UK health watchdog approves CRISPR therapy for severe sickle cell disease

The UK’s health watchdog, NICE, has approved the use of CRISPR gene editing therapy, exagamglogene autotemcel (exa-cel), for some patients with severe sickle cell disease. This marks a significant change from earlier guidance that rejected its use. Previously, NICE had only approved exa-cel for beta thalassemia. The therapy is now available for patients aged 12 and older who have severe complications and cannot find a suitable stem cell donor. Exa-cel involves editing a patient’s blood stem cells in a lab before returning them to the patient. While it offers hope for some, it is not a cure for all sickle cell patients, and further research is needed.