| INSMINSMED Inc | 415,269 | $67.9M |
| ACADACADIA PHARMACEUTICALS INC | 2,872,695 | $63.9M |
| PTCTPTC THERAPEUTICS, INC. | 921,607 | $62.8M |
| BMRNBIOMARIN PHARMACEUTICAL INC | 685,182 | $38.7M |
| UTHRUNITED THERAPEUTICS Corp | 62,809 | $37.2M |
| ANABANAPTYSBIO, INC | 638,161 | $35.4M |
| MIRMMirum Pharmaceuticals, Inc. | 325,175 | $30.0M |
| DYNDyne Therapeutics, Inc. | 1,652,972 | $30.0M |
| BIIBBIOGEN INC. | 140,719 | $25.8M |
| IRONDisc Medicine, Inc. | 401,719 | $25.7M |
| IOVAIOVANCE BIOTHERAPEUTICS, INC. | 6,349,563 | $22.3M |
| SNDXSyndax Pharmaceuticals Inc | 864,986 | $20.2M |
| ANNXAnnexon, Inc. | 2,948,090 | $16.3M |
| FOLDAMICUS THERAPEUTICS, INC. | 1,113,045 | $16.1M |
| RAREUltragenyx Pharmaceutical Inc. | 596,495 | $12.5M |
| PRTAPROTHENA CORP PUBLIC LTD CO | 1,208,568 | $11.7M |
| BCAXBicara Therapeutics Inc. | 550,408 | $10.9M |
| CLDXCelldex Therapeutics, Inc. | 296,408 | $9.4M |
| ALKSAlkermes plc. | 242,560 | $8.6M |
| CMPXCompass Therapeutics, Inc. | 1,514,504 | $8.0M |
| AGIOAGIOS PHARMACEUTICALS, INC. | 213,601 | $7.2M |
| CAPRCAPRICOR THERAPEUTICS, INC. | 229,763 | $7.0M |
| TVTXTravere Therapeutics, Inc. | 230,453 | $6.8M |
| TSHATaysha Gene Therapies, Inc. | 1,391,382 | $6.2M |
| ACRSAclaris Therapeutics, Inc. | 1,613,430 | $6.1M |
| DNLIDenali Therapeutics Inc. | 234,582 | $4.5M |
| XENEXenon Pharmaceuticals Inc. | 51,345 | $3.0M |
| ORKAOruka Therapeutics, Inc. | 59,985 | $2.9M |
| STOKStoke Therapeutics, Inc. | 84,631 | $2.8M |
| NBIXNEUROCRINE BIOSCIENCES INC | 18,117 | $2.4M |
| NUVBNuvation Bio Inc. | 508,846 | $2.2M |
| CYTKCYTOKINETICS INC | 32,195 | $2.1M |
| CRSPCRISPR Therapeutics AG | 39,605 | $1.9M |
| BNTXBioNTech SE | 19,614 | $1.7M |
| PYXSPyxis Oncology, Inc. | 1,010,510 | $1.5M |
| VSTMVerastem, Inc. | 246,093 | $1.3M |
| BCYCBICYCLE THERAPEUTICS PLC | 201,890 | $937K |
| MDGLMADRIGAL PHARMACEUTICALS, INC. | 1,787 | $935K |
| CMPSCOMPASS Pathways plc | 115,616 | $639K |
| XERSXeris Biopharma Holdings, Inc. | 104,410 | $606K |
| ABEOABEONA THERAPEUTICS INC. | 108,865 | $488K |
| INSPInspire Medical Systems Inc. | 8,772 | $452K |
| URGNUroGen Pharma Ltd. | 24,500 | $441K |
| PRQRProQR Therapeutics N.V. | 188,807 | $306K |
| ALDXAldeyra Therapeutics, Inc. | 155,709 | $263K |