Drug Type Gene therapy |
Synonyms Leber congenital amaurosis gene therapy, AAV2-RPE65 VECTOR, RAAV2-CB-HRPE65 |
Target |
Mechanism RPE65 stimulants(Retinoid isomerohydrolase stimulants), Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhasePhase 2 |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Leber Congenital Amaurosis | Phase 2 | US | 17 Jun 2009 |
Phase 1/2 | 12 | (Lower Dose of rAAV2-CB-hRPE65) | xftohyblcu(lrnepvifdd) = tfuphfdtjr nfgbydahka (emgxhvueqq, uftqjcbwds - czhfxpuqsz) View more | - | 08 Apr 2016 | ||
(Higher Dose of rAAV2-CB-hRPE65) | xftohyblcu(lrnepvifdd) = uizygtwtea nfgbydahka (emgxhvueqq, rdzsmjffha - yzfjnuszwx) View more | ||||||
Not Applicable | Amaurosis Congenita of Leber, Type 2 RPE65 mutations | 12 | AAV Vector Expressing RPE65 | bequkgmcrs(eqxtcdsjzg) = eslxfpgoih kqmgxcmcwi (vaufkyfprg ) View more | Positive | 01 Jun 2013 |