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BSH19‐OR‐023 : Efficacy and safety of LentiGlobin gene therapy in patients with transfusion‐dependent β‐thalassaemia and non‐β0/β0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar‐2 studies

Researchers

Presenter

  • J.B. Porter

Principal Investigators

  • M.C. Walters

  • J.L. Kwiatkowski

  • S. Hongeng

  • J.E.J. Rasko

  • M.G. Sauer

  • A.J. Thrasher

  • I. Thuret

  • G.J. Schiller

  • H. Elliot

  • B. Deary

  • Y. Chen

  • G. Tao

  • M. Asmal

  • F. Locatelli

  • A.A. Thompson

Medical Centers

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

  • Hematology/Oncology, UCSF Benioff Childrens Hospital Oakland, Oakland, CA

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

  • Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

  • Department of Cell & Molecular Therapies, Royal Prince Alfred Hospital, Camperdown, Australia

  • Pediatric Hematology/Oncology/Blood Cell Transplantation, 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

  • Department of Hematology-Oncology,David Geffen School of Medicine at UCLA,Los Angeles,United States

  • bluebird bio Inc, Cambridge, United States,

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

  • Pediatrics, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States,

Locations

  • United Kingdom

  • United States

  • Thailand

  • Australia

  • Germany

  • France

  • Italy

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Transfusion‐dependent β‐thalassaemia

  • Neutropenia

Biomarkers

  • Cluster of Differentiation 34

  • Ubiquitin A-52 Residue Ribosomal Protein Fusion Product 1

  • Cerebrospinal fluid

  • Red Blood Cells

  • Tumor doubling time

Drug/Treatment

  • Plerixafor

  • Busulfan

Outcome

  • N/A

Study Design

  • Pharmacokinetics

Phase

  • III

Study Id's

  • NCT01745120

  • NCT02906202

Sponsors

  • N/A

Result

  • N/A

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