Gene Therapy Trial Significantly Reduces Bleeding Risk in Hemophilia B Patients | Genetic

A new gene therapy has dramatically reduced the risk of bleeding in people with the rare haemophilia B disease, experts have said.

The researchers found that a single injection of the gene therapy, called FLT180a, removed the need for people to inject themselves with clotting factors weekly. The study was conducted by experts from University College London (UCL), Royal Free Hospital London and biotech company Freeline Therapeutics.

Hemophilia is a rare disease that affects the blood’s ability to clot. It is usually hereditary and mainly affects men. Normally, when a person cuts themselves, clotting factors mix with blood cells called platelets to stop the bleeding. But people with hemophilia lack clotting factors and are at risk for heavy bleeding.

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About 85% of people with hemophilia have hemophilia A, caused by a lack of blood-clotting factor VIII, while hemophilia B is caused by a deficiency of factor IX.

People with hemophilia B need to inject regularly – usually weekly – to make up for the clotting factor IX deficiency, but may continue to see debilitating joint damage.

In the 26-week trial, published in the New England Journal of Medicine, experts found that a single treatment with FLT180a resulted in sustained production of the protein by the liver in nine out of 10 patients with severe or moderate haemophilia. strict.

This removed the need for their regular injections by correcting their genetic defect and has lasting effects.

Lead author of the study, Professor Pratima Chowdary, from UCL, said: “Eliminating the need for haemophilia patients to regularly inject the missing protein is an important step in improving their quality of life. The long-term follow-up study will monitor patients for durability of expression and monitoring for late effects.

Patients in the trial had to take immunosuppressive drugs for several weeks to months to prevent their immune systems from rejecting the therapy.

Although the treatment was generally well tolerated, all patients experienced some form of side effects, with an abnormal blood clot in the one who received the highest dose of FLT180a and who had the highest levels of protein .

Freeline co-founder Professor Amit Nathwani of UCL, co-author of the study, said: “Gene therapy is still a young field pushing the boundaries of science for people with diseases serious genetics.

He said the trial adds to “the growing body of evidence that gene therapy has the potential to free patients from the challenges of having to adhere to therapy for life or could provide treatment where it doesn’t.” ‘does not exist today’.

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