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.

The heterogenous composition of thrombi limits the efficacy of existing therapies.

The FDA has awarded orphan drug designation to GC Biopharma Corp.’s GC-1126A for thrombotic thrombocytopenic purpura (TTP).