Vertex reported early pediatric results for its CRISPR-based sickle cell therapy Casgevy (exa‑cel) that met key objectives in children, accelerating plans to expand labeling and access. The data presented add to momentum for moving one-time gene-editing treatments into younger age groups to prevent organ damage and lifelong complications. ASH briefings also highlighted the broader push to improve manufacturing, lower costs, and expand commercialization for SCD gene therapies—where initial wholesale acquisition costs reached millions. Speakers emphasized treating younger patients before chronic end‑organ damage accrues. Clarification: Casgevy is an ex vivo CRISPR/Cas9 gene‑editing therapy intended as a one-time intervention for sickle cell disease and transfusion-dependent beta‑thalassemia.