Drug Type AAV based gene therapy |
Synonyms AAV based microdystrophin gene therapy (Ultragenyx/Solid Biosciences), UX-810 |
Target |
Mechanism dystrophin replacements(Dystrophin replacements), 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 |
---|---|---|---|---|
Muscular Dystrophy, Duchenne | Preclinical | US | - | |
Muscular Dystrophy, Duchenne | Preclinical | US | - |