Drug Type AAV based gene therapy |
Synonyms AVXS 401, AVXS-401 |
Target |
Mechanism FXN agonists(Frataxin, mitochondrial agonists), 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 |
---|---|---|---|---|
Friedreich Ataxia | Preclinical | US | 28 Apr 2020 |