Vertex and CRISPR spotlight another important gene editing advance in a march to a hoped-for cure
Vertex and CRISPR Therapeutics have successfully navigated another stretch of the maiden clinical journey of the gene editing therapy CTX001 in a tiny group of patients suffering from beta thalassemia and sickle cell disease. And the researchers involved say they have good reason to believe that they are still on the right track to proving they are advancing a potentially curative approach with a shot at best-in-class status.
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