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UK Approves World's First CRISPR Gene Therapy for Blood Disorders

U.S.-based companies Vertex Pharmaceuticals and CRISPR Therapeutics welcomed the approval, with optimism for treating various diseases.

Screenshot 2023 11 26 At 3 26 10 Pm
Reuters

According to a recent Reuters article, the United Kingdom has authorized the use of a gene therapy called Casgevy to treat sickle-cell disease and another inherited blood disorder in patients over the age of 12. The move marks the first instance of approval for a medicine using CRISPR, which was awarded the Nobel Prize in 2020. Sickle cell disease and β-thalassemia result from genetic errors affecting the production of hemoglobin, crucial for transporting oxygen in red blood cells. 

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