First-ever approved CRISPR-based therapy

This historic milestone validates CRISPR/Cas9 gene-editing technology as we look ahead to the future of medicine.

CASGEVY™ (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, is approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia. Sickle cell disease and beta thalassemia are inherited blood disorders that affect the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. Complications from either disease can result in reduced life expectancy.1,2

Vertex is the manufacturer and exclusive license holder of CASGEVY™. The CASGEVY™ word mark and design are trademarks of Vertex Pharmaceuticals Incorporated. Learn more about CASGEVY™ at

1. Beta thalassemia: Genetics Home Reference, U.S. National Library of Medicine.
2. Sickle cell disease: Genetics Home Reference, U.S. National Library of Medicine.


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