Gene-editing therapy for blood disorder

The NHS is set to offer the world's first gene-editing therapy, Casgevy, as a potential cure for beta thalassaemia, a hereditary blood disorder requiring regular blood transfusions. Developed by Vertex Pharmaceuticals using CRISPR technology, the therapy modifies patients' bone marrow stem cells to produce functioning haemoglobin and is expected to benefit around 460 eligible patients. Endorsed by the National Institute for Health and Care Excellence (NICE), Casgevy is available through a five-year deal that provides a discount on its list price of £1.65 million. This landmark treatment represents a significant advancement in genetic medicine, offering hope for a better quality of life free from the burdens of lifelong transfusions. However, NICE has expressed concerns regarding the therapy's long-term benefits and cost-effectiveness. As the field of genetic medicines expands, it underscores the importance of patient trust and involvement in clinical development processes.