First Time Ever CRISPR Gene-Editing Therapy Approved in the US
Published on December 9, 2023
On Friday, the U.S. Food and Drug Administration (FDA) approved CRISPR and lentiviral vector-based gene therapies for treating sickle cell disease in patients aged 12 years or older. “Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. The two therapies are Casgevy, a CRISPR-based gene therapy manufactured by Vertex, and Lyfgenia from Bluebird Bio, which does not use CRISPR but lentiviral vectors to edit genes. Clinical trial findings showed both treatments could reduce pain from vaso-occlusive events, hallmarks of sickle cell disease....