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Last update 08 May 2025
AAV9-hAGA (Rare Trait Hope Fund)
Last update 08 May 2025
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms recombinant serotype 9 adeno-associated virus encoding a codon-optimized human aspartylglucosaminidase (AGA) transgene (Rare Trait Hope Fund) |
Target |
Action modulators |
Mechanism AGA modulators(aspartylglucosaminidase modulators) |
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 |
|---|---|---|---|---|
| Aspartylglucosaminuria | Preclinical | United States | 26 Nov 2024 |
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Clinical Result
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