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