NHS to Offer Groundbreaking Gene Therapy for Sickle Cell Disease

The UK's National Health Service (NHS) is set to introduce a groundbreaking gene therapy for sickle cell disease, costing approximately £1.65 million per treatment course, with an estimated 50 patients expected to benefit annually. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, this one-off treatment, known as Casgevy or Exa-cel, aims to provide a functional cure by editing genes in a patient's stem cells to produce healthy red blood cells. NHS chief executive Amanda Pritchard described the therapy as a monumental step forward, with the potential to transform the lives of patients suffering from the debilitating condition. The therapy has already shown promising results in trials, significantly reducing hospital stays for treated patients. Additionally, the MHRA became the first global regulator to approve this innovative CRISPR-based treatment in 2023. The initiative has been hailed as a significant milestone by health campaigners and the sickle cell community alike.