The UK’s Medicines and Healthcare Regulatory Agency (MHRA) has granted approval for Casgevy, the world’s first gene therapy for sickle cell disease, utilizing the CRISPR gene-editing tool. This landmark decision could bring relief to thousands of individuals in the UK suffering from this debilitating condition. Casgevy, developed by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics, is authorized for patients aged 12 and older with sickle cell disease and thalassemia. Until now, bone marrow transplants were the primary treatment, associated with severe side effects.

Casgevy targets problematic genes in a patient’s bone marrow stem cells, enabling the production of healthy hemoglobin. The therapy involves chemotherapy, stem cell extraction, genetic editing, and reinfusion, requiring at least two hospitalizations. MHRA’s approval is based on studies that showed significant pain reduction in sickle cell patients and a decreased need for red blood cell transfusions in thalassemia patients.

Gene therapy treatments can be expensive, but efforts are underway to negotiate access and reimbursement. The US FDA is currently reviewing Casgevy, with a decision expected soon. The therapy has the potential to benefit millions of people worldwide with sickle cell disease, particularly those from regions where malaria is prevalent.

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