CRISPR Gene Therapy Shows Clinical Benefit in Children with Blood Disorders

A report in the New England Journal of Medicine highlights that CRISPR gene therapy has achieved clinical benefits for the first time in children aged 5-11 years with blood disorders. Specifically, exa-cel gene editing therapy demonstrated significant clinical benefits in children with transfusion-dependent β-thalassemia and sickle cell disease, with all children followed for at least 16 months achieving primary efficacy endpoints.