Presentations at ASH preview data suggesting exa-cel (Casgevy) could provide a one-time functional cure for children aged 5–11 with transfusion-dependent beta-thalassemia or sickle cell disease. Companies and investigators highlighted potential benefit of treating patients before chronic organ damage accrues and discussed operational learnings for commercial gene-therapy rollouts. Speakers named Genetix, Vertex and others as contributors to evolving pediatric gene-therapy infrastructure and stressed the need for manufacturing capacity and reimbursement strategies.