Gene treatment hope for haemophiliacs

 A cure for haemophilia is on the horizon following “mind-blowing” results of a landmark gene therapy trial led by the NHS in London.

Researchers at Barts Health NHS Trust and Queen Mary University of London have found that 19 months on from a single treatment with a gene therapy drug, participants with the most common type of haemophilia are effectively cured.

A single infusion of the drug showed improved levels of the essential blood clotting protein Factor VIII, with 85 per cent of patients achieving normal or near-normal levels months later.

There are about 2,000 people with severe haemophilia A in the UK. It puts those affected at risk of excessive bleeding, even from the slightest injury, as well as causing spontaneous internal bleeding, which can be life-threatening.

The gene therapy drug trial saw patients across England injected with a copy of a missing gene, which allows their cells to produce the missing blood clotting protein.