Drug Type AAV based gene therapy |
Synonyms AAV9 gene therapy containing codon optimized human FIG4 cDNA coding sequence (Elpida Therapeutics), AAV9-hFIG4 (Elpida Therapeutics) |
Target |
Mechanism FIG4 modulators(FIG4 phosphoinositide 5-phosphatase modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Charcot-Marie-Tooth Disease, Type 4j | Preclinical | US | 08 Oct 2023 |