Drug Type Gene therapy |
Synonyms Lenti-GAA-S1S3, M 023, M023 |
Target |
Action modulators |
Mechanism DPAGT1 modulators(dolichyl-phosphate N-acetylglucosaminephosphotransferase 1 modulators), α-glucosidase modulators(Acidic alpha-glucosidase modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Glycogen Storage Disease Type II | Preclinical | United States | 01 Nov 2024 |