In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease
The FDA has approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease.
The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.
The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.
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