Last update 21 Nov 2024
AAV9-EF1S-NPC1 (NHGRI/NIH)
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms AAV9-hNPC1 is an Adeno-Associated Virus 9 (AAV9) vector expressing a human cDNA encoding Niemann-Pick C1 (NPC1), under control of the Elongation Factor 1 alpha Short (EF1S) promoter |
Target |
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- |
RegulationOrphan Drug (US) |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Niemann-Pick Disease, Type C | Preclinical | US | 23 Aug 2024 |
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Clinical Result
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