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LB2367 : EFFICACY AND SAFETY OF A SINGLE DOSE OF CTX001 FOR TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA AND SEVERE SICKLE CELL DISEASE

Researchers

Presenter

  • Franco Locatelli

Principal Investigators

  • Selim Corbacioglu

  • Josu de la Fuente

  • Donna Wall

  • Maria Domenica Capellini

  • Mariane de Montalembert

  • Antonis Kattamis

  • Stephan Lobitz

  • Damiano Rondelli

  • Sujit Sheth

  • Martin Steinberg

  • Mark C. Walters

  • Yael Bobruff

  • Chris Simard

  • Yang Song

  • Lanju Zhang

  • Anjali Sharma

  • Suzan Imren

  • Bill Hobbs

  • Stephan Grupp

Medical Centers

  • Hospital for Sick Children, University of Toronto, Toronto, ON, Canada

  • University of Athens, Athens, Greece

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

  • CRISPR Therapeutics,Cambridge,United States

  • IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza,University of Rome,Rome,Italie

  • University of Illinois at Chicago, Pharmacy, Chicago, United States

  • UCSF Benioff Children’s Hospital,Oakland,United States

  • Vertex Pharmaceuticals Incorporated, Boston, United States of America

  • University of Regensburg, Regensburg, Germany

  • University of Milan, Milan, Italy

  • Joan and Sanford I Weill Medical College of Cornell University,New York,United States

  • Imperial College Healthcare NHS Trust St Marys Hospital, London, United Kingdom

  • Gemeinschaftsklinikum Mittelrhein gGmbH, Koeln, Germany

  • Sarah Cannon Center for Blood Cancer at The Children’s Hospital at TriStar Centennial,Nashville,United States

  • Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris,Paris,France

  • Boston University School of Medicine, Boston, MA, United States

Locations

  • Italy

  • United States

  • Germany

  • United Kingdom

  • Canada

  • France

  • Greece

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

  • Gastroenterology (GU)

Disease

  • Sickle cell anemia

  • Thalassemia

Biomarkers

  • Stearoyl-CoA desaturase

  • Volatile organic compounds

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • Pharmacokinetics

Phase

  • N/A

Study Id's

  • NCT03655678

  • NCT03745287

Sponsors

  • N/A

Result

  • N/A

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