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Last update 20 Jul 2025
AAV-Mediated Calpain 3 Gene Transfer(Généthon)
Last update 20 Jul 2025
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms AAV9-desmin-hCAPN3 |
Target |
Action modulators |
Mechanism CAPN3 modulators(Calpain-3 modulators) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization- |
Drug Highest PhasePendingPreclinical |
First Approval Date- |
Regulation- |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Limb-Girdle Muscular Dystrophy Type 2A | Preclinical | France | 27 Nov 2019 | |
| Limb-Girdle Muscular Dystrophy Type 2A | Preclinical | Sweden | 27 Nov 2019 |
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Clinical Result
Clinical Result
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