Figure 1.
Figure 1. Novel alternative hemostatic agents for hemophilia. These hemostatic agents in early-phase trials in subjects with hemophilia A and B, with and without inhibitors, include the following: (1) coagulation factor mutations or mimics (eg, the bispecific monoclonal antibody mimicking FVIII, ACE910; a site-specific mutation of factor V, superFVa; an amino acid substituted factor Xa, factor XaI16→L; and the PACE/furin cleaved FVIII, FVIII ΔP/F); and (2) natural anticoagulant knock-down or disruption (eg, RNA targeting antithrombin, ALN-AT; an albumin-fusion inhibitor of TFPI, an anti-TFPI fusion peptide; and an APC-specific serpin, KRK α1AT).

Novel alternative hemostatic agents for hemophilia. These hemostatic agents in early-phase trials in subjects with hemophilia A and B, with and without inhibitors, include the following: (1) coagulation factor mutations or mimics (eg, the bispecific monoclonal antibody mimicking FVIII, ACE910; a site-specific mutation of factor V, superFVa; an amino acid substituted factor Xa, factor XaI16→L; and the PACE/furin cleaved FVIII, FVIII ΔP/F); and (2) natural anticoagulant knock-down or disruption (eg, RNA targeting antithrombin, ALN-AT; an albumin-fusion inhibitor of TFPI, an anti-TFPI fusion peptide; and an APC-specific serpin, KRK α1AT).

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