Drug Type Gene editing |
Synonyms- |
Target |
Mechanism UBE3A modulators(ubiquitin protein ligase E3A modulators), Ube3a-ATS modulators(small nucleolar RNA host gene 14 modulators) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhasePendingPreclinical |
First Approval Date- |
Regulation- |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Angelman Syndrome | Preclinical | US | 31 Oct 2020 |