Last update 21 Nov 2024
AAV9-hSUMF1 (NCATS/NIH)
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms Recombinant serotype 9 adeno-associated virus encoding codon optimized human sulfatase modifying factor 1 (NCATS/NIH) |
Target |
Mechanism SUMF1 modulators(sulfatase modifying factor 1 modulators) |
Active Indication |
Inactive Indication- |
Originator 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 |
|---|---|---|---|---|
| Multiple Sulfatase Deficiency Disease | Preclinical | US | 20 Sep 2024 | |
| Multiple Sulfatase Deficiency Disease | Preclinical | US | 20 Sep 2024 |
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Clinical Result
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