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Last update 23 Jan 2025
NCATS-BL0746
Last update 23 Jan 2025
Overview
Basic Info
Drug Type Gene therapy |
Synonyms AAV9-hPCCA |
Target |
Mechanism PCCA modulators(propionyl-CoA carboxylase subunit alpha modulators) |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Propionic Acidemia | Preclinical | US | 17 Jun 2022 |
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Clinical Result
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