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Another step forward for CIMR research into blood clotting disorders

last modified Oct 24, 2019 04:27 PM

The goal of CIMR is to understand the cellular mechanisms of disease in order to improve human health. Translating laboratory discoveries to new treatments is a challenging process with many complex steps. One outstanding example of navigating that path comes from a collaboration on blood clotting disorders which combines the biochemistry and drug discovery expertise of the CIMR’s Prof. Jim Huntington with the clinical knowledge of haematologist Dr Trevor Baglin (formerly of Cambridge University Hospitals). Their collaborative research led to the formation of ApcinteX Ltd in 2014, and this week they announced the successful delivery of the first dose of their drug SerpinPC in a first in human Phase I/II clinical trial. SerpinPC acts by prolonging the activity of the protein complex which generates thrombin through covalent inhibition of Activated Protein C. SerpinPC’s mechanism of action therefore makes it potentially suitable to treat all forms of haemophilia as a once-monthly subcutaneous prophylactic injection. This first clinical study of SerpinPC will focus on safety, tolerability and dosing, with reduction in bleeding as an exploratory endpoint when dosing moves from healthy volunteers to haemophilia patients. This critical step for the company and its founders reflects the commitment of the CIMR to translate our laboratory research into benefits for patients.