First-ever approved CRISPR-based therapy

This historic milestone marks an important step forward.

We are pleased to announce that CASGEVY™ (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy arising out of our collaboration with Vertex Pharmaceuticals Incorporated, has now been approved in some countries for certain eligible patients with sickle cell disease or transfusion-dependent beta thalassemia.

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 our full pipeline of investigational CRISPR-based therapies.

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Gene Editing

Discover more about our CRISPR/Cas9 technology platform.

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