Drug Type AAV based gene therapy |
Synonyms adeno-associated viral vector 9 delivering human Glutaryl-CoA Dehydrogenase (GCDH) gene |
Target |
Mechanism GCDH stimulants(glutaryl-CoA dehydrogenase stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 1 |
First Approval Date- |
RegulationOrphan Drug (US), Orphan Drug (EU) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Glutaric Acidemia I | Phase 1 | CN | 25 Dec 2023 |