Posted by Michael Wonder on 02 Jul 2026
FDA approves first gene therapy for young children with sickle cell disease
1 July 2026 - The US FDA today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β thalassaemia.
This is the first gene therapy approved for patients aged 2 years and older with sickle cell disease.
