A groundbreaking gene-editing therapy for sickle cell disease, known as Exa-cel, will be made available on the NHS for £1.65 million. Experts estimate that around 50 patients per year will benefit from this single-dose treatment, which modifies a specific gene to create healthier red blood cells. Trials indicate significant success, with patients remaining hospital-free for considerable periods after treatment. The therapy is hailed as a monumental step forward, particularly for the 15,000 patients in England, predominantly of Black African and Caribbean descent, who suffer from this debilitating condition.
A gene-editing therapy for sickle cell disease, priced at £1.65m, will be offered on the NHS in England, potentially changing lives for those affected.
Prof Bola Owolabi describes the treatment as a 'monumental step forward', calling it a 'very real prospect of a cure' for sickle cell disease.
In trials, all patients who received the therapy avoided hospital stays for a year; many experienced symptom relief for up to three-and-a-half years.
NHS chief Amanda Pritchard emphasized the therapy's transformative potential, allowing patients to live free from the fear of sickle cell crises.
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