Drug Type AAV based gene therapy |
Synonyms Adeno-associated virus pseudotype 2/5 human Nephrocystin-5 (NIH/NCATS) |
Target |
Mechanism IQCB1 modulators(IQ motif containing B1 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Retinal Dystrophies | Preclinical | US | 11 Sep 2024 | |
Retinal Dystrophies | Preclinical | US | 11 Sep 2024 |