The prophylaxis of bleeding disorders usually requires frequent intravenous infusions that result in excessive treatment burden and residual bleeding risk. TGM-148 is a siRNA developed by Tangram Therapeutics plc designed to repress the hepatocyte expression of a gene target to rebalance hemostasis, and has shown efficacy in murine models of hemophilia and von Willebrand disease.
Hemab ApS recently presented preclinical data on its human monoclonal antibody against von Willebrand factor (vWF), HMB-002, for the treatment of von Willebrand disease.
Novo Nordisk A/S recently presented data regarding their antibody fragment Inno-8 for the potential treatment of hemophilia A. Inno-8 was characterized in vitro alone or in combination with hemostatic agents used for treating hemophilia A.
Bispecific antibodies mimicking coagulation factor VIIIa (FVIIIa) have yielded successful results in the treatment of hemophilia A, but there remain some issues in regard to their production.
Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.
Beijing Gensciences Inc. has developed a FVIIIa-mimetic bispecific antibody named SS-315 for the treatment of hemophilia A. SS-315 was developed by targeting FX with its upper Fab arms and FIXa with its down-side scFv arms, respectively; the hemostatic potential of SS-315 was investigated in vitro and in vivo.
RSS
