Drug Type AAV based gene therapy |
Synonyms Recombinant, replication incompetent adeno-associated virus viral vector platform with a serotype 8 capsid and a cDNA encoding the human BBS10 gene |
Target |
Mechanism BBS10 modulators(Bardet-Biedl syndrome 10 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Bardet-Biedl Syndrome | Preclinical | US | 11 Oct 2024 |