An overview of novel innovative treatments for patients with von Willebrand disease 

Caterina Casari, PhD, Paris-Saclay University, Inserm UMR-S1176 Hemostasis Inflammation and Thrombosis, Paris, France, gives an overview of novel innovative treatments for patients with von Willebrand disease (vWD). First, some therapies aim to increase endogenous von Willebrand factor (vWF) levels, such as an albumin- and vWF-targeted bispecific nanobody for patients with vWD type 1, or BT200, a vWF-targeted aptamer, for those with type 2b disease. Second, treatments that increase procoagulant potential, such as emicizumab or BIVV001, could benefit specific subsets of patients. Thirdly, general hemostatic agents such as platelet inspired nanoparticles could help to reduce bleeding symptoms. Dr Casari then goes on to discuss the challenges of using gene therapies in this disease, as well as the potential of silencing RNA (siRNA) approaches. This interview took place at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Bangkok, Thailand, and virtually.
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