Julie Kanter, MD¹, Alexis A. Thompson, MD, MPH^2,3, Janet L. Kwiatkowski, MD, MSCE^2,4, Suhag Parikh, MD^5*, Markus Mapara, MD⁶, Stacey Rifkin-Zenenberg, DO⁷, Banu Aygun, MD^8,9, Kimberly A. Kasow, DO^10*, Ashish O. Gupta, MBBS, MPH¹¹, Lixin Zhang, PhD^12*, Emily Sheldon-Waniga, PhD^12*, Meghan Gallagher, MSc^12*, Katiana Gruppioni, MPH^12*, Anjulika Chawla, MD, FAAP¹², Heidi Elliot^12*, Francis J. Pierciey, MSc^12*, Mark C. Walters, MD¹³ and John F. Tisdale, MD¹⁴

¹Division of Hematology and Oncology, University of Alabama, Birmingham, AL
²Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
⁴Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
⁵Cellular Therapies for Nonmalignant Diseases Clinic, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA
⁶Blood and Marrow Stem Cell Transplantation Program, Division of Hematology & Oncology, Columbia University Irving Medical Center, New York, NY
⁷Pediatric Hematology/Oncology Division, Hackensack University Medical Center, Hackensack, NJ
⁸Department of Pediatrics, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY
⁹Division of Pediatric Hematology/Oncology and Cellular Therapy, Cohen Children's Medical Center, New Hyde Park, NY
¹⁰University of North Carolina, Chapel Hill, NC
¹¹Division of Pediatric Blood and Marrow Transplant and Cellular Therapies, University of Minnesota, Minneapolis, MN
¹²bluebird bio, Inc., Somerville, MA
¹³UCSF Benioff Children's Hospital, Oakland, CA
¹⁴Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD

Haydar Frangoul, MD¹, Franco Locatelli, MD, PhD², Akshay Sharma, MBBS³, Monica Bhatia, MD^4*, Markus Mapara, MD⁵, Lyndsay Molinari, MD⁶, Donna Wall, MD⁷, Robert I. Liem, MD⁸, Paul Telfer, MD^9*, Ami J. Shah, MD¹⁰, Marina Cavazzana, MD, PhD¹¹, Selim Corbacioglu, MD, PhD^12*, Damiano Rondelli, MD^13*, Roland Meisel, MD^14*, Laurence Dedeken, MD¹⁵, Stephan Lobitz, MSc^16*, Mariane de Montalembert, MD, PhD¹⁷, Martin H. Steinberg, MD¹⁸, Mark C. Walters, MD¹⁹, Laura Bower, MD^20*, Suzan Imren, MD^20*, Christopher Simard, MD^20*, Fengjuan Xuan, PhD^20*, Weiyu Zhou, PhD^20*, Phuong Khanh Morrow, MD^21*, William Hobbs, MD, PhD²⁰ and Stephan Grupp, MD, PhD²²

