Nov 3, 2026 —
The UK’s MHRA has expanded the authorization of Casgevy (exagamglogene autotemcel) to children aged 5 to under 12 with severe sickle cell disease or transfusion-dependent beta-thalassemia, with reimbursement extended to the younger group. Casgevy, from Vertex and CRISPR Therapeutics, was the first CRISPR gene-editing therapy approved anywhere.
Casgevy edits a patient’s own blood stem cells to switch on fetal hemoglobin, compensating for the faulty adult hemoglobin. The expansion brings a one-time, potentially curative option to younger patients earlier in the course of disease.
Access remains gated by the complex manufacturing and conditioning the therapy requires, but widening the eligible age range is a meaningful step.
