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Last update 18 Oct 2025
AAV9-GBA1 (NIH/NHGRI)
Last update 18 Oct 2025
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms Adeno-associated virus 9 vector expressing a functional human codon optimized cDNA encoding glucosylceramidase beta 1 (NIH/NHGRI) |
Target |
Action modulators |
Mechanism GBA modulators(Beta-glucocerebrosidase modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (United States) |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Gaucher Disease | Preclinical | United States | 09 Sep 2024 |
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Clinical Result
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