Franco Locatelli, Prof., MD, PhD¹, Peter Lang, Dr.^2*, Amanda Li, MD^3*, Selim Corbacioglu, MD⁴, Josu de la Fuente, PhD⁵, Donna A. Wall⁶, Robert Liem, MD⁷, Roland Meisel, MD⁸, Markus Y. Mapara, MD, PhD⁹, Ami J Shah, MD¹⁰, Maria Domenica Domenica Cappellini, MD¹¹, Antonis Kattamis, MD¹², Sujit Sheth, MD¹³, Yael Bobruff, PhD^14*, Laura Bower, MD^14*, Lanju Zhang, PhD^14*, Anjali Sharma, MD^15*, Yang Song, PhD^14*, William Hobbs, MD, PhD^14* and Haydar Frangoul, MD¹⁶

¹Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy
²University of Tübingen, Tuebingen, Germany
³BC Children’s Hospital, University of British Columbia, Vancouver, BC, Canada
⁴University of Regensburg, Regensburg, Germany
⁵Imperial College Healthcare NHS Trust, St Mary's Hospital, London, United Kingdom
⁶The Hospital for Sick Children, Toronto, ON, Canada
⁷Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁸Heinrich-Heine-University, Duesseldorf, Germany
⁹Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY
¹⁰Stanford University School of Medicine, Stanford, CA
¹¹University of Milan, Milan, Italy
¹²National and Kapodistrian University of Athens, Athens, Greece
¹³Joan and Sanford I Weill Medical College of Cornell University, New York, NY
¹⁴Vertex Pharmaceuticals Incorporated, Boston, MA
¹⁵CRISPR Therapeutics, Cambridge, MA
¹⁶Sarah Cannon Center Research Institute and The Children’s Hospital at TriStar Centennial, Nashville, TN

Ami J Shah, MD^1,2,3, José Luis López Lorenzo, MD^4,5*, Julián Sevilla, MD, PhD^6,7*, Susana Navarro, PhD^5,7,8*, Lucía Llanos, MD, PhD^4,5*, Begoña Pérez de Camino Gaisse, MD^4,5*, Sol Sanchez, MD^4,5*, Josune Zubicaray, PhD^6*, Bert Glader, MD, PhD^1,3, May Chien, MD^1,3, Oscar Quintana Bustamante, PhD^5,7,8*, Miriam Zeini, PhD^9*, Grace Choi^9*, Eileen Nicoletti, MD^9*, Gayatri R. Rao, MD, JD^9*, Maria Grazia Roncarolo, MD, PhD^1,2,10, Juan A. Bueren, PhD^5,7,8*, Jonathan D. Schwartz, MD⁹ and José C Segovia, PhD^5,7,8*

¹Lucile Packard Children’s Hospital, Palo Alto, CA
²Center for Definitive and Curative Medicine, Stanford University, Stanford, CA
³Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University, Stanford, CA
⁴Instituto de Investigación Sanitaria Fundación Jiménez Díaz, Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
⁵Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
⁶Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
⁷Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
⁸Unidad de Innovación Biomédica, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain
⁹Rocket Pharmaceuticals, Inc., Cranbury, NJ
¹⁰Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University, Stanford, CA

Steven W. Pipe, MD¹, Frank W.G. Leebeek², Michael Recht, MD, PhD, MBA³, Nigel S. Key⁴, Susan Lattimore^3*, Giancarlo Castaman^5*, David Cooper^6,7*, Stephanie Verweij^6,7*, Ricardo Dolmetsch^6,7*, Jacqueline Tarrant^8*, Yanyan Li^8*, Paul E. Monahan^8* and Wolfgang A. Miesbach^9*

¹University of Michigan, Ann Arbor, MI
²Erasmus Medical Center, University Medical Center, Rotterdam, Netherlands
³Oregon Health & Science University, Portland, OR
⁴University of North Carolina, Chapel Hill, NC
⁵Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
⁶uniQure BV, Amsterdam, Netherlands
⁷uniQure Inc., Lexington, MA
⁸CSL Behring, King of Prussia, PA
⁹University Hospital Frankfurt, Frankfurt, Germany

Asif Alavi, MD¹, Mehrdad Abedi, MD², Suhag Parikh, MD^3*, Richard Boismenu, PhD^4*, Michael Chen, MSc^4*, Ben L Hsu^4*, Bettina M. Cockroft, MD, MBA^4*, Isobelle Galeon, MD, MS, MPH^5*, Pablo Rendo, MD⁵ and Mark C. Walters, MD⁶

¹Karmanos Cancer Institute, Wayne State University, Detroit, MI
²Department of Internal Medicine, Division of Cellular Therapy, Bone Marrow Transplantation and Malignant Hematology, University of California Davis School of Medicine, Sacramento, CA
³Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
⁴Sangamo Therapeutics, Inc., Brisbane, CA
⁵Sanofi, Cambridge, MA
⁶UCSF Benioff Children's Hospital Oakland, Oakland, CA

Steven W. Pipe, MD¹, Frank W.G. Leebeek², Michael Recht, MD, PhD, MBA³, Nigel S. Key⁴, Susan Lattimore^3*, Giancarlo Castaman^5*, Michiel Coppens^6,7*, David Cooper^8,9*, Robert Gut^8,9*, Sergio Slawka^8,9*, Stephanie Verweij^8,9*, Ricardo Dolmetsch^8,9*, Yanyan Li^10*, Paul E. Monahan^10* and Wolfgang A. Miesbach^11*

