Vertex reported that its CRISPR-based therapy Casgevy reached early efficacy goals in children with sickle cell disease and beta-thalassemia, supporting efforts to expand the label and accelerate commercial uptake. Regulators have flagged Casgevy as a national priority, and the pediatric outcomes may support filing updates and broader pediatric access. The company emphasized safety and durable hematologic improvements in the dataset presented, while noting next steps include confirmatory studies and manufacturing scale-up to meet demand.