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| IONSIONSIonis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | $43.91 | $7.3B |
| PTCTPTCTPTC Therapeutics, Inc. | PTC Therapeutics, Inc. | $63.04 | $5.3B |
| EWTXEWTXEdgewise Therapeutics, Inc. | Edgewise Therapeutics, Inc. | $39.99 | $4.3B |
| DYNDYNDyne Therapeutics, Inc. | Dyne Therapeutics, Inc. | $15.77 | $2.9B |
| SRPTSRPTSarepta Therapeutics, Inc. | Sarepta Therapeutics, Inc. | $18.56 | $2.0B |
| SLDBSLDBSolid Biosciences Inc. | Solid Biosciences Inc. | $6.19 | $663.7M |
| WVEWVEWave Life Sciences Ltd. | Wave Life Sciences Ltd. | $3.35 | $660.1M |
| PEPGPEPGPepGen Inc. | PepGen Inc. | $2.70 | $186.8M |
| TRDATRDAEntrada Therapeutics, Inc. | Entrada Therapeutics, Inc. | $4.73 | $184.2M |