Drug Type AAV based gene therapy |
Synonyms Autologous CD34+HSCs, Beta-globin lentiviral vector transduced autologous HSCs |
Target |
Mechanism HBB gene modulators(Hemoglobin beta chain gene modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 1/2 |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Beta-Thalassemia | Phase 2 | CN | 11 May 2024 | |
Transfusion-dependent Beta Thalassemia | Phase 2 | CN | 11 May 2024 |