Forbes December 8, 2023
TOPLINE
The Food and Drug Administration on Friday approved a new therapy based on Crispr gene editing to treat sickle cell disease, marking the first time a treatment using the technology has secured a regulatory green light in the U.S. amid hopes the revolutionary tool will emerge from the lab and transform medicine.
KEY FACTS
The FDA authorized Crispr-based therapy Casgevy for the treatment of sickle cell disease in patients ages 12 years and older.
Casgevy, which is produced by Boston-based Vertex Pharmaceuticals and Switzerland’s Crispr Therapeutics and is also known as exagamglogene autotemcel, or exa-cel, is a first-of-its-kind treatment that uses the powerful gene-editing tool Crispr to target the underlying cause of sickle cell.
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