FDA Expands Gene Therapy Approval for Young Children with Sickle Cell Disease

The U.S. Food and Drug Administration has broadened the approval for Vertex Pharmaceuticals' gene therapy, Casgevy. This allows its use in children as young as two years old who suffer from inherited blood disorders, including sickle cell disease. This marks the first time such a treatment has been cleared for this younger age group, previously only available for those aged 12 and above.