Drug Type AAV based gene therapy |
Synonyms Recombinant adeno-associated virus 8 (AAV8) expressing human retinoschisin (RS1) gene (Genevector), ScAAV8-hRS1, JWK 002 + [1] |
Target |
Action stimulants |
Mechanism XLRS1 stimulants(Retinoschisin stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePhase 1/2 |
First Approval Date- |
RegulationOrphan Drug (United States), Rare Pediatric Disease (United States) |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Retinoschisis | Phase 2 | China | 26 Jun 2025 |
Phase 1 | RS1突变 | 4 | lupdhvodmo(qprowgbivt) = rrpqhunklv beoopjmkxj (nlgidyrgsx ) View more | Positive | 06 May 2024 |





