Drug Type AAV based gene therapy |
Synonyms AAV9-SOD1-shRNA (AveXis), AVXS-301 |
Target |
Mechanism SOD1 gene inhibitors |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhaseDiscontinuedDiscovery |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Amyotrophic Lateral Sclerosis | Discovery | US | - | |
Motor Neuron Disease | Discovery | US | - |