Vertex reported that Casgevy (exa‑cel), its CRISPR‑based gene editing therapy, met an early clinical goal in pediatric patients with sickle cell disease and transfusion‑dependent beta‑thalassemia. Company statements said the pediatric results support potential label expansion and could strengthen commercial uptake as Casgevy is treated as a national priority in some jurisdictions. Speakers at ASH and company communications emphasized the therapy’s disease‑modifying potential for younger patients—specifically ages 5–11—where earlier treatment could prevent chronic organ damage. Vertex framed the results as evidence that exa‑cel delivers durable clinical improvements in transfusion dependence and vaso‑occlusive events. Clarification: exa‑cel is a one‑time autologous hematopoietic stem‑cell therapy using CRISPR/Cas9 to edit patient cells ex vivo; regulatory expansion to younger age groups requires pediatric safety and efficacy data plus manufacturing scale‑up.