Queen Mary University of London

Ground-breaking gene therapy trial to cure haemophilia

Around 2,000 people in the UK live with severe haemophilia A. They are at risk of excessive bleeding even from the slightest injury as well as spontaneous, life-threatening internal bleeding. Currently the only treatment available involves multiple weekly injections to control and prevent bleeding, and there is no cure.

The first successful gene therapy trial for haemophilia A was carried out in 2017 by John Pasi, Professor of Haemostasis and Thrombosis at Queen Mary University of London and Haemophilia Centre Director at Barts Health NHS Trust. Professor Pasi found that patients treated with just a single treatment of a gene therapy drug are showing vastly improved levels of the essential blood clotting protein Factor VIII, with six out of seven patients achieving normal levels even many months after their single treatment. This research, which was published in the New England Journal of Medicine, offers the potential to transform care for haemophilia patients and brings a cure one step closer.

One patient, Jake Omer, who is married with two young children, said: “The gene therapy has changed my life. I now have hope for my future. It is incredible to now hope that I can play with my kids, kick a ball around and climb trees well into my kids’ teenage years and beyond.”

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