Vertex reported early results showing its CRISPR-based therapy Casgevy produced clinical benefit in pediatric patients with sickle cell disease and transfusion-dependent beta-thalassemia, strengthening efforts to expand use in younger children. Presentations and briefings during ASH highlighted potential for a label extension and improved commercial uptake. ASH session materials and company releases noted manufacturing and access remain barriers: approved gene-editing therapies carry multi-million-dollar wholesale acquisition costs and complex delivery pathways. Genetix executives and other industry speakers underscored lessons for scaling one-time curative treatments into sustainable commercial operations. Investigators emphasized that treating children earlier could prevent chronic organ damage, while payers and health systems will need new approaches to financing and distribution to broaden patient access.