Drug Type AAV based gene therapy |
Synonyms Rrecombinant adeno-associated virus serotype 9 vector containing the codon optimized human alpha-N-acetylglucosaminidase (NAGLU) gene |
Target |
Mechanism NAGLU replacements(Alpha-N-acetylglucosaminidase replacements) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseDiscovery |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Mucopolysaccharidosis III | Discovery | US | 01 Aug 2023 |