Drug Type AAV based gene therapy |
Synonyms Adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region, Stargardt disease therapy programs (Splicebio), SB 007 + [2] |
Target |
Mechanism ABCA4 modulators(ATP binding cassette subfamily A member 4 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseIND Approval |
First Approval Date- |
RegulationOrphan Drug (US), Orphan Drug (EU) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Stargardt Disease | IND Approval | US | 12 Dec 2024 |