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154 : Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Researchers

Presenter

  • Alexis A. Thompson

Principal Investigators

  • Janet L. Kwiatkowski

  • John B. Porter

  • Suradej Hongeng

  • Evangelia Yannaki

  • Andreas E. Kulozik

  • Martin G. Sauer

  • Adrian J. Thrasher

  • Isabelle Thuret

  • Ashutosh Lal

  • Ruiting Guo

  • Weijian Liu

  • Richard A. Colvin

  • Mark C. Walters

  • Franco Locatelli

Medical Centers

  • Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

  • Department of Dermatology, Northwestern University Feinberg School of Medicine, Chicago, IL

  • Department of Pathology, The Children's Hospital of Philadelphia, Philadelphia, PA

  • Division of Hematology/Oncology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

  • Haematology Department, University College London Hospitals, London, United Kingdom, United Kingdom

  • Mahidol University and Ramathibodi Hospital, Bangkok, Thailand,

  • Hematology Department/ BMT unit, G.Papanicolaou General Hospital, Thessaloniki, Greece

  • Department of Pediatric Oncology, Hematology and Immunology,, University of Heidelberg, Heidelberg, Germany

  • Molecular Medicine Partnership Unit, European Molecular Biology Laboratory, University of Heidelberg, Heidelberg, Germany

  • Dept. of Hematology/Oncology, Medizinische Hochschule Hannover, Hannover, Germany

  • Great Ormond Street Institute of Child Health, UCL, London, United Kingdom

  • Pediatric Hematology Department, Hopital de La Timone, Marseille, France

  • UCSF Benioff Childrens Hospital Oakland, Oakland, CA

  • bluebird bio, Inc., Cambridge, MA.

  • Department of Pediatric Hematology and Oncology, Cell and Gene Therapy,IRCCS Bambino Gesù Children's Hospital,Roma,Italy

Locations

  • United States

  • United Kingdom

  • Thailand

  • Greece

  • Germany

  • France

  • Italy

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Transfusion-dependent β-thalassemia

Biomarkers

  • Cluster of Differentiation 34

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • N/A

Phase

  • III

Study Id's

  • NCT02906202

  • NCT03207009

Sponsors

  • N/A

Result

  • N/A

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