Presentations at the American Society of Hematology previewed data and strategies to expand exa‑cel (Casgevy) and other gene-editing therapies into children aged 5–11 with transfusion-dependent beta‑thalassemia or sickle cell disease. Companies and investigators said earlier treatment could prevent chronic organ damage and deliver one-time, durable benefits. Speakers including Haydar Frangoul and Genetix executives described operational and manufacturing lessons for scaling gene therapies and noted high wholesale acquisition costs remain a barrier to access. For context: exa‑cel is the first FDA‑approved CRISPR/Cas9 therapy and is currently approved for patients 12 and older; pediatric expansion would require new clinical and regulatory steps.