Drug Type Gene therapy, Hematopoietic stem cell therapy |
Synonyms BEAM 104, BEAM104 |
Target |
Action modulators |
Mechanism HBG1 modulators(hemoglobin subunit gamma 1 modulators), HBG2 modulators(hemoglobin subunit gamma 2 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Anemia, Sickle Cell | Preclinical | United States | 01 Feb 2025 | |
Beta-Thalassemia | Preclinical | United States | 01 Feb 2025 |