UK approves CRISPR treatment

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The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved the world's first CRISPR gene-editing therapy, called Casgevy, for the treatment of sickle-cell disease and β-thalassaemia. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is a one-time treatment administered via intravenous infusion. The therapy involves extracting blood-producing stem cells from the bone marrow of patients with either disease, using CRISPR to edit the genes encoding for haemoglobin in these cells, and then reintroducing the edited cells back into the patient. In clinical trials, Casgevy completely relieved 28 out of 29 sickle-cell disease participants of debilitating pain episodes for at least one year after treatment. The US Food and Drug Administration and the European Medicines Agency are also reviewing the treatment for both diseases. The cost of Casgevy has not yet been determined in the UK, but estimates suggest it could be around $2 million per patient.
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