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EP1301 : INTERIM RESULTS OF BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA (TDT) TREATED IN THE PHASE 3 NORTHSTAR-2 AND NORTHSTAR-3 STUDIES.

Researchers

Presenter

  • Andreas E. Kulozik

Principal Investigators

  • Isabelle Thuret

  • Janet L. Kwiatkowski

  • Alexis A. Thompson

  • John B. Porter

  • Suradej Hongeng

  • Evangelia Yannaki

  • Martin Sauer

  • Adrian J. Thrasher

  • Ashutosh Lal

  • Ruiting Guo

  • Weijian Liu

  • Richard A. Colvin

  • Mark C. Walters

  • Franco Locatelli

Medical Centers

  • University of California, San Francisco, San Francisco, California, United States

  • Sapienza University of Rome, ROMA, Italy

  • Gene and Cell Therapy Center of the Hematology Dept /Hematopoietic Cell Transplantation Unit,Papanicolaou Hospital,Thessaloniki,Greece

  • bluebird bio Inc, Cambridge, United States,

  • Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

  • Department of Medical Oncology, University College London Hospital, London, United Kingdom

  • Pediatrics, Benioff Children's Hospital Oakland, Oakland, CA, United States,

  • Division of Pediatric Rheumatology, The Children's Hospital of Philadelphia, Philadelphia, United States

  • Hematology Section,Feinberg School of Medicine,Chicago,United States

  • bluebird bio Inc, Cambridge, United States,

  • Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Bangkok, Thailand

  • Department Hematology and Oncology, Medical University Hannover, Hannover, Germany

  • Department of Pediatric Hematology,Marseille University,Marseille,France

  • University Medical Center for Children and Adolescents,Heidelberg University,Heidelberg,Germany

Locations

  • United States

  • Italy

  • Greece

  • United Kingdom

  • Thailand

  • Germany

  • France

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Thalassemia

Biomarkers

  • Cluster of Differentiation 34

  • Tumor doubling time

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • Pharmacokinetics

Phase

  • III

Study Id's

  • NCT02906202

  • NCT03207009

Sponsors

  • N/A

Result

  • N/A

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