Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Steven W Pipe¹, Frank W G Leebeek¹, Michael Recht¹+35

• ¹From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L.), Amsterdam, and University Medical Center Groningen, Groningen (K.M.) - all in the Netherlands; Yale University School of Medicine, New Haven, CT (M.R., S.L.); American Thrombosis and Hemostasis Network, Rochester, NY (M.R.); the Department of Medicine and UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (G.C.); the Department of Hemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine and Immunohematology, University Hospital Frankfurt, Frankfurt (W.M.), the Comprehensive Care Hemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn (R. Klamroth) - all in Germany; the Department of Vascular Medicine and Hemostasis, Hemophilia Center, University Hospitals Leuven, Leuven (K.P.), the Division of Hematology, Cliniques Universitaires Saint-Luc, Brussels (C.R.J.R.H.), and Université Catholique de Louvain, Louvain-la-Neuve (C.R.J.R.H.) - all in Belgium; the Department of Hematology, Rigshopitalet Copenhagen, Copenhagen (P.K.); National Coagulation Centre, St. James's Hospital, Dublin (N.O.); Barts and the London School of Medicine and Dentistry, Queen Mary University of London (K.J.P., D.P.H.), and the Royal London Hospital Haemophilia Centre, Barts Health NHS Trust (D.P.H.), London, University Hospital Southampton and National Institute for Health and Care Research Clinical Research Facility, Southampton (R. Kazmi), and Cambridge University NHS Foundation Trust, Addenbrooks Hospital, Cambridge (E.S.) - all in the United Kingdom; the Department of Translational Medicine, Lund University, and the Department of Hematology Oncology and Radiation Physics, Skåne University Hospital - both in Malmö, Sweden (J.A.); the Department of Pediatrics, University of Utah, and Primary Children's Hospital, Salt Lake City (R.L.); University of South Florida, Tampa (N.V.); the Department of Medicine, Hemophilia and Thrombosis Treatment Center, San Diego (A.D.), the Cancer and Blood Disorders Institute, Children's Hospital Los Angeles (G.Y.), the Orthopaedic Hemophilia Treatment Center, the Luskin Orthopaedic Institute for Children (D.V.Q.), and the University of Southern California Keck School of Medicine (G.Y.), Los Angeles, and the Center for Inherited Blood Disorders, Orange (E.G.) - all in California; Arkansas Children's Hospital, Pulaski, and University of Arkansas for Medical Sciences, Little Rock (S.E.C.); University of Texas Health Science Center, McGovern Medical School, and Gulf States Hemophilia and Thrombophilia Center - both in Houston (M.E.); Washington Center for Bleeding Disorders and University of Washington, Seattle (R.K.-J.); Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the Department of Pathology, Microbiology and Immunology, Vanderbilt University Medical Center, Nashville (A.P.W.); uniQure, Lexington, MA (R.G., R.E.D., D.L.C.); and CSL Behring, King of Prussia, PA (Y.L., B.G., P.E.M.).