Drug Type AAV based gene therapy |
Synonyms AAV8_ABCA4 RNA Exon Editor (Ascidian Therapeutics) |
Target |
Mechanism ABCA4 stimulants(ATP binding cassette subfamily A member 4 stimulants) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization Ascidian Therapeutics, Inc.Startup |
Active Organization Ascidian Therapeutics, Inc.Startup |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Stargardt Disease | Preclinical | US | Ascidian Therapeutics, Inc.Startup | - |