The U.K. approved its first CRISPR gene-editing drug—and the U.S. is expected to follow
The treatment for blood disorders is widely anticipated to be approved in the U.S. this year—and that’s just the beginning for gene editing in medicine.
Last week, in a global milestone for medicine, U.K. regulators approved the first-ever CRISPR gene-editing drug. The treatment, from Vertex and CRISPR Therapeutics, will be licensed under the brand name Casgevy in the U.K. for patients with sickle cell disease (SCD) and beta thalassemia. The regulatory green light abroad isn’t surprising given the therapy’s established record of clinical trial success in treating these painful, and sometimes fatal, inherited blood disorders. But it marks a historic turning point for CRISPR and the genetic medicines of the future that are expected to reach the U.S. by year’s end.
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