Single injection of FLT180 removes need for people to inject weekly with clotting factors, study finds
A new gene therapy has dramatically cut the risk of bleeding in people with the rare condition haemophilia B, experts have said.that a single injection of the gene therapy, called FLT180a, removed the need for people to inject themselves weekly with clotting factors. The study was led by experts from University College London , the Royal Free hospital in London and the biotechnology company Freeline Therapeutics.
People with haemophilia B need to inject themselves regularly – usually weekly – to make up for the deficiency in clotting factor IX, but can continue to see debilitating joint damage. This removed the need for their regular injections by correcting their genetic fault and has long-lasting effects.The study’s lead author, Prof Pratima Chowdary, from UCL, said: “Removing the need for haemophilia patients to regularly inject themselves with the missing protein is an important step in improving their quality of life. The long-term follow-up study will monitor the patients for durability of expression and surveillance for late effects.
While the treatment was generally well tolerated, all patients experienced some form of side-effects, with an abnormal blood clot in one who received the highest FLT180a dose and had the highest levels of the protein.
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