Vertex reported early positive outcomes for its CRISPR-based therapy in children with blood disorders, and ASH briefings signaled broader industry momentum to extend one-time gene therapies into younger pediatric populations. Presentations and company releases highlighted improved biomarkers and early clinical benefit in pediatric cohorts for ex vivo gene-editing and gene-addition approaches. Speakers at ASH and company briefings noted manufacturing, safety monitoring and long-term follow-up as immediate barriers to wider pediatric use. Companies referenced include Vertex and Genetix (formerly Bluebird Bio), and presenters emphasized that treating earlier in life may prevent accrued organ damage. For context, exa-cel (CRISPR/Cas9) is the first approved CRISPR therapy and is being evaluated for younger patients; regulators will weigh efficacy signals against lifelong risk-management needs.