It is estimated that around 8,000 people in France suffer from hemophilia, a genetic disease that prevents blood from clotting. Today, the treatment consists of receiving regular injections of the defective clotting factor. A restrictive treatment but which makes it possible to prevent hemorrhagic accidents.
Also, the gene therapy trial just carried out by researchers at University College London (Great Britain) could well advance the treatment. A dose of the gene therapy called FLT180 a was given to 10 patients in a phase 2 clinical trial. Nine of these patients with severe or moderately severe hemophilia B then produced the clotting protein, and that their body does not normally make. And all saw a decrease in bleeding.
The treatment was well tolerated but patients in the trial had to take immunosuppressive drugs for several weeks to prevent their immune systems from rejecting the therapy. They will be followed for the next 15 years to verify that the therapy is durable and does not cause late side effects.
“Removing the need for haemophiliac patients to regularly inject the missing protein is an important step in improving their quality of life,” said Professor Pratima Chowdary, lead author of the study at the UCL Cancer Institute.
Source : Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia BNew England journal of medicine, July 2022
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