London: Britain’s medicines regulator has authorised the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the UK. In a statement Thursday, the medicines and healthcare regulatory agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize.
The agency approved the treatment for patients with sickle cell disease and thalassemia over 12 years old. Casgevy is made by Vertex Pharmaceuticals (Europe) Ltd and CRISPR Therapeutics.
Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen. Casgevy works by targeting the problematic gene in a patient’s bone marrow stem cells so the body can make properly functioning hemoglobin. Doctors take stem cells from the patient’s bone marrow and use genetic editing techniques in a laboratory to fix the gene. The cells are then infused back into the patient for a permanent treatment.
Vertex Pharmaceuticals said they had not yet established a price for the treatment in Britain and were working with health authorities “to secure reimbursement and access for eligible patients as quickly as possible.”
Casgevy is currently being reviewed by the US Food and Drug Administration; the agency is expected to make a decision early next month.





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