Lev Gorfinkel, MD^1,2, Erica B. Esrick, MD³, Alexander Lake^1,2*, Miki Nishitani, MD, BS^1,4, Malika Kapadia^1,2*, Amy Federico^5*, Colleen Dansereau, MS^2*, David A. Williams, MD⁶ and Leslie E. Lehmann²

¹Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA
²Dana-Farber Cancer Institute, Boston, MA
³Dana–Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
⁴Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
⁵Boston Children's Hospital, Boston, MA
⁶Hematology/Oncology, Boston Children's Hospital, Boston, MA

Laurent Frenzel, MD, PhD^1*, Kaan Kavakli^2*, Andrew D. Leavitt, MD³, Ali Bülent Antmen, MD, PhD^4*, Davide Matino, MD, MSc^5,6*, Margareth Ozelo, MD⁷, Hazza Alzahrani, MD^8*, Barbara A. Konkle, MD⁹, Maria de los Angeles Resa, PhD^10*, Annie F. Fang, MD, PhD^10*, Jasmine Healy, PhD^11*, Delphine Agathon, PhD^12*, Francesca Biondo, MD^13* and Frank Plonski, MA, RN, BS^14*

¹Department of Haematology, Institut Necker, Paris, France
²Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
³Department of Medicine and Department of Laboratory Medicine, University of California San Francisco, San Francisco, CA
⁴Department of Pediatric Hematology, Acibadem Adana Hospital, Adana, Turkey
⁵McMaster University, Hamilton, ON, Canada
⁶Thrombosis and Atherosclerosis Research Institute (TaARI), Hamilton, ON, Canada
⁷Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
⁸Adult Hematology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁹Washington Center for Bleeding Disorders and the University of Washington, Seattle, WA
¹⁰Pfizer Inc, New York, NY
¹¹Pfizer Canada ULC, Kirkland, QC, Canada
¹²Pfizer Inc, Paris, France
¹³Pfizer S.r.l., Rome, Italy
¹⁴Pfizer Inc, Collegeville, PA

Jennifer J. Jaroscak, MD¹, Julie Kanter, MD², Robert I. Liem, MD³, Anjulika Chawla, MD, FAAP⁴, Melissa A. Kinney^4*, Ankit Lodaya, MS^4*, Lin Pan^4*, Emily Sheldon-Waniga, PhD^4*, Francis J. Pierciey Jr., MSc^4* and Alexis Thompson, MD, MPH^5,6

¹Medical University of South Carolina, Charleston, SC
²University of Alabama at Birmingham, Birmingham, AL
³Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁴bluebird bio, Inc., Somerville, MA
⁵Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁶Children's Hospital of Philadelphia, Philadelphia, PA

Ben Samelson-Jones, MD, PhD^1,2, Laurent Frenzel, MD, PhD^3*, Kaan Kavakli^4*, Adam Cuker, MD⁵, Jerome M. Teitel, MD⁶, Pengling Sun^7*, Lisa J Wilcox, PhD^8*, Francesca Biondo, MD^9*, Benjamin Hutter, MBBS, BSc^10*, John McKay, MS^11*, Delphine Agathon, PhD^12* and Frank Plonski, MA, RN, BS^13*

¹Children's Hospital of Philadelphia, Philadelphia, PA
²University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA
³Department of Haematology, Institut Necker, Paris, France
⁴Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
⁵Department of Medicine and Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁶Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada
⁷Pfizer Inc, Cambridge, MA
⁸Senior Director – Global Medical Affairs at Pfizer, Pfizer, Toronto, ON, Canada
⁹Pfizer S.r.l., Rome, Italy
¹⁰Pfizer Inc, London, United Kingdom
¹¹Pfizer Inc, Pearl River, NY
¹²Pfizer Inc, Paris, France
¹³Pfizer Inc, Collegeville, PA

Frank W.G. Leebeek^1,2, Karina Meijer, MD, PhD^3*, Michiel Coppens, MD, PhD^4*, Peter Kampmann, MD^5*, Dr. Klamroth^6*, Paul van der Valk^7*, Paul E. Monahan^8*, Karen Pinachyan^8*, Sandra LeQuellec^9* and Wolfgang Miesbach^10*

¹Dept. Hematology, Erasmus University Medical Center, Rotterdam, Netherlands
²Hematology, Erasmus university Medical Center, Rotterdam, Netherlands
³Division of Thrombosis and Haemostasis, Department of Haematology, University Medical Center Groningen, University of Groningen, Groningen, Netherlands
⁴Amsterdam University Medical Centers, Department of Vascular Medicine, University of Amsterdam and Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
⁵Rigshospitalet, Copenhagen, Denmark
⁶Vivantes Klinikum im Friedrichshain, Berlin, Berlin, Germany
⁷University Medical Centre Utrecht and University Utrecht, University of Utrecht, Utrecht, Netherlands
⁸CSL Behring, King of Prussia
⁹CSL Behring, King of Prussia, PA
¹⁰Goethe University Hospital, Coagulation and Haemophilia Centre, Medical Clinic 2, Frankfurt Am Main, Germany

