ITTP is a serious and life-threating condition characterized by microvascular thrombosis and end-organ damage due to autoantibodies against the ADAMTS13 enzyme. It is a rare disease that affects thousands of patients each year and despite advances in care, there are no approved drugs capable of degrading the pre-existing thrombi. As such, many patients suffer neurological complications and long-term organ damage.

“We are excited to announce that the first patient has been successfully dosed in the iTTP study,” said Kristof Vercruysse, CEO of TargED. “This is an important milestone and we are tremendously grateful to the study physicians, patients and teams who have made this happen”.

TGD001, TargED’s lead program, is a first-in-class, targeted thrombolytic designed to effectively dissolve blood clots in a broad range of thrombotic indications. By degrading both von Willebrand factor (VWF) and fibrin, TGD001 has the potential to dissolve clots of varying composition and location. At the American Society of Hematology (ASH) Annual Meeting in December 2025, and more recently at the European Stroke Organization Conference (ESOC) 2026, TargED reported positive Phase 1 first-inhuman results, demonstrating a favorable safety and tolerability profile, with no spontaneous bleeding events observed. In addition, TGD001 showed potent thrombolytic activity, including rapid plasminogen activation and increased fibrin degradation products (D-dimer), in this Phase 1 study. 

“Based on the very encouraging results of our healthy volunteer trial and our ongoing study in acute ischemic stroke, we are delighted to now be advancing TGD001 for evaluation in patients with iTTP” said Josefin-Beate Holz, MD, Chief Medical Officer at TargED Biopharmaceuticals. “We believe that TGD001 has the potential to redefine the standard of care for iTTP patients by resolving their clot burden and consequently improving outcomes, providing patients and physicians with a new therapeutic option that can reduce severe disability and save lives.” 

About the Phase 1/2 clinical study

The aim of the study is to establish tolerated doses of TGD001 that show signs of pharmacological activity during the dose escalation part, and then to further establish TGD001’s safety profile and early clinical activity in the Phase 2 part, which will also include patients with other types of TMA. The trial expects to enroll up to 70 patients (EU Clinical Trial Identifier: 2025-523802-34-00 and Clinical Trials.gov: NCT07459114). The multi-regional study is currently open for enrollment at sites in Europe and the US, with additional regions and sites in activation.

About TargED Biopharmaceuticals

TargED Biopharmaceuticals B.V. is a clinical-stage biotechnology company pioneering targeted enzyme delivery for improved treatment of thrombotic diseases. Founded in 2020 as a spin-off from the University Medical Center Utrecht, the company has raised more than €80 million to advance its pipeline and was awarded a prestigious blended finance grant from the European Innovation Council (EIC) Accelerator Program in 2025. For more information, please visit www.targedbio.com.

About TGD001

TGD001 is a first-in-class, targeted thrombolytic designed to treat a broad range of thrombotic events. As an antibody-enzyme fusion protein, it binds to von Willebrand factor (VWF) and promotes sitespecific clot breakdown through the enzyme urokinase. Unlike currently approved therapies, TGD001 leverages the ubiquitous presence of VWF in all clots, enabling broad clot lysis by degrading both VWF and fibrin. TGD001 demonstrated compelling Phase 1 results and is in development for the treatment of acute ischemic stroke (AIS) and thrombotic microangiopathies (TMAs), including immune-mediated thrombotic thrombocytopenic purpura (iTTP).