Matthew Dickerson^*, Amber Lee^*, Anne Hale^* and Keith A. Moskowitz, PhD

Cellphire Inc., Rockville, MD

Midori Shima, MD¹, Sayaka Nagami, BSc^2*, Koichiro Yoneyama, MSc^2*, Akira Nomura, MSc^2*, Yoshiyuki Ogawa, MD, PhD^3*, Kagehiro Amano, MD, PhD^4* and Keiji Nogami, MD, PhD^5*

¹Thrombosis and Hemostasis Research Center, Nara Medical University, Kashihara, Japan
²Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
³Department of Hematology, Gunma University Graduate School of Medicine, Maebashi, Japan
⁴Department of Laboratory Medicine, Tokyo Medical University, Tokyo, Japan
⁵Department of Pediatrics, Nara Medical University, Kashihara, Japan

Mari Thomas^1*, Nishkantha Arulkumaran^2*, David Brearley^3*, Ferras Alwan⁴, Deepak Singh^5*, Michael P Lunn^6*, Anna Welch^7*, Samuel Clark^8*, Eamon Raith^9*, Ugan Reddy^9*, Ryan Low^10*, David Leverett^7*, Mervyn Singer^11* and Marie Scully, MD¹²

¹Department of Haematology, University College London Hospitals, Cardiometabolic Program, National Institute for Health Research UCLH/UCL Biomedical Research Centre, London, ENG, United Kingdom
²Bloomsbury Institute of Intensive Care Medicine, University College London Hospitals, London, GBR
³Bloomsbury Institute of Intensive Care Medicine, UCLH, London, United Kingdom
⁴Department of Haematology, University College London Hospital, London, United Kingdom
⁵Special Coagulation Laboratory UCLH-HSL, London, United Kingdom
⁶Department of Neurology, University College Hospital, London, London, United Kingdom
⁷University College London Hospitals, London, GBR
⁸Intensive Care Unit, University College London Hospitals, London, GBR
⁹National Hospital for Neurology and Neurosurgery, UCLH, London, United Kingdom
¹⁰Department of Haematology, UCLH, London, United Kingdom
¹¹Bloomsbury Institute of Intensive Care, University College London Hospital, London, GBR
¹²Department of Haematology, University College London Hospitals, Cardiometabolic Program, National Institute for Health Research UCLH/UCL Biomedical Research Centre, London, United Kingdom

Angela C. Weyand, MD¹, Kenneth D Friedman, MD², Sweta Gupta, MD^3*, Kristina M. Haley, DO⁴, Chunla He^5*, Salley Pels, MD⁶, Jonathan C. Roberts, MD⁷, Crystal Watson, BS^8* and Robert F. Sidonio Jr., MD, MSc.⁹

¹University of Michigan, Ann Arbor, MI
²Blood Research Institute, Versiti, Milwaukee, WI
³Indiana Hemophilia & Thrombosis Center, Indianapolis, IN
⁴The Hemophilia Center, Oregon Health Science University, Portland, OR
⁵American Thrombosis and Hemostasis Network, Rochester, NY
⁶Department of Pediatrics, The Warren Alpert Medical School of Brown University, Providence, RI
⁷The Medical College of Wisconsin Inc., Peoria, IL
⁸American Thrombosis and Hemostasis Network, Decatur, GA
⁹Emory University, Aflac Cancer and Blood Disorders, ATLANTA, GA

Jill M Johnsen, MD^1,2, Sarah Ruuska, MPH^1*, Peter A. Kouides, MD³ and Barbara A. Konkle, MD^1,2

¹Bloodworks Research Institute, Seattle, WA
²Department of Medicine, University of Washington, Seattle, WA
³Department of Medicine, Mary M. Gooley Hemophilia Center, Rochester, NY

Vincent P. Diego, Ph.D.^1,2*, Bernadette W. Luu, B.S., M.T. (CLS)^1,2,3*, Hari Movva, B.S.^2*, Marco Hofmann, M.S.^4*, Marcio A. Almeida, Ph.D.^1,2*, Long V. Dinh, Ph.D.^3*, Jerry S. Powell, M.D.^3,5, Raja Rajalingam, Ph.D.^6*, Juan M Peralta, M.S.^1,2*, Henry Mead, MBA, MPH^7*, Satish Kumar, Ph.D.^1,2*, Joanne E. Curran, Ph.D.^1,2*, Miguel A. Escobar, M.D.^8*, Sarah Williams-Blangero, Ph.D.^1,2*, Eugene Maraskovsky, Ph.D.^9*, John Blangero, Ph.D.^1,2* and Tom E. Howard, M.D., Ph.D.^1,2,3,10*

