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EP733 : CTX001 FOR TRANSFUSION-DEPENDENT Β-THALASSEMIA: SAFETY AND EFFICACY RESULTS FROM THE ONGOING CLIMB THAL-111 STUDY OF AUTOLOGOUS CRISPR-CAS9-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS

Researchers

Presenter

  • Franco Locatelli

Principal Investigators

  • Yael Bobruff

  • Maria Domenica Cappellini

  • Selim Corbacioglu

  • Jennifer Domm

  • Juergen Foell

  • Josu de la Fuente

  • Rupert Handgretinger

  • Tony W. Ho

  • William Hobbs

  • Antonis Kattamis

  • Amanda Li

  • Yimeng Lu

  • Lindsay Palomino

  • Niraj Shanbhag

  • Anjali Sharma

  • Sujit Sheth

  • Parin Sripakdeevong

  • Donna Wall

  • Haydar Frangoul

Medical Centers

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

  • Children’s University Hospital, University of Tübingen,Tübingen,Germany

  • University of Regensburg, Regensburg, Germany

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

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

  • University of Milan, Milan, Italy

  • Vertex Pharmaceuticals Incorporated, Boston, United States of America

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

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

  • CRISPR Therapeutics,Cambridge,United States

  • British Columbia Childrens Hospital & University of British Columbia, Vancouver, Canada

  • University of Athens, Athens, Greece

Locations

  • United Kingdom

  • Germany

  • United States

  • Canada

  • Italy

  • Greece

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Thalassemia

Biomarkers

  • Cluster of Differentiation 34

  • Granulocyte colony-stimulating factor

  • Tumor doubling time

Drug/Treatment

  • Filgrastim

  • Plerixafor

  • Busulfan

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • NCT03655678

Sponsors

  • N/A

Result

  • N/A

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