The first targeted
clot busting therapy
HIRING - Management Assistant vacancy
March 2022 - TargED is rapidly expanding its activities, and therefore in need of a new team member. Will you become our new management assistant? Click 'read more' for the job profile!
FUNDING - TargED raises €39 million in Series A round
February 2022 - TargED is extremely pleased and proud to have raised €39 million in a Series A investment round! Together with an international investor syndicate we’ll pursue our common goal: to bring Microlyse into clinical development!
PUBLICATION – Microlyse for TTP in Blood journal
January 2022 - Our new clot-buster Microlyse is now published in- and on the cover of Blood. Click read more to download the paper or to listen to the Blood podcast.
FUNDING – TargED partners with GBS (Leiden) and is granted MIT R&D
March 2021 - TargED joins forces with Good Biomarker Sciences (GBS) to develop a biomarker assay that supports TargED lead compound, Microlyse towards and throughout clinical studies. The collaboration is supported with a MIT R&D grant from the Economic Board Utrecht.
Kristof Vercruysse has over 15 years of experience in bringing biopharmaceutical compounds from preclinical proof of concept towards market authorization. Kristof gained hands-on experience with the development of Caplacizumab for treatment of Thrombotic Thrombocytopenic Purpura at Ablynx between 2007 and 2013. Subsequently, he joined Sourcia, a full-service boutique CRO that delivers tailor-made clinical drug development for biotech companies. At TargED, Kristof’s objective is to lead MICROLYSE towards the market as an entrepreneur. Kristof holds a Masters in Biomedical Science from Vrije Universiteit Brussel.
Marc van Moorsel holds a Masters in Innovation Sciences from Vrije Universiteit Amsterdam and is currently finishing his PhD at University Medical Centre Utrecht, investigating blood vessel markers towards innovative diagnostics and therapeutics in cardiovascular disease. Marc aims to combine his scientific understanding of drug development gained during his PhD with his passion: establishing a biopharmaceutical start-up.
Coen Maas is an expert in thrombosis and hemostasis and associate professor at University Medical Centre Utrecht. Coen studies multiple rare, hematological diseases and believes that these misunderstood conditions can often be directly extrapolated to more prevalent multifactorial diseases, including stroke, atherosclerosis, diabetes, and Alzheimer’s disease. Coen is (co)author of >80 scientific publications and has been invited for >45 scientific presentations. In addition, he is inventor of two patents, one of which that covers our lead compound. Coen holds a Masters in Medical Biology and received his PhD in 2008, both at University Medical Centre Utrecht.
Board of Directors
Our lead product, Microlyse, is a first-in-class proprietary clot-busting compound which binds to a protein present in all forms of thrombosis. Microlyse is the first compound to achieve targeted enzyme delivery, using a single domain antibody (VhH), directly to blood clots. Due to the targeted nature of the therapy, it is expected to lead to a superior side effect profile alongside the potential for exceptional potency compared to current agents. Microlyse’s unique mechanism of action allows for potential applications in a variety of indications involving thrombosis, including the life-threatening disease aTTP. The ground-breaking work demonstrating that Microlyse specifically targets blood clots leading to their desolution faster and safer compared to standard of care recently featured on the cover of the journal Blood (Microlyse: a thrombolytic agent that targets VWF for clearance of microvascular thrombosis).
The human body controls rupturing of vessels by beneficial wound healing. Wound healing is initiated by the binding of platelets to unrolled Von Willebrand Factor (VWF), a multimeric blood protein. The developing molecular ‘band-aid’ can later be strengthened by the formation of fibrin. When this process is uncontrolled, obstructive blood clots can develop, known as ‘thrombosis’. These clots prevent sufficient blood supply to organs. Dependent on the affected organ, this can result in for example stroke, myocardial infarction or kidney failure. Interestingly, thrombi always contain platelets and VWF, but they do not always contain fibrin. We are currently developing Microlyse for the thrombotic indications TTP and acute ischemic stroke.
Thrombotic Thrombocytopenic Purpura (TTP)
TTP has an orphan EMA/FDA designation and it affects 2-10 patients per million per year. In physiology, the enzyme ADAMTS13 cleaves VWF and prevents progressing into pathological thrombosis. In TTP patients, ADAMTS13 activity is impaired. Consequently, TTP patients experience acute attacks during which they develop countless thrombi composed of ultra large VWF molecules (UL-VWF) and platelets. These thrombi generally affect the microvasculature of organs such as the kidneys, heart and brain, leading to tissue ischemia and multi-organ failure. TTP patients risk shortened life expectancy and long-term complications. When untreated, mortality is 90%.
Microlyse is revolutionary in inducing the active degradation of VWF strands. As a result, the duration of tissue ischemia can be reduced, resulting in less organ failure and lower mortality.
Acute ischemic stroke (AIS)
AIS is the largest thrombotic indication worldwide, affecting 17 million people per year. Since the late 90s, AIS patients are treated with recombinant tPA (tissue plasminogen activator) to recanalize the occluded blood vessel. tPA becomes less effective when administered later after symptom onset. Consequently, administration >4.5 hours after onset is avoided as efficacy is outweighed by the unavoidable bleeding risk that comes with tPA therapy.
The reasons for the limited efficacy of tPA are still largely unclear. However, tPA requires the availability of fibrin in order to degrade thrombi. In AIS, thrombi often originate from vessel damage elsewhere, after which thrombi-parts dislodge, embolize and occlude arteries in the brain. These thrombi always contain VWF, but the presence of fibrin is uncertain and varies between cases. We and others therefore suggest that degrading VWF in addition to fibrin forms an attractive strategy to treat thrombosis in stroke.