BioPharma Dive December 8, 2023
Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first medicine based on the Nobel Prize-winning technology to be cleared in the U.S.
The Food and Drug Administration on Friday approved a gene editing treatment for the blood disease sickle cell, following a few weeks behind regulators in the U.K. to clear the world’s first medicine built from the Nobel Prize-winning technology CRISPR.
Called Casgevy, the treatment can free people with severe sickle cell from the excruciating pain crises that are a hallmark of the disease. Researchers expect that benefit to last many years, if not a lifetime, as Casgevy works by permanently — and precisely — changing the DNA of a patient’s own stem cells.
Its approval in...
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