Presentations previewing ASH data indicated exa‑cel (CRISPR/Cas9 gene editing) produced durable benefits in pediatric patients with transfusion‑dependent beta‑thalassemia and sickle cell disease, supporting study of the therapy in children aged 5–11. Clinicians including Haydar Frangoul highlighted the potential to prevent chronic organ damage by treating younger patients before disease complications accrue. Speakers also outlined commercial and manufacturing learnings from other gene therapy launches, noting cost and supply considerations as companies plan pediatric expansions. Exa‑cel remains one of the leading one‑time gene‑editing therapies for inherited blood disorders.