Drug Type AAV based gene therapy |
Synonyms AAV-based NPC1 therapy- Sarepta Therapeutics/StrideBio, STRX 210, STRX-210 |
Target |
Mechanism NPC1 gene transference(NPC intracellular cholesterol transporter 1 gene transference) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization StrideBio, Inc.Startup |
Active Organization StrideBio, Inc.Startup |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Niemann-Pick Disease, Type C | Preclinical | US | StrideBio, Inc.Startup | - |