Vertex and collaborators presented pediatric data and program updates showing exa‑cel (Casgevy) producing clinically meaningful outcomes in children with sickle cell disease (SCD) and transfusion‑dependent beta‑thalassemia, prompting discussion of label expansion to younger age groups. Company and academic presenters noted the therapy’s potential to provide a one‑time functional cure for children aged 5 to 11, and emphasized earlier intervention before organ damage accrues. The ASH presentations and company statements underscored ongoing work to improve manufacturing, access and commercialization for gene‑edited, one‑time therapies. Speakers included Joanne Lager of Genetix (commenting on commercial learnings) and Haydar Frangoul, who highlighted pediatric clinical benefit. Vertex called Casgevy a national priority in some contexts and said positive pediatric findings could boost uptake and reimbursement discussions. Exa‑cel is a CRISPR/Cas9‑based ex vivo gene editing therapy; pediatric expansion would require trial data, regulatory filings and payer negotiations to address price and access hurdles.