Becker's Healthcare December 11, 2023
The FDA made a historic approval Dec. 8 when it cleared Casgevy, the nation’s first medicine based on CRISPR gene-editing technology, as a treatment for sickle cell disease. Its makers are now working with hospitals to establish a network of specialized treatment centers, and so far nine have been authorized to deliver the treatment.
Federal regulators approved exagamglogene autotemcel, or exa-cel, marketed as Casgevy, to treat sickle cell disease in patients 12 and older. The one-time treatment is made by Vertex Pharmaceuticals and CRISPR Therapeutics and works by using CRISPR technology to remove the disease-causing gene from a patient’s stem cells.
“The administration of Casgevy requires specialized experience in stem cell transplantation,” Vertex said in a news release....
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