AAV8-FIX

**AAV8-FIX** is an investigational adeno-associated virus serotype 8 (AAV8) vector-based gene therapy designed to treat hemophilia B, a bleeding disorder caused by deficiency in coagulation **factor IX (FIX)**. It delivers a functional human FIX gene to liver cells via intravenous administration, enabling sustained endogenous FIX production and potentially reducing or eliminating the need for frequent FIX infusions. Developed through early clinical efforts including trials sponsored by institutions like St. Jude Children's Research Hospital and University College London, it has been tested in dose-escalation studies for severe or moderately severe hemophilia B, showing dose-dependent FIX expression but with risks of liver inflammation managed by corticosteroids.[1][2]

Other names

AAV8.FIXAAV-8.FIXAAV 8.FIX