Ablynx‘s Nanobody platform has used the smaller than human monoclonal antibodies from an alpaca to develop the phase III Orphan designated drug Caplacizumab. A phase III study ‘Hercules’ is now pioneering Caplacizumab as the first ever anti-Von Willebrand factor (vWF) nanobody for treating an ultra-rare blood clotting disorder.
Caplacizumab is a pioneering monoclonal antibody (mAb) derived drug developed by Ablynx, which is a spin-off biotech from Vlaams Institute for Biotechnology and the Free University of Brussels, now based in Ghent (Belgium). Caplacizumab has already been rewarded Orphan Drug designation by the EMA and FDA (in 2009), as the first therapy for the congenital and acquired types of the ultra-rare blood clotting disorder thrombocytopenic purpura (TTP).
The use of Alpaca (Camelidae) mAbs has inspired a unique Nanobody platform. This is because of the significantly smaller size of Alpaca antibodies when compared to other mammalian sources, such as mice. By engineering a smaller sized mAb, this therefore allows Nanobodies to target intracellular drug targets as well,