Drug Type Gene therapy |
Synonyms Autologous CD34+ hematopoietic stem and progenitor cells modified ex vivo with a lentiviral vector that restores endogenously regulated expression of WASp |
Target |
Action modulators |
Mechanism CD34 modulators(Hematopoietic progenitor cell antigen CD34 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseDiscovery |
First Approval Date- |
RegulationOrphan Drug (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Wiskott-Aldrich Syndrome | Discovery | United States | 18 Oct 2023 |