Drug Type Gene therapy |
Synonyms- |
Target |
Mechanism UBE3A modulators(ubiquitin protein ligase E3A modulators), Gene expression 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 |
---|---|---|---|---|
Angelman Syndrome | Preclinical | US | 23 Apr 2024 |