Drug Type AAV based gene therapy |
Synonyms- |
Target |
Action activators |
Mechanism CPS1 activators(Carbamoyl phosphate synthetase 1 activators) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
License Organization- |
Drug Highest PhasePendingPreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Carbamoyl-Phosphate Synthase I Deficiency Disease | Preclinical | United States | 22 Apr 2019 |