Presentations previewed at ASH indicate exagamglogene autotemcel (exa‑cel, Casgevy) could benefit children aged 5–11 with transfusion‑dependent beta‑thalassemia or sickle cell disease. Vertex and clinical investigators reported pediatric datasets showing durable hematologic improvements that may support lowering the treatment age from the current 12+ approval. Speakers from Vertex and Genetix emphasized potential for one‑time functional cures if treated before chronic organ damage accrues; discussions also highlighted manufacturing, commercialization costs, and access challenges tied to current multi‑million dollar wholesale acquisition costs.