Drug Type AAV based gene therapy |
Synonyms CapStellar rAAV-hAIPL1 gene therapy |
Target |
Action modulators |
Mechanism AIPL1 modulators(aryl hydrocarbon receptor interacting protein like 1 modulators), Gene modulators, Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Leber Congenital Amaurosis | Preclinical | China | 07 May 2024 |