Vertex reported early pediatric results for its CRISPR-based sickle cell and beta-thalassemia therapy, Casgevy (exa‑cel), showing clinical improvements in children that meet initial study goals. Presentations and pooled data previewed at ASH 2025 indicate the therapy reduced transfusion dependence and vaso‑occlusive events in younger cohorts, prompting companies and investigators to push for label expansion and pediatric access. The ASH briefing and company updates outlined operational and manufacturing challenges remaining for broader pediatric rollout; cost and one‑time administration logistics remain central to commercial planning. For readers: exa‑cel is a CRISPR/Cas9 edited autologous HSC therapy that modifies hematopoietic stem cells to correct hemoglobinopathies.