Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.

Frangoul H, de la Fuente J, Chopra Y, Meisel R, Amrolia PJ, Algeri M, Sharma A, Cappellini MD, Corbacioglu S, Kattamis A, Lobitz S, de Montalembert M, Rondelli D, Sheth S, Steinberg MH, Walters MC, Boerner K, Liu T, Zairis S, Hobbs W, Grupp SA, Locatelli F, CLIMB THAL-141 and CLIMB SCD-151 Study Groups