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| IONSIONSIonis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | $42.93 | $7.1B |
| PTCTPTCTPTC Therapeutics, Inc. | PTC Therapeutics, Inc. | $60.59 | $5.1B |
| SRPTSRPTSarepta Therapeutics, Inc. | Sarepta Therapeutics, Inc. | $18.56 | $2.0B |
| STOKSTOKStoke Therapeutics, Inc. | Stoke Therapeutics, Inc. | $25.04 | $1.6B |
| SLDBSLDBSolid Biosciences Inc. | Solid Biosciences Inc. | $5.92 | $634.8M |
| WVEWVEWave Life Sciences Ltd. | Wave Life Sciences Ltd. | $3.18 | $626.6M |
| CAPRCAPRCapricor Therapeutics, Inc. | Capricor Therapeutics, Inc. | $6.13 | $355.0M |
| LRMRLRMRLarimar Therapeutics, Inc. | Larimar Therapeutics, Inc. | $2.35 | $244.1M |
| PEPGPEPGPepGen Inc. | PepGen Inc. | $2.52 | $174.3M |
| TRDATRDAEntrada Therapeutics, Inc. | Entrada Therapeutics, Inc. | $4.41 | $171.7M |