NHS England Approves Groundbreaking CRISPR Gene Therapy for Sickle Cell Disease

The National Institute for Health and Care Excellence (NICE) has approved Casgevy (exagamglogene autotemcel) for use in the NHS in England for patients with severe sickle cell disease1 6.

Casgevy is the world's first CRISPR-based gene-editing therapy to receive regulatory authorization5.

The treatment will be available for eligible patients aged 12 and over with severe sickle cell disease who experience recurrent vaso-occlusive crises1 6.

Clinical trials showed that 28 out of 29 sickle cell patients treated with Casgevy were free of severe pain for at least a year3.

NHS England estimates that approximately 50 sickle cell disease patients per year will initially receive Casgevy2.

The therapy has a list price of £1.65 million, but a confidential pricing agreement has been reached to make it affordable within the NHS2 8.

Casgevy will be offered at specialist NHS centers in London, Manchester, and Birmingham3 6.

The approval is seen as a significant step in addressing health inequalities, as sickle cell disease disproportionately affects people from African, Caribbean, Middle Eastern, and South Asian backgrounds1 2.

The treatment involves collecting a patient's stem cells, editing them using CRISPR technology, and then infusing them back into the patient3.

This approval follows a previous agreement in August 2024 for NHS England to access Casgevy for eligible patients with transfusion-dependent beta-thalassemia6.