NHS approves gene therapy exa-cel as potential cure for sickle cell disease in England

A new gene therapy called exa-cel has been approved for use on the NHS in England, offering a potential cure for sickle cell disease. This one-time treatment can edit faulty genes in patients' stem cells and has shown a "functional cure" in 96.6% of trial participants. Previously, the National Institute for Health and Care Excellence rejected the treatment last March, citing a need for more effectiveness data. Now, about 50 patients each year, who lack a matched donor for a stem cell transplant, are expected to benefit from this therapy. Sickle cell disease affects around 17,000 people in England, with 4,000 eligible for exa-cel. The condition causes severe pain and can significantly shorten life expectancy. The approval marks a significant advancement in treatment options for those affected.