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S257 : BETIBEGLOGENE AUTOTEMCEL GENE THERAPY FOR THE TREATMENT OF TRANSFUSION-DEPENDENT Β-THALASSEMIA: UPDATED LONG-TERM EFFICACY AND SAFETY RESULTS

Researchers

Presenter

  • Evangelia Yannaki

Principal Investigators

  • Franco Locatelli

  • Janet L. Kwiatkowski

  • Mark C. Walters

  • John B. Porter

  • Suradej Hongeng

  • Andreas E. Kulozik

  • Martin G. Sauer

  • Adrian J. Thrasher

  • Isabelle Thuret

  • Ashutosh Lal

  • Marina Cavazzana

  • John E.J. Rasko

  • Ruiting Guo

  • Weijian Liu

  • Richard A. Colvin

  • Alexis A. Thompson

Medical Centers

  • The University of Sydney, North Sydney, Australia

  • Mahidol University and Ramathibodi Hospital, Bangkok, Thailand,

  • Bambino Gesu Childrens Hospital, Rome, Italy

  • bluebird bio Inc, Cambridge, United States,

  • Hospital de la Timone, Marseille, France

  • Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, Illinois, United States

  • Medizinische Hochschule Hannover, Hannover, Germany

  • Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM,Paris,France

  • Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States

  • Royal Prince Alfred Hospital, Camperdown, Sydney, Australia

  • University Clinic Heidelberg, Heidelberg, Germany

  • Centenary Institute, Campderdown, Australia

  • George Papanicolaou Hospital, Thessaloniki, Greece

  • INSERM U1016, IMAGINE Institute, Universite Paris Descartes, Sorbonne Paris Cite, Paris, France

  • Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

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

  • University College London Hospitals NHS Trust, London, United Kingdom

  • Ann &amp

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

  • Necker Hospital, Assistance Publique-Hopitaux de Paris, Paris, France

  • Radiology, Children's Hospital of Philadelphia, Philadelphia, United States

Locations

  • Australia

  • Thailand

  • Italy

  • United States

  • France

  • Germany

  • Greece

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Thalassemia

Biomarkers

  • Cluster of Differentiation 34

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • Pharmacokinetics

Phase

  • III

Study Id's

  • NCT02633943

Sponsors

  • N/A

Result

  • N/A

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