Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Author:

Locatelli Franco1,Lang Peter2,Li Amanda3,Corbacioglu Selim4,de la Fuente Josu5,Wall Donna A.6,Liem Robert7,Meisel Roland8,Mapara Markus Y.9,Shah Ami J.10,Cappellini Maria Domenica Domenica11,Kattamis Antonis12,Sheth Sujit13,Bobruff Yael14,Bower Laura14,Zhang Lanju14,Sharma Anjali15,Song Yang14,Hobbs William14,Frangoul Haydar16

Affiliation:

1. 1Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy

2. 2University of Tübingen, Tuebingen, Germany

3. 3BC Children's Hospital, University of British Columbia, Vancouver, Canada

4. 4University of Regensburg, Regensburg, Germany

5. 5Imperial College Healthcare NHS Trust, St Mary's Hospital, London, United Kingdom

6. 6The Hospital for Sick Children, Toronto, Canada

7. 7Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

8. 8Heinrich-Heine-University, Duesseldorf, Germany

9. 9Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY

10. 10Stanford University School of Medicine, Stanford, CA

11. 11University of Milan, Milan, Italy

12. 12University of Athens, Kaisariani, Greece

13. 13Joan and Sanford I Weill Medical College of Cornell University, New York, NY

14. 14Vertex Pharmaceuticals Incorporated, Boston, MA

15. 15CRISPR Therapeutics, Cambridge, MA

16. 16Sarah Cannon Center Research Institute and The Children's Hospital at TriStar Centennial, Nashville, TN

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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