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TGD001 is a first-in-class fusion protein thrombolytic drug developed by TargED Biopharmaceuticals for the treatment of thrombotic disorders. It is designed to break down blood clots of all sizes and compositions, including those responsible for acute ischemic stroke (AIS) and immune-mediated thrombotic thrombocytopenic purpura (iTTP). The drug employs a unique two-step mechanism of action. First, an antibody fragment (VHH) targets and binds to a non-functional domain of von Willebrand Factor (VWF), which is present in all types of thrombi. In the immediate vicinity of the clot, it then activates endogenous enzyme systems that degrade both VWF and fibrin—two key structural components in blood clots. This targeted approach enables rapid and effective clot dissolution without systemic activation of the lysis cascade, thereby reducing bleeding risk compared to conventional thrombolytics. TGD001 can be administered as a short or bolus injection, making it suitable for emergency settings[1][3][4][6][8].
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