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Last update 16 Jul 2025
RZ-g001BE
Last update 16 Jul 2025
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms AAV9-hSCN5A, Recombinant self-complementary AAV9 containing a base editor encoding human SCN5A |
Target |
Action modulators |
Mechanism Nav1.5 modulators(Sodium channel protein type V alpha subunit 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 |
|---|---|---|---|---|
| Long QT Syndrome | Preclinical | United States | 15 Apr 2025 |
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Clinical Result
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