Exagamglogene Autotemcel for Severe Sickle Cell Disease

Haydar Frangoul¹, Franco Locatelli¹, Akshay Sharma¹+26

• ¹From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.).