $9.32 | +2.6% | 3.4M |
 AMLX | Amylyx Pharmaceuticals, Inc. Common Stock | $2.3b | $24.10 | +10.7% | 3.1M |
 PHVS | Pharvaris N.V. Ordinary Shares | $2.3b | $34.74 | +2.6% | 535.6K |
 SION | Sionna Therapeutics, Inc. Common Stock | $2.2b | $49.63 | +4.6% | 712.2K |
 VRDN | Viridian Therapeutics, Inc. Common Stock | $2.1b | $22.27 | +6.8% | 3.9M |
 HRMY | Harmony Biosciences Holdings, Inc. Common Stock | $2.0b | $38.60 | +1.3% | 657.6K |
 PVLA | Palvella Therapeutics, Inc. Common Stock | $2.0b | $154.84 | -0.1% | 77.6K |
 AUPH | Aurinia Pharmaceuticals Inc | $1.9b | $16.11 | +6.1% | 2.0M |
 OCUL | Ocular Therapeutix, Inc. | $1.8b | $8.75 | +2.9% | 1.3M |
 STOK | Stoke Therapeutics, Inc. Common Stock | $1.8b | $31.91 | -0.0% | 564.8K |
 ANIP | ANI Pharmaceuticals, Inc. | $1.8b | $84.00 | +3.5% | 650.5K |
 AGIO | Agios Pharmaceuticals, Inc. | $1.8b | $31.21 | +0.7% | 829.0K |
 DMRA | Damora Therapeutics, Inc. Ordinary Shares | $1.8b | $29.89 | -3.1% | 626.6K |
 SRPT | Sarepta Therapeutics,, Inc. Common Stock | $1.7b | $16.77 | +5.3% | 3.7M |
 TSHA | Taysha Gene Therapies, Inc. Common Stock | $1.7b | $6.40 | -1.9% | 1.7M |
 FBRX | Forte Biosciences, Inc. Common Stock | $1.6b | $76.81 | +0.3% | 1.7M |
 NTLA | Intellia Therapeutics, Inc | $1.5b | $11.58 | +2.5% | 3.8M |