Medical Economics December 11, 2023
Therapies are first to use gene editing technology
In what it calls a “milestone” development, the U.S. Food and Drug Administration (FDA) announced it has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients age 12 and older.
One of the therapies, Casgevy, is the first FDA-approved treatment to use a novel type of gene editing technology, signaling an innovative advance in gene therapy.
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said in the announcement. He added that they reflect “the FDA’s...
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