recombinant factor IX-FIAV

Recombinant Factor IX-FIAV (FIX-FIAV) is an engineered, recombinant biologic variant of blood coagulation Factor IX (FIX) developed by researchers at Erasmus MC (Universitair Medisch Centrum Rotterdam). It is characterized by four specific amino acid substitutions (Y94F, A95aK, K98T, and Y177F) that enable the molecule to function independently of its natural cofactor, activated Factor VIII (FVIIIa). This "bypass" mechanism allows FIX-FIAV to activate Factor X (FX) and restore thrombin generation in patients with hemophilia A, regardless of whether they have developed inhibitors against Factor VIII. Preclinical in vitro and in vivo studies have demonstrated its potential to mitigate the hemophilia A phenotype across all severity levels (severe, moderate, and mild).

Other names

recombinant Factor IX-FIAVFactor IX-FIAV