UK approves gene therapy for sickle cell, thalassaemia

London, November 16

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 on Thursday, the Medicines and Healthcare Regulatory Agency (MHRA) said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.

The agency approved the treatment for patients with sickle cell disease and thalassaemia who are over 12 years old. — AP

 

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