FDA Approves Sickle Cell Gene Therapy for Young Kids

Posted on: 3 July 2026, source: medpagetoday The FDA on Wednesday expanded the approval for exagamglogene autotemcel (exa-cel, Casgevy) in severe sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta thalassemia to include children 2 years and up. Exa-cel, the first FDA-approved therapy utilizing CRISPR/Cas9 gene editing technology, was previously only approved for adults and pediatric patients 12 years and older. The expanded indication makes exa-cel the first gene therapy approved for kids as young as 2 with these debilitating diseases. {fastsocialshare}