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1699 : Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Researchers

Presenter

  • Mark C. Walters

Principal Investigators

  • David H.K. Chui

  • John J Farrell

  • Ashutosh Lal

  • Franco Locatelli

  • Janet L. Kwiatkowski

  • John B. Porter

  • Martin G. Sauer

  • Isabelle Thuret

  • Suradej Hongeng

  • Andreas E. Kulozik

  • Adrian J. Thrasher

  • Evangelia Yannaki

  • Julia Yang

  • Dustin Whitney

  • Alexandria Petrusich

  • Richard A. Colvin

  • Alexis A. Thompson

Medical Centers

  • UCSF Benioff Childrens Hospital Oakland, Oakland, CA

  • Tufts University School of Medicine, Boston, MA

  • The Department of Pediatrics, Sapienza, University of Rome, and the Department of Pediatric Hematology–Oncology, IRCCS Bambino Gesù Children’s Hospital, Rome, Italy

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

  • Childrens Hospital of Philadelphia, Philadelphia, PA

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

  • Medizinische Hochschule Hannover, Hannover, Germany

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

  • Mahidol University and Ramathibodi Hospital, Bangkok, Thailand,

  • University Clinic Heidelberg, Heidelberg, Germany

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

  • George Papanicolaou Hospital, Thessaloniki, Greece

  • bluebird bio, Inc., Cambridge, MA.

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

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

Locations

  • United States

  • Italy

  • United Kingdom

  • Germany

  • France

  • Thailand

  • Greece

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Transfusion-dependent β-thalassemia

Biomarkers

  • Red Blood Cells

  • Tumor doubling time

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • N/A

Phase

  • III

Study Id's

  • NCT02906202

  • NCT03207009

Sponsors

  • N/A

Result

  • N/A

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