Drug Type AAV based gene therapy |
Synonyms AAV-based NPC1 therapy- Sarepta Therapeutics/StrideBio |
Target |
Action- |
Mechanism NPC1 gene transference(NPC intracellular cholesterol transporter 1 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 |
---|---|---|---|---|
Niemann-Pick Disease, Type C | Preclinical | United States | - |