NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition.
The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’), has been approved for use on the NHS in England by the National Institute for Health and Care Excellence (NICE) for older children and adults with a severe form of sickle cell disease.
Clinical trials suggest exa-cel can stop painful and unpredictable sickle cell crises – the most common symptom of sickle cell disease – where blood vessels become blocked causing severe pain, with experts saying the therapy offers patients a chance of disease-free life.
Researchers concluded there was a ‘functional cure’ in 96.6% of exa-cel trial participants that received it.
NHS chief executive, Amanda Pritchard, said: “More than a step, this is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition.
“This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them.
“It is just the latest in a series of revolutionary gene therapies NHS England has secured for patients, and we are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.”
Source: NHS England, 31 January 2025
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