Vertex Pharmaceuticals reported early data showing its CRISPR-based gene-editing therapy Casgevy achieved key efficacy goals in children with sickle cell disease and transfusion-dependent beta-thalassemia. Presentations and briefings at the American Society of Hematology (ASH) meeting outlined pediatric results that could support a label expansion for younger patients. Speakers at ASH emphasized potential benefits of treating children earlier to prevent chronic organ damage; Vertex and other gene-therapy developers also discussed manufacturing and commercialization lessons as these high-cost, one-time therapies scale. Casgevy is already considered a national-priority treatment and the pediatric results may drive stronger uptake and potential label changes. Physicians and company executives noted the need to balance access, cost and real-world rollout logistics as payers and health systems prepare for expanded use in younger populations.