A revolutionary gene-editing treatment for sickle cell disease, priced at £1.65m, is set to be made available on the NHS in England. The therapy, known as Casgevy or Exa-cel, is expected to benefit approximately 50 patients each year. Experts have hailed it as a significant breakthrough, with NHS England’s Prof Bola Owolabi describing it as a “monumental step forward” and offering the potential for a cure.

Sickle cell disease, a life-threatening condition that affects around 15,000 people in England, is caused by faulty haemoglobin, which leads to misshapen red blood cells. These cells block blood vessels, causing pain and reducing oxygen flow to vital organs. The new gene therapy, which involves editing a specific gene to increase the production of healthy red blood cells, has shown promising results in clinical trials. Patients who received the treatment avoided hospital stays for up to three and a half years.

NHS CEO Amanda Pritchard called the treatment “transformative,” offering patients relief from the chronic pain and fear of sickle cell crises. The therapy is available in specialist centres in London, Manchester, and Birmingham for patients aged 12 and over who have recurrent crises and cannot find a suitable stem cell donor.

While campaigners and patients alike have welcomed the move, there are still concerns about ensuring all individuals with sickle cell disease have access to necessary treatments and support.