Evidence generation and reproducibility in cell and gene therapy research: A call to action

• Mohamed Abou-el-Enein,
• Aris Angelis,
• Frederick R. Appelbaum,
• Nancy C. Andrews,
• Susan E. Bates,
• Arlene S. Bierman,
• Malcolm K. Brenner,
• Marina Cavazzana,
• Michael A. Caligiuri,
• Hans Clevers,
• Emer Cooke,
• George Q. Daley,
• Victor J. Dzau,
• Lee M. Ellis,
• Harvey V. Fineberg,
• Lawrence S.B. Goldstein,
• Stephen Gottschalk,
• Margaret A. Hamburg,
• Donald E. Ingber,
• Donald B. Kohn,
• Adrian R. Krainer,
• Marcela V. Maus,
• Peter Marks,
• Christine L. Mummery,
• Roderic I. Pettigrew,
• Joni L. Rutter,
• Sarah A. Teichmann,
• Andre Terzic,
• Fyodor D. Urnov,
• David A. Williams,
• Jedd D. Wolchok,
• Mark Lawler,
• Cameron J. Turtle,
• Gerhard Bauer,
• John P.A. Ioannidis

Affiliations

Mohamed Abou-el-Enein

Division of Medical Oncology, Department of Medicine and Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA; Joint USC/CHLA Cell Therapy Program, University of Southern California and Children’s Hospital Los Angeles, Los Angeles, CA, USA; Corresponding author: Mohamed Abou-el-Enein, Division of Medical Oncology, Department of Medicine and Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.

Aris Angelis

Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, UK; Department of Health Policy and LSE Health, London School of Economics and Political Science, London, UK

Frederick R. Appelbaum

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Division of Medical Oncology, Department of Medicine, University of Washington, Seattle, WA, USA

Nancy C. Andrews

Department of Pharmacology and Cancer Biology and Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA

Susan E. Bates

Department of Medicine, Division of Hematology/Oncology, Columbia University Irving Medical Center, New York, NY, USA

Arlene S. Bierman

Center for Evidence and Practice Improvement, Agency for Healthcare Research and Quality, Rockville, MD, USA

Malcolm K. Brenner

Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA

Marina Cavazzana

Biotherapy Department, Necker Children’s Hospital, Assistance Publique-Hopitaux de Paris, Paris, France; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Quest, INSERM, Paris, France

Michael A. Caligiuri

Hematologic Malignancies and Stem Cell Transplantation Institute, City of Hope National Medical Center, Duarte, CA, USA

Hans Clevers

Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW), Utrecht, the Netherlands; University Medical Center Utrecht, Utrecht, the Netherlands

Emer Cooke

European Medicines Agency, Amsterdam, the Netherlands

George Q. Daley

Boston Children’s Hospital, Boston, MA, USA; Harvard Medical School, Boston, MA, USA

Victor J. Dzau

US National Academy of Medicine, Washington, DC, USA

Lee M. Ellis

Department of Surgical Oncology and Molecular & Cellular Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

Harvey V. Fineberg

Gordon and Betty Moore Foundation, Palo Alto, CA, USA

Lawrence S.B. Goldstein

Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA, USA; Sanford Consortium for Regenerative Medicine, La Jolla, CA, USA

Stephen Gottschalk

Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN, USA

Margaret A. Hamburg

American Association for the Advancement of Science (AAAS), Washington, DC, USA; National Academy of Medicine, Washington, DC, USA

Donald E. Ingber

Boston Children’s Hospital, Boston, MA, USA; Harvard Medical School, Boston, MA, USA; Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, USA; Harvard John A. Paulson School of Engineering and Applied Sciences, Cambridge, MA, USA

Donald B. Kohn

Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; The Eli & Edith Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA

Adrian R. Krainer

Cold Spring Harbor Laboratory, Cold Spring Harbor, NY, USA

Marcela V. Maus

Harvard Medical School, Boston, MA, USA; Massachusetts General Hospital Cancer Center, Charlestown, MA, USA

Peter Marks

Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA

Christine L. Mummery

Department of Anatomy and Embryology, Leiden University Medical Center, Leiden, the Netherlands

Roderic I. Pettigrew

ENMED, Colleges of Medicine and Engineering, Texas A&M University, Houston, TX, USA

Joni L. Rutter

National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA

Sarah A. Teichmann

Wellcome Sanger Institute, Wellcome Genome Campus, Cambridge, UK; Theory of Condensed Matter, Cavendish Laboratory, University of Cambridge, JJ Thomson Ave, Cambridge, UK

Andre Terzic

Center for Regenerative Medicine, Mayo Clinic, Rochester, MN, USA; Department of Cardiovascular Medicine, Mayo Clinic, Rochester, MN, USA

Fyodor D. Urnov

Innovative Genomics Institute, University of California, Berkeley, CA, USA

David A. Williams

Boston Children’s Hospital, Boston, MA, USA; Harvard Medical School, Boston, MA, USA; Division of Hematology/Oncology, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA, USA

Jedd D. Wolchok

Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA; Parker Institute for Cancer Immunotherapy, San Francisco, CA, USA

Mark Lawler

Patrick G Johnston Centre for Cancer Research, Faculty of Medicine, Health and Life Sciences, Queen’s University Belfast, Belfast, UK

Cameron J. Turtle

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Division of Medical Oncology, Department of Medicine, University of Washington, Seattle, WA, USA

Gerhard Bauer

Institute for Regenerative Cures, University of California, Davis, Sacramento, CA, USA

John P.A. Ioannidis

Stanford Prevention Research Center, Department of Medicine, Stanford University, Stanford, CA, USA; Department of Epidemiology and Population Health and Department of Biomedical Data Sciences, Stanford University, Stanford, CA, USA