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Sanquin ADAMTS13 compound

Development stage: Preclinical
Lead developer: Takeda
Modality: Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

Sanquin Innovatie is developing a novel therapeutic compound targeting ADAMTS13 for the treatment of Thrombotic Thrombocytopenic Purpura (TTP). TTP is a rare and life-threatening thrombotic microangiopathy characterized by systemic microvascular thrombi, which result from a deficiency in the ADAMTS13 protease. This deficiency leads to the presence of ultra-large von Willebrand factor (vWF) multimers that spontaneously aggregate platelets. The Sanquin candidate, currently in preclinical development, aims to restore ADAMTS13 activity to normalize vWF processing and prevent the microvascular occlusion and organ damage associated with TTP.

Other names: Sanquin ADAMTS13 modulator

Targets

VWF A2 (von Willebrand factor A2 domain)

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