Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients
A “groundbreaking” £1.65m treatment offering a potential cure for people in England living with sickle cell disease has been approved for use on the NHS, the medicines watchdog has announced.
Campaigners welcomed news of the approval of the one-time gene therapy, known as exagamglogene autotemcel, or exa-cel, which edits the faulty gene in a patient’s own stem cells.
