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| BBIOBBIOBridgeBio Pharma, Inc. | BridgeBio Pharma, Inc. | $69.52 | $13.6B |
| BMRNBMRNBioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. | $55.99 | $10.8B |
| PTCTPTCTPTC Therapeutics, Inc. | PTC Therapeutics, Inc. | $62.29 | $5.2B |
| QUREQUREuniQure N.V. | uniQure N.V. | $25.01 | $1.7B |
| MGTXMGTXMeiraGTx Holdings plc | MeiraGTx Holdings plc | $9.48 | $910.0M |
| FDMTFDMT4D Molecular Therapeutics, Inc. | 4D Molecular Therapeutics, Inc. | $12.14 | $670.7M |
| RGNXRGNXREGENXBIO Inc. | REGENXBIO Inc. | $6.78 | $447.6M |
| RCKTRCKTRocket Pharmaceuticals, Inc. | Rocket Pharmaceuticals, Inc. | $2.51 | $275.0M |
| ABEOABEOAbeona Therapeutics Inc. | Abeona Therapeutics Inc. | $4.49 | $256.5M |
| MREOMREOMereo BioPharma Group plc | Mereo BioPharma Group plc | $0.29 | $47.0M |