Last update 26 Nov 2024
BGT-NPC
Last update 26 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms BGT NPC, BGTNPC |
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 (EU), Orphan Drug (US) |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Niemann-Pick Disease, Type C | Preclinical | GB | - |
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Clinical Result
Clinical Result
Indication
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