Gene Therapy Breakthrough: A Potential Cure for Sickle Cell Disease Approved for NHS Use
Gene Therapy Breakthrough: A Potential Cure for Sickle Cell Disease Approved for NHS Use
A groundbreaking gene therapy that could potentially cure people with severe sickle cell disease has been approved for use on the NHS in England. The one-time treatment, known as exa-cel, edits the faulty gene in a patient's own stem cells. Clinical trials have shown promising results, with a functional cure in 96.6% of participants. Around 50 patients per year will receive the cutting-edge treatment.