Vertex reported early pediatric results for its CRISPR-based exa-cel (Casgevy) showing clinical benefit in children with sickle cell disease and transfusion-dependent beta-thalassemia, outcomes that the company says support pursuing label expansion into younger age groups. Presentations at ASH 2025 highlighted potential to treat 5–11-year-olds before organ damage accrues. Speakers at ASH and company statements emphasized manufacturing scale and commercialization hurdles remain, including cost and infusion infrastructure. Casgevy’s pediatric data could accelerate commercial uptake if regulators grant expanded indications, but payers and centers will scrutinize long-term durability and access pathways.