Syn-VWFA1

Syn-VWFA1 (also known as anti-vWF A1 domain VHH) is a recombinant, llama-derived single-domain antibody fragment (VHH, or Nanobody) that targets the active, exposed conformation of the A1 domain of von Willebrand factor (vWF). Under high shear stress or due to gain-of-function mutations (such as those in von Willebrand disease type 2B), vWF undergoes a conformational change that exposes its A1 domain, allowing it to bind to the platelet receptor glycoprotein Ibα (GPIbα) and initiate platelet adhesion and aggregation. Syn-VWFA1 selectively binds to this active conformation, blocking the vWF-GPIbα interaction and preventing pathological platelet-vWF aggregate formation. Developed by Maastricht University and the Synapse Research Institute, Syn-VWFA1 is being investigated in exploratory ex vivo human blood studies for its potential to treat or prevent thrombosis in conditions like von Willebrand disease type 2B and thrombotic thrombocytopenic purpura (TTP) without disrupting physiological hemostasis.

Other names

anti-vWF A1 domain VHHanti-von Willebrand factor A1 domain VHHactive von Willebrand factor VHH