GMA-001

Background

Factor IX (FIX) is a vitamin K-dependent zymogen that plays an essential role in the coagulation cascade leading to thrombus formation. In the presence of calcium, activated Factor IX (FIXa) complexes with Factor VIIIa on phospholipid surfaces to create the tenase complex, which converts Factor X to its activated form. Absent or defective FIX is the cause of the X-linked recessive bleeding disorder hemophilia B. GMA-001 (BC2) binds to the N-terminal Gla domain of FIX and the light chain of FIXa, as detected by Western blot and ELISA. In addition, GMA-001 prolongs clotting time in APTT-based plasma clotting assays, and prevents arterial thrombosis in vivo in a rat model¹.