Drug Type AAV based gene therapy |
Synonyms liver-targeted factor IX gene therapy (Intellia/Regeneron) |
Target |
Mechanism factor IX stimulants(Coagulation factor IX stimulants), Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Hemophilia B | Phase 3 | US | - | |
Hemophilia B | Preclinical | US | - |