Vertex reported that its CRISPR‑based therapy (Casgevy/exa‑cel) met early clinical goals in children with sickle cell disease and transfusion‑dependent beta‑thalassemia, signaling potential for label expansion. Trial data presented at ASH indicate meaningful clinical benefit in younger patients, reinforcing Casgevy's status as a high‑priority program. Company and clinical presenters noted the results could accelerate pediatric development and adoption, but they also flagged manufacturing and access challenges. CRISPR/Cas9 gene editing modifies patients' hematopoietic stem cells to correct or mitigate disease-causing mutations in blood disorders.