¹South Texas Diabetes and Obesity Institute, The University of Texas Rio Grande Valley, Brownsville, TX
²Department of Human Genetics, School of Medicine, The University of Texas Rio Grande Valley, Brownsville, TX
³Haplogenics, Brownsville, TX
⁴Research, CSL Behring GmbH, Marburg, Germany
⁵Division of Hematology and Oncology, Department of Internal Medicine, School of Medicine, The University of California at Davis, Davis, CA
⁶Immunogenetics and Transplantation Laboratory, Department of Surgery, School of Medicine, The University of California at San Francisco, San Francisco, CA
⁷CSL Behring, King of Prussia, PA
⁸University of Texas Health Science Center, Houston, TX
⁹Research, Bio21 Institute, CSL Limited, Melbourne, Australia
¹⁰Department of Pathology and Laboratory Medicine, VA Valley Coastal Bend Healthcare System, Harlingen, TX

Miguel A. Escobar, M.D.^1*, Amy Dunn², Doris Quon, MD, PhD³, Ben Trzaskoma^4*, Lucy Lee^4*, Richard H Ko⁴ and Shannon L Carpenter, MD⁵

¹University of Texas Health Science Center, Houston, TX
²Nationwide Children's Hospital, Columbus, OH
³Los Angeles Orthopedic Hemophilia Treatment Center, Orthopedic Institute for Children, Los Angeles, CA
⁴Genentech, Inc., South San Francisco, CA
⁵Children's Mercy Hospital, Kansas City, MO

Kelly A Bush, MD^1,2*, Hilda Ding, MD, MS^1,2*, Kate O’Flaherty-Keese, RN, MSN, CPNP^1*, Rosalie Brooks, RN^1*, Katharine Farrow, RN, BSN^1*, Jovanee Recendez, MPH^1*, Jacqueline Limjoco, RN^1* and Courtney D Thornburg, MD^1,2

¹Hemophilia and Thrombosis Treatment Center, Rady Children’s Hospital San Diego, San Diego, CA
²Department of Pediatrics, Division of Hematology/Oncology, UC San Diego School of Medicine, La Jolla, CA

Andrew Johnsrud, MD¹, Juliana Craig, BA^1*, John H. Baird, MD^1,2, Jay Y. Spiegel, MD, FRCPC¹, Lori Muffly, MD, MS³, James Zehnder, MD⁴, Robert S. Negrin, MD¹, Laura J. Johnston, MD¹, Sally Arai, MD, MS¹, Judith A Shizuru, MD, PhD¹, Robert Lowsky, MD, FRCP¹, Everett H Meyer, MD, PhD^1*, Wen-Kai Weng, MD, PhD¹, Parveen Shiraz, MD¹, Andrew R. Rezvani, MD¹, Theresa Latchford, RN^1*, Crystal L. Mackall, MD⁵, David B. Miklos, MD, PhD¹, Matthew J. Frank, MD, PhD¹ and Surbhi Sidana, MD⁶

¹Division of Blood and Marrow Transplantation, Stanford University, Stanford, CA
²Center for Cancer Cell Therapy, Stanford University School of Medicine, Stanford, CA
³Division of Blood and Marrow Transplantation, Department of Medicine, Stanford University, Stanford, CA
⁴Department of Pathology, Stanford University, Palo Alto, CA
⁵Department of Pediatrics, Division of Hematology and Oncology, Stanford University, Stanford, CA
⁶Division of Blood and Marrow Transplantation, Stanford University School of Medicine, Stanford, CA

Satyaprakash Nayak, PhD^1*, Sangeeta Raje, PhD^2*, John Teeter, MD^3*, Lutz Harnisch, MD^4* and Steven Arkin, MD¹

¹Pfizer Inc., Cambridge, MA
²Pfizer Inc., Collegeville, PA
³Pfizer Inc., Groton, CT
⁴Pfizer Inc., London, United Kingdom

Miguel A. Escobar, M.D.^1*, Doris Quon, MD, PhD², Cindy A. Leissinger, MD³, James V Luck Jr., MD^4*, Ahmad Al-Sabbagh, MD^5*, Daniel Bonzo, PhD^5*, Ian S Mitchell, PhD^6*, W Allan Alexander, MD^7* and Cedric Hermans, MD, MRCP, PhD⁸

¹University of Texas Health Science Center, Houston, TX
²Orthopaedic Institute for Children, Los Angeles, CA
³Tulane University, New Orleans, LA
⁴Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
⁵LFB, Framingham, MA
⁶GLOVAL, LLC, Lafayette, CO
⁷HEMA Biologics, Louisville, KY
⁸Cliniques Univ. Saint-Luc, Brussels, Belgium

Anshul Vagrecha, MBBS^1*, Joseph Stanco, RN CRA^1*, Daphenee Ulus, FNP^1* and Suchitra Acharya, MD²

¹Pediatric Hematology, Oncology and Cellular Therapy, Cohen Children's Medical Center, New Hyde Park, NY
²Pediatric Hematology, Oncology and Cellular Therapy, Cohen Children's Medical Center of New York, New Hyde Park, NY

Timothy Clifford Carll, MD^1* and Geoffrey D. Wool, MD, PhD²

¹University of Chicago Medicine, Chicago, IL
²Department of Pathology, University of Chicago, Chicago, IL

