A GROUNDBREAKING gene-editing treatment for sickle cell disease has been approved for use in the UK by the NHS, offering the potential for a cure. Known as exagamglogene autotemcel (exa-cel), this one-time therapy uses CRISPR technology to edit genes in a patient’s bone marrow stem cells, allowing the body to produce healthy haemoglobin and stop painful sickle cell crises.
Sickle cell disease, which primarily affects people of Black African and Black Caribbean descent, causes debilitating pain, frequent hospitalisations, and long-term organ damage. Clinical trials have shown that 96.6% of patients who received exa-cel no longer experienced these painful episodes, marking what experts are calling a “functional cure”. All patients who received exa-cel also avoided hospitalisation for a year following treatment, and almost 98% avoided hospitalisation around 3.5 years later.
Exa-cel is now available for older children and adults with the most severe forms of the disease, particularly those who are suitable for a stem cell transplant but lack a matching donor. The treatment will be available at specialist NHS centres in London, Manchester, and Birmingham, with around 50 patients expected to benefit annually. Though the list price is 1.65 million GBP per patient, a reduced cost has been negotiated, making it accessible through the NHS’s Innovative Medicines Fund.
Exa-cel represents a major step forward in the treatment of sickle cell disease, offering hope for a future free from its debilitating effects.
Helena Bradbury, EMJ
Reference
NHS. Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients. Available here: https://www.england.nhs.uk/2025/01/revolutionary-gene-editing-therapy-for-sickle-cell/. Last accessed: 3rd February 2025.
