The first-of-its-kind gene-editing tool for sickle cell disease was accelerated to market through a regulatory initiative.
The UK has become the first country to regulate the authorisation of a CRISPR-based gene-editing therapy in the world.
On Thursday, the government announced that new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over.
Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, a technology that edits genes by “precisely cutting DNA and then harnessing natural DNA repair processes to modify the gene in the desired manner.”
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