FIGURE 2.
The schematic diagram of the coagulation cascades in conjunction with pharmacological agents targeting specific coagulation factors. The current illustration delineates the connection between the extrinsic coagulation cascade via factor VII and the intrinsic coagulation cascade through factor XII, leading to the common pathway that encompasses the transformation of prothrombin into thrombin, and the ensuing conversion of fibrinogen into fibrin. Pharmacological interventions, including AAV-based gene therapies for the synthesis of recombinant factor IX (Hemgenix) and factor VIII (Roctavian), bispecific antibody directing factor IXa and factor X (Emicizumab), monoclonal antibody against the tissue factor pathway inhibitor (TFPI) (Concizumab), an activated recombinant variant of factor VII (Marzeptacog alfa), and antithrombin-directed siRNA (Fitusiran), have received approval from the United States Food and Drug Administration (FDA) for the prophylaxis or treatment of hemophilia.
