Drug Type AAV based gene therapy |
Synonyms NMNAT1 gene therapy (Opus Genetics), OPGx-NMNAT1 + [1] |
Target |
Mechanism NMNAT1 modulators(nicotinamide nucleotide adenylyltransferase 1 modulators), Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Leber Congenital Amaurosis | Preclinical | US | - |