¹Sarah Cannon Research Institute at The Children’s Hospital at TriStar Centennial, Nashville, TN
²IRCCS, Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy
³St. Jude Children's Research Hospital, Memphis, TN
⁴Department of Pediatrics, Columbia University Irving Medical Center, New York – Presbyterian-Morgan Stanley Children’s Hospital, New York, NY
⁵Division of Hematology and Oncology, Columbia University, New York, NY
⁶Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children’s Hospital, San Antonio, TX
⁷SickKids, Toronto, Canada
⁸Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁹Royal London Hospital, Barts Health NHS Trust, London, United Kingdom
¹⁰Center for Definitive and Curative Medicine, Stanford University, Palo Alto, CA
¹¹Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris, Paris, France
¹²University of Regensburg, Regensburg, Germany
¹³Division of Hematology/Oncology, University of Illinois at Chicago, Chicago, IL
¹⁴Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany
¹⁵Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium
¹⁶Gemeinschaftsklinikum Mittelrhein, Koblenz, Germany
¹⁷Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris-Cité, Paris, France
¹⁸Boston University Chobanian & Avedisian School of Medicine, Boston, MA
¹⁹UCSF Benioff Children's Hospital, Oakland, CA
²⁰Vertex Pharmaceuticals, Boston, MA
²¹CRISPR Therapeutics, Cambridge, MA
²²Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Franco Locatelli, MD, PhD¹, Peter Lang, MD, PhD^2*, Selim Corbacioglu, MD, PhD^3*, Donna Wall, MD⁴, Roland Meisel, MD^5*, Amanda M Li, MD⁶, Josu de La Fuente, PhD⁷, Ami J. Shah, MD⁸, Ben Carpenter, MD, PhD^9*, Janet L. Kwiatkowski, MD, MSCE¹⁰, Markus Mapara, MD¹¹, Robert I. Liem, MD¹², Maria Domenica Cappellini, MD¹³, Mattia Algeri, MD^1*, Antonis Kattamis, MD, PhD¹⁴, Sujit Sheth, MD¹⁵, Stephan Grupp, MD, PhD¹⁶, Puja Kohli, MD, MMSc^17*, Daoyuan Shi, PhD^17*, Leorah Ross, MD, PhD^17*, Yael Bobruff, PhD^17*, Christopher Simard, MD^17*, Lanju Zhang, PhD^17*, Phuong Khanh Morrow, MD^18*, William Hobbs, MD, PhD¹⁷ and Haydar Frangoul, MD¹⁹

¹IRCCS, Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy
²University of Tübingen, Tübingen, Germany
³University of Regensburg, Regensburg, Germany
⁴The Hospital for Sick Children/University of Toronto, Toronto, Canada
⁵Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany
⁶BC Children’s Hospital, University of British Columbia, Vancouver, Canada
⁷Imperial College Healthcare NHS Trust, St Mary's Hospital, London, United Kingdom
⁸Center for Definitive and Curative Medicine, Stanford University, Palo Alto, CA
⁹University College London Hospitals NHS Foundation Trust, London, United Kingdom
¹⁰Children’s Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia, PA
¹¹Division of Hematology and Oncology, Columbia University, New York, NY
¹²Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
¹³University of Milan, Milan, Italy
¹⁴National and Kapodistrian University of Athens, Athens, Greece
¹⁵Joan and Sanford I Weill Medical College of Cornell University, New York, NY
¹⁶Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
¹⁷Vertex Pharmaceuticals, Boston, MA
¹⁸CRISPR Therapeutics, Cambridge, MA
¹⁹Sarah Cannon Research Institute at The Children’s Hospital at TriStar Centennial, Nashville, TN

Thomas J. Harrington, MD¹, Adam Giermasz, MD, PhD^2*, Nathan Visweshwar, MD³, Andrew D. Leavitt, MD⁴, Barbara A. Konkle, MD^5,6, Jeremy Rupon, MD, PhD⁷, Gregory Dirusso, MD⁷, Li-Jung Tseng, PhD, MBA^8*, Maria de los Angeles Resa, PhD^9*, Florence Ganne^10*, Delphine Agathon, PhD^10*, Frank Plonski, MA, RN, BS^7*, Didier Rouy, MD, PhD^11*, Bettina M. Cockroft, MD, MBA^11*, Annie F. Fang, MD, PhD^9* and Steven Arkin, MD¹²

¹Division of Hematology/Oncology, University of Miami Miller School of Medicine, Miami, FL
²University of California, Davis, Sacramento, CA
³University of South Florida, Tampa, FL
⁴University of California San Francisco, San Francisco, CA
⁵University of Washington, Seattle, WA
⁶Washington Center For Bleeding Disorders, Seattle, WA
⁷Pfizer Inc., Collegeville, PA
⁸Pfizer Inc., Peapack, NJ
⁹Pfizer Inc., New York, NY
¹⁰Pfizer Inc., Paris, France
¹¹Sangamo Therapeutics, Brisbane, CA
¹²Rare Hematology Clinical Research, Pfizer Inc, Cambridge, MA

