- Data from pivotal studies of CASGEVY in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia demonstrate transformative potential in younger patients, consistent with the durable benefits established in patients 12 years and older -

- Data simultaneously published in the New England Journal of Medicine -

- Regulatory review underway in the United States to expand the use of CASGEVY, and Vertex has recently completed submissions in the Kingdom of Saudi Arabia and United Kingdom -