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07 February 2017

A novel drug therapy is to be developed to treat people with haemophilia after a British research team secured £14 million funding.

ApcinteX Limited, the University of Cambridge spin-out company led by Dr Trevor Baglin, of Cambridge University Hospitals, and Professor Jim Huntington from Cambridge Institute for Medical Research, have received the money from Medicxi, Touchstone Innovations and Cambridge Enterprise.

It will enable the academics to begin work on the therapy, which is based on the clinical observation that patients with severe haemophilia who also inherit factor V Leiden – a common genetic mutation associated with thrombosis – have less severe bleeding.

The scientists hope that by reducing the activity of a natural anticoagulant pathway, the drug will restore the balance between coagulation and anticoagulation.

Prof Huntington’s laboratory engineered a protein that 'turns down' the activity of the natural anticoagulant protein C pathway, which then resulted in the blood’s normal clotting capacity.

The standard treatment for haemophilia is administering the missing clotting factor, but this is not always effective and requires regular intravenous infusions.

However, the new drug that is being developed will block the formation of anti-clotting antibodies and it would be administered every few weeks by simple injection under the skin instead of frequent intravenous infusion.

Dr Baglin said: 'Bearing in mind that the majority of people in the world with haemophilia have no access to effective therapy, a stable, easily administered, long-acting, drug that can be used in all patients, regardless of the type of haemophilia, could bring treatment to a great deal many more people who suffer from haemophilia.'


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