Exagamglogene autotemcel, which is also known as exa‑cel or Casgevy, is a groundbreaking one‑off gene‑editing therapy now available on the NHS in England for eligible patients with sickle cell disease. This one-time treatment offers the possibility of a functional cure for people living with one of the most common and debilitating inherited blood disorders in the UK.
What is sickle cell disease?
Sickle cell disease affects around 15,000 people in England, primarily from Black African and Black Caribbean communities. The condition causes red blood cells to become rigid and sickle-shaped, restricting blood flow, damaging organs, and causing episodes of excruciating pain known as sickle cell crises. These crises can lead to frequent hospitalisations, chronic fatigue, and serious long-term complications, including stroke and heart failure.
Until now, the mainstay of treatment has involved lifelong blood transfusions, strong pain relief, and other supportive care measures. A small number of patients have been able to access stem cell transplants, but this requires a matched donor, which is often unavailable. Exa-cel represents a significant shift in the way sickle cell disease can be treated by offering eligible patients the potential to live free from the burden of their condition.
How does Exa-cel work?
Exa-cel uses Nobel Prize-winning CRISPR-Cas9 gene-editing technology to modify the patient’s own blood stem cells. These cells are collected, edited in a laboratory to correct the faulty gene, and then reintroduced into the patient’s body through a single infusion. The edited cells then begin producing healthy red blood cells, reducing or even eliminating the symptoms of sickle cell disease.
In clinical trials, the results have been remarkable. Over 96% of patients treated with exa-cel no longer experienced sickle cell crises. Nearly all participants remained out of hospital for at least a year following treatment, with most showing no need for hospital care even after three years. This suggests the therapy could offer long-term relief or even a lifetime cure for many patients.
Who can receive this therapy?
The therapy is now available through the NHS following approval by the National Institute for Health and Care Excellence (NICE), with funding support from the Innovative Medicines Fund. Although the list price of the treatment is £1.65 million, a confidential discount has been agreed with the NHS to ensure affordability. Eligible patients must be 12 years or older, have a history of recurrent sickle cell crises, be suitable for a stem cell transplant, but lack a matched donor.
Manchester is one of just three centres in England selected to deliver exa-cel to NHS patients, alongside sites in London and Birmingham. Specialist teams at Manchester will play a key role in administering this therapy, supported by the city’s strong expertise in genomics, cell therapy, and rare disease care.
The arrival of exa-cel marks a new chapter in the treatment of sickle cell disease. For many patients, it brings the prospect of living without pain, without hospital visits, and without the constant uncertainty that has defined their lives. It is a powerful example of how scientific innovation, when made accessible through the NHS, can transform lives.