Drug Type CRISPR/Cas9 |
Synonyms CRISPR/Cas13X RNA base-editing gene therapy (Cholgene), HG 205, HG205 |
Target |
Action modulators |
Mechanism OTOF Q829X modulators |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhaseDiscontinuedEarly Phase 1 |
First Approval Date- |
RegulationOrphan Drug (United States), Rare Pediatric Disease (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Hearing Loss, Sensorineural | Phase 1 | China | 30 Mar 2023 |