Drug Type Gene therapy |
Synonyms Dual-function gene replacement vector therapy, MSUD AAV9 gene therapy (ASC Therapeutics/University of Massachusetts) |
Target- |
Mechanism 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 |
---|---|---|---|---|
Maple Syrup Urine Disease | Preclinical | US | 02 Sep 2024 |