The World’s First Gene Therapy For Sickle Cell Disease Approved In Britain
November 16, 2023 12:53PM EST
FILE - This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and Prevention shows crescent-shaped red blood cells from a sickle cell disease patient in 1972. Britain's medicines regulator has authorized 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 U.K. In a statement on Thursday, Nov. 16, 2023, 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 in 2020. (Dr. F. Gilbert/CDC via AP, File )
LONDON (AP) — Britain’s medicines regulator has authorized 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 illness in the U.K.
In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.
The medicine was approved for patients with sickle cell and thalassemia.
Both diseases are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen.
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