U.K. Approves CRISPR Therapeutic for Sickle Cell Disease
Britain has authorized this therapy - a first of its kind
Britain has authorized a gene therapy that aims to cure sickle-cell disease and another type of inherited blood disorder for patients aged 12 and over, the country's medical regulator said on Thursday, becoming the first in the world to do so.
Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain's Medicines and Healthcare products Regulatory Agency (MHRA) said.
Sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body.
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