Yani B Suber^1*, Bhavya S Doshi, MD¹, Robert A French^1*, Elizabeth Merricks^2*, Timothy Nichols, MD² and Ben Samelson-Jones, MD, PhD³

¹The Children's Hospital of Philadelphia, Philadelphia, PA
²University of North Carolina At Chapel Hill, Chapel Hill, NC
³Children's Hospital of Philadelphia, Philadelphia, PA

Rebecca Kruse-Jarres¹, Bruce Ewenstein^2*, Paul E. Monahan^2*, Nicholas Galante^2*, Nathalie W.D. Jansen^2*, Sean Gill^2* and Niamh O'Connell^3*

¹Washington Center for Bleeding Disorders, University of Washington, Seattle, WA
²CSL Behring, King of Prussia, PA
³National Coagulation Centre, St James’s Hospital, Dublin, Ireland

Debra D Pittman, MS¹, Swapnil Rakhe, MS^1*, Lisa J Wilcox, PhD^2*, Daniel LeBlanc, BS^1*, Vicente Pagan, BS^1*, Megan L Brophy, PhD^1*, Diana Sapashnik, MS^1* and Annette Sievers, PhD^1*

¹Pfizer Inc, Cambridge, MA
²Senior Director – Global Medical Affairs at Pfizer, Pfizer, Toronto, ON, Canada

Feng Xue, MD^1*, Mankai Ju^2*, Tienan Zhu, MD, PhD^3*, ZePing Zhou, MD, PhD⁴, Jing Sun^5*, Linhua Yang^6*, Zhenyu Yan^7*, Hu Zhou⁸, Xin Du^9*, Changcheng Zheng, MD^10*, Xia Wu^11*, Xiao Xiao^12*, Renchi Yang, MD^13,14 and Lei Zhang¹³

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China, Tianjin, China
²institute of haematology and blood diseases hospital, Chinese academy of medical sciences and peking union medical college, Tianjin, CHN
³Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, CHN
⁴Second Affiliated Hospital of Kunming Medical University, Kunming, China
⁵Nanfang Hospital Southern Medical University, Guangzhou, China
⁶The Second Hospital of Shanxi Medical University, Taiyuan, China
⁷North China University of Science and Technology Affiliated Hospital, Tangshan, China
⁸Department of Hematology, Henan Provincial Cancer Hospital, Zhengzhou, China
⁹Department of Hematology, The Second People's Hospital of Shenzhen, The First Affiliated Hospital of Shenzhen University, Shenzhen, China
¹⁰Anhui provincial hospital, Hefei, China
¹¹East China University of Science and Technology, shanghai, China
¹²Belief BioMed, shanghai, China
¹³Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
¹⁴Institute of Hematology & Blood Disease, Tianjin, China

Wei Liu^1*, Xiaolei Pei^1*, Bingqi Xu, phD^1*, Xinyue Dai, MD^1*, Feng Xue, MD^1*, Linging Chen^1*, Yuhua Wang, MD^1*, Yunfei Chen, MD^1*, Xiaofan Liu, MD^1*, Rongfeng Fu, MD^1*, Wentian Wang, PhD^2*, Ying Chi^2*, Huiyuan Li^1*, Renchi Yang, MD¹ and Lei Zhang²

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China

Ami J Shah, MD^1,2,3, José Luis López Lorenzo, MD^4,5*, Rachael F Grace, MD⁶, E J van Beers, MD, PhD⁷, M Bartels, MD, PhD^7*, Andreas Glenthoej, MD, PhD⁸, Susana Navarro, PhD^5,9,10*, Lucia Llanos, MD, PhD^4,5*, Begoña Pérez de Camino Gaisse, MD^4,5*, Sol Sanchez, MD^4,5*, Josune Zubicaray, MD, PhD^11*, May Chien, MD^2,3*, Oscar Quintana Bustamante, PhD^5,9,10*, M González-Vicent, MD, PhD^9,11*, June Iriondo, MD^9,11*, Miriam Zeini, PhD^12*, Grace Choi, BS^12*, Eileen Nicoletti, MD^12*, Maria Chitty-Lopez, MD^12*, Gayatri R Rao, MD, JD^12*, Juan Bueren, PhD^5,9,10*, Jonathan D Schwartz, MD¹², Jose Carlos Segovia, PhD^5,9,10* and Julián Sevilla, MD, PhD^9,11*

¹Center for Definitive and Curative Medicine, Stanford University, Stanford, CA
²Lucile Packard Children’s Hospital, Palo Alto, CA
³Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
⁴Hospital Universitario Fundación Jiménez Díaz, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
⁵Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
⁶Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA
⁷University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
⁸Danish Red Blood Cell Center, Department of Hematology, Copenhagen University Hospital – Rigshospitalet, Copenhagen, Denmark
⁹Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
¹⁰Unidad de Innovación Biomédica, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain
¹¹Hematología y Hemoterapia, Fundación para la Investigación Biomédica, Hosp Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
¹²Rocket Pharmaceuticals, Inc., Cranbury, NJ