Drug Type AAV based gene therapy |
Synonyms- |
Target |
Mechanism HEXA modulators(hexosaminidase subunit alpha modulators), HEXB modulators(hexosaminidase subunit beta modulators), Gene transference |
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 |
---|---|---|---|---|
Sandhoff Disease | Preclinical | ES | 23 Apr 2024 | |
Tay-Sachs Disease | Preclinical | ES | 23 Apr 2024 |