Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, offering relief to thousands of people in the UK. The treatment, called Casgevy, is approved for patients aged 12 and over with sickle cell disease and thalassemia.
It is made by Vertex Pharmaceuticals (Europe) Ltd. and CRISPR Therapeutics. This approval marks a significant milestone in the treatment of these inherited blood disorders.
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