Adrienne Lee, MD¹, Federico Germini, MD, MSc^2*, Man-Chiu Poon, MD³, Arun Keepanasseril^2*, Quazi Ibrahim, MSc^2*, Aijing Shang, MD, PhD^4*, Francis Nissen^4*, Oliver Meier^4*, Cami S. Sima, MD^5*, Davide Matino^2* and Alfonso Iorio, MD, PhD^6,7

¹Department of Medicine, Cumming School of Medicine University of Calgary, Calgary, AB, Canada
²Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada
³University of Calgary Cumming School of Medicine, Alberta, Canada
⁴F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁵Genentech, Inc., South San Francisco, CA
⁶Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada
⁷McMaster-Bayer Endowed Research Chair in Clinical Epidemiology of Congenital bleeding Disorders, Department of Medicine, McMaster University, Hamilton, Canada

Robert F. Sidonio Jr., MD, MSc.¹, Sarah A Hale, MS, PhD^2*, Imrran Halari^3* and Ayesha Zia⁴

¹Emory University & Children's Healthcare of Atlanta, Atlanta, GA
²Bleeding Disorders, Takeda Pharmaceutical Company Limited, Lexington, MA
³Charles River Associates, Boston, MA
⁴University of Southwestern Medical Center, Dallas, TX

Linda Neuman, MD, Shraddha Desai, PhD, MS^*, Tom Knudsen, PhD, DVM^*, Frank Del Greco, MBA^* and Howard Levy, PhD, MBBChir^*

Catalyst Biosciences, South San Francisco, CA

Stephanie Valer Seremetis, MD¹, Katarina Cepo, MD^2*, Josephine Skovgaard Rasmussen, DVM^3*, Trine Høyer Rose, PhD^4*, Søren Tamer, MSc^3*, Thomas Porstmann, PhD^5* and Jesper Haaning, PhD^3*

¹Novo Nordisk A/S, Plainsboro, NJ
²Biopharm Medical & Science, Novo Nordisk A/S, Søborg, Denmark
³Novo Nordisk A/S, Søborg, Denmark
⁴Quantitative Clinical Pharmacology, Novo Nordisk A/S, Søborg, Denmark
⁵Novo Nordisk Health Care AG, Zurich, Switzerland

Sanjay Ahuja, MD¹, Kathelijn Fischer^2*, Tina Biss^3*, Monika Maas Enriquez^4*, Maria-Elisa Mancuso^5*, MacGregor Steele, MD, FRCPC⁶, Maria Wang^7* and Gili Kenet⁸

¹Case Western Reserve University, Cleveland, OH
²Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
³Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
⁴Bayer, Wuppertal, Germany
⁵Center for Thrombosis and Hemorrhagic Diseases, Humanitas Clinical and Research Center – IRCCS, Rozzano, Milan, Italy
⁶Cumming School of Medicine, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada
⁷Bayer, Whippany, NJ
⁸Israel National Hemophilia Center and Thrombosis Institute, Chaim Sheba Medical Center, Tel Hashomer, Israel

Satish Maharaj, MBBS, MD¹, Simone Chang, MBBS¹, Karan Seegobin, MBBS², Marwan Shaikh, MD² and Vivek R. Sharma, MD¹

¹University of Louisville, Louisville, KY
²Mayo Clinic, Jacksonville, FL

Charles Nakar, MD^1*, Neelam Thukral, CCRC^1*, Heather L. McDaniel, MD², Joseph M Parker, MD^3*, Diane Trybul, BS^3* and Amy D Shapiro, MD¹

¹Indiana Hemophilia & Thrombosis Center, Indianapolis, IN
²Vanderbilt University, Nashville, TN
³Prometic Biotherapeutics, Rockville, MD

Michael U. Callaghan, MD¹, Claude Négrier², Ido Paz-Priel, MD³, Tiffany Chang³, Sammy Chebon^4*, Michaela Lehle^4*, Johnny Mahlangu⁵, Guy Young, MD⁶, Rebecca Kruse-Jarres, MD⁷, Maria Elisa Mancuso^8*, Markus Niggli^4*, Peter Kuebler^3*, Nives Selak Bienz^4*, Midori Shima, MD⁹, Victor Jimenez-Yuste, Medical Degree. PhD^10*, Christophe Schmitt^4*, Elina Asikanius^4*, Gallia Levy^3*, Steven W. Pipe, MD¹¹ and Johannes Oldenburg¹²

¹Children's Hospital of Michigan, Detroit, MI
²Edouard Herriot University Hospital, Lyon, France
³Genentech, Inc., South San Francisco, CA
⁴F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁵University of the Witwatersrand and NHLS, Johannesburg, South Africa
⁶University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA
⁷BloodWorks NorthWest, Seattle, WA
⁸Angelo Bianchi Bonomi Haemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy
⁹Nara Medical University Hospital, Kashihara, Japan
¹⁰Hospital Universitario La Paz, Autonoma University, Madrid, Spain
¹¹University of Michigan, Ann Arbor, MI
¹²University of Bonn, Bonn, Germany