UK's NHS begins providing Casgevy gene-editing therapy for severe blood disorders like beta-thalassaemia.

The UK's National Health Service will begin providing the first gene-editing therapy, developed by Vertex Pharmaceuticals and Crispr Therapeutics, for patients with severe blood disorders such as beta-thalassaemia. Exagamglogene autotemcel, also known as Casgevy, was approved for use in patients with severe beta-thalassaemia when no bone marrow transplant is available. The treatment works by modifying a faulty gene in the patient's bone marrow stem cells, allowing the body to produce functioning hemoglobin, reducing the need for regular blood transfusions, and improving life expectancy. Casgevy holds the potential to represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions.

August 07, 2024
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