Steven Pipe, MD¹, Paul van der Valk, MD^2*, Peter Verhamme^3*, Peter Kampmann^4*, Frank W.G. Leebeek, MD, PhD⁵, Michiel Coppens, MD^6,7*, Karina Meijer, MD, PhD^8*, Priyanka Raheja^9*, Nigel S. Key, MD¹⁰, Nathan Visweshwar, MD¹¹, Guy Young, MD¹², Richard S. Lemons, MD, PhD^13*, Robert Klamroth, MD^14*, Wolfgang Miesbach, MD^15*, Jan Astermark^16,17, Niamh O'Connell^18*, Rashid Saeed Kazmi, MBBS, FRCP, FRCPath^19*, Nicholas Galante, PhD^20*, Sandra LeQuellec, PhD^20*, Paul Monahan²⁰ and Cedric R. Hermans, MD, PhD²¹

¹University of Michigan, Ann Arbor, MI
²Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
³University Hospitals Leuven, Belgium, Leuven, Belgium
⁴Rigshospitalet, Copenhagen, Denmark
⁵Department of Haematology, Erasmus University Medical Center-Erasmus MC, Rotterdam, Rotterdam, Netherlands
⁶Amsterdam University Medical Centers, Department of Vascular Medicine, University of Amsterdam, Amsterdam, Netherlands
⁷Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
⁸Department of Hematology, University Medical Center Groningen, Groningen, Groningen, Netherlands
⁹Royal London Haemophilia Centre, Bart health NHS trust, London, United Kingdom
¹⁰UNC Blood Reserach Center, Department of Medicine, University of North Carolina, Chapel Hill, NC
¹¹University of South Florida, Tampa, FL
¹²Keck School of Medicine, University of Southern California, Los Angeles, CA
¹³University of Utah, Salt Lake City, UT
¹⁴Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Clinic Friedrichshain, Berlin, Germany
¹⁵Goethe University Hospital, Frankfurt, Germany
¹⁶Department of Hematology, Oncology and Radiation Physics, Skane University Hospital, Malmo, Sweden
¹⁷Department of Translational Medicine, Lund University, Malmö, Sweden
¹⁸National Coagulation Centre, St James’s Hospital, Dublin, Dublin, Ireland
¹⁹University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
²⁰CSL Behring, King of Prussia, PA
²¹Cliniques Universitaires Saint-Luc, Brussels, BEL

Ulrike M. Reiss, MD¹, Andrew M. Davidoff, MD², Edward G. D. Tuddenham, MD^3,4*, Pratima Chowdary^3,4*, Jenny H. McIntosh, PhD^5*, Anne Riddell^6*, Arnulfo Pie^4*, Paul Batty, MBBS, PhD^4,7*, Joanna C. M. Calvert^4,8*, Sarah Mangles^9*, Johnny Mahlangu, MBBCh, MMed¹⁰, Kristina M. Haley, DO^11*, Michael Recht, MD, PhD, MBA^12,13, Yu-Min Shen, MD¹⁴, Kathleen G. Halka, MD¹⁵, Junfang Zhou^2*, Guolian Kang, PhD, MS¹⁶, Victoria Okhomina^16*, Christopher L Morton^17* and Amit C. Nathwani, MD, PhD^18,19

¹Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
²Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
³UCL, London, United Kingdom
⁴Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, United Kingdom
⁵Cancer Institute, University College London, London, United Kingdom
⁶Royal Free London NHS Foundation Trust, London, GBR
⁷Cancer Institute, University College, London, United Kingdom
⁸Cancer Institute, University College London (UCL, London, United Kingdom
⁹Hampshire Hospitals NHS Foundation Trust, Basingstoke, GBR
¹⁰University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
¹¹Oregon Health Science University, Portland, OR
¹²American Thrombosis and Hemostasis Network, Rochester, CT
¹³Yale Center for Bleeding and Clotting Disorders, Yale University School of Medicine, New Haven, CT
¹⁴UT Southwestern Med. Ctr., Dallas, TX
¹⁵Texas A&M Health Science Center College of Medicine, Temple, TX
¹⁶Department of Biostatistics, St. Jude Children’s Research Hospital, Memphis, TN
¹⁷Department of Surgery, St Jude Children's Research Hospital, Memphis, TN
¹⁸Royal Free London - NHS Foundation Trust, London, United Kingdom
¹⁹Cancer Institute, UCL, London, ENG, United Kingdom