Drug Type CRISPR/Cas9 |
Synonyms CRISPR/Cas13X RNA base-editing gene therapy (Cholgene) |
Target |
Mechanism OTOF Q829X modulators |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseEarly Phase 1 |
First Approval Date- |
RegulationRare Pediatric Disease (US), Orphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Hearing Loss | Phase 1 | CN | 06 Sep 2023 |