Drug Type AAV based gene therapy |
Synonyms Adeno-associated virus serotype 9 human choline acetyltransferase (MDA) |
Target |
Action modulators |
Mechanism CHAT modulators(choline O-acetyltransferase modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Myasthenic Syndromes, Congenital | Preclinical | United States | 22 Oct 2024 |