CASGEVY® Gene Therapy Data for Children with Blood Disorders Presented

Vertex Pharmaceuticals presented new clinical data on its gene therapy, CASGEVY® (exagamglogene autotemcel), at the European Hematology Association Congress, including the first European presentation of results in children aged 5–11. The pivotal studies demonstrated efficacy and safety outcomes in this age group consistent with those observed in adult and adolescent patients. These findings, which relate to sickle cell disease and transfusion-dependent beta thalassemia, were also published in The New England Journal of Medicine.