¹University of Michigan, Ann Arbor, MI
²Erasmus Medical Center, University Medical Center, Rotterdam, Netherlands
³Oregon Health & Science University, Portland, OR
⁴University of North Carolina, Chapel Hill, NC
⁵Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
⁶Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
⁷Amsterdam UMC location, University of Amsterdam, Vascular Medicine, Amsterdam, Netherlands
⁸uniQure BV, Amsterdam, Netherlands
⁹uniQure Inc., Lexington, MA
¹⁰CSL Behring, King of Prussia, PA
¹¹University Hospital Frankfurt, Frankfurt, Germany

Wolfgang A. Miesbach^1*, Michael Recht, MD, PhD, MBA², Nigel S. Key³, Krupa Sivamurthy^4*, Paul E. Monahan^4* and Steven W. Pipe, MD⁵

¹University Hospital Frankfurt, Frankfurt, Germany
²Oregon Health & Science University, Portland, OR
³University of North Carolina, Chapel Hill, NC
⁴CSL Behring, King of Prussia, PA
⁵University of Michigan, Ann Arbor, MI

Rongrong Liu, PhD, MD^1*, Hui Xu, PhD^2*, Junbin Liang, PhD^2*, Wenqiang Xie^1*, Gaohui Yang, PhD, MD^1*, Lei Shi, MD^2*, Lingling Shi, PhD, MD^1*, Jianpei Fang, MD^3*, Chunfu Li, MD⁴, Zeyan Shi, MD^1*, Xuemei Zhou^1*, Junjiu Huang, PhD^5* and Yongrong Lai, PhD, MD^1*

¹Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
²Reforgene Medicine, Guangzhou, China
³Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou, China
⁴Nanfang-Chunfu Children's Institute of Hematology & Oncology, Taixin Hospital, Dongguan, China
⁵School of Life Sciences, Sun Yat-sen University, Guangzhou, China

Socheatraksmey Ung, PharmB^1,2*, Pongsakorn Choochuen, MD^1,3*, Wannakorn Khopanlert, MD^1,2*, Kajornkiat Maneechai, BSc^1,2*, Surasak Sangkhathat, MD, PhD^3*, Seitaro Terakura, MD, PhD^4* and Jakrawadee Julamanee, MD, PhD^2*

¹Department of Biomedical Sciences and Biomedical Engineering, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
²Stem Cell Laboratory, Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
³Translational Medicine Research Center, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
⁴Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan

Lucas Van Gorder, BS^1,2,3*, Bhavya S Doshi, MD^2,3,4, Elinor Willis, VMD^5*, Matthew Lanza, VMD^6*, Valder R Arruda, MD, PhD^1,2,3, Mary Beth Callan, VMD^7* and Ben J Samelson-Jones, MD, PhD^1,2,3

¹Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
³Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Department of Pediatrics, University of Pennsylvania, Philadelphia, PA
⁵Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA
⁶Department of Pathobiology,, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA
⁷Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA

Haokun Yuan, MSc^1*, Hongwei Liu, MSc^1*, Xiaomei Wu, BSc^1*, Yuchen Liu, MSc^2*, Rui Zhang, MSc^2* and Lung-Ji Chang, PhD^1,2

¹School of Medicine, University of Electronic Science and Technology of China, Chengdu, China
²Geno-Immune Medical Institute, Shenzhen, China

Omar Banda, PhD¹, Mohamad-Gabriel Alameh, PhD^2*, Seul Jung, BS^3*, Drew Weissman, MD, PhD^4*, Stefano Rivella, PhD⁵ and Peter Kurre, MD⁶

¹Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Medicine, University of Pennsylvania, Philadelphia, PA
³Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁴University of Pennsylvania Medical Center, PHILADELPHIA, PA
⁵Department of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
⁶Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Oregon Health & Science University (OHSU), Philadelphia, PA

Wei Liu^1*, Feng Xue, MD^1*, Ting Sun, MD^1*, Bingqi Xu, PhD^1*, Xiaolei Pei, PhD^1*, Tenghui Yu, PhD^2*, Fenglan Wu, PhD^2*, Lijun Shen^2*, Renchi Yang, MD¹ and Lei Zhang, MD^1*

¹State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
²Gritgen Therapeutics, Suzhou, China, Suzhou, China

Diana M. Abraham, BA^1*, Yifan Zhou^1,2,3*, Richard Lozano, BS^1*, Gregory A. Newby, PhD^4,5,6*, David R. Liu, PhD^5,6,7*, Chuanfeng Wu, MD, PhD^1* and Cynthia E. Dunbar, MD¹

¹Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Wellcome Trust Sanger Institute, University of Cambridge, Cambridge, United Kingdom
³Wellcome-MRC Cambridge Stem Cell Institute, University of Cambridge, Cambridge, United Kingdom
⁴Broad Institute of Harvard and MIT, Harvard University, Cambridge, MA
⁵Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA
⁶Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA
⁷Howard Hughes Medical Institute, Harvard University, Cambridge, MA

Sandeep RP Kumar, PhD^1*, Moanaro Biswas, PhD², Ype P De Jong, MD^3* and Roland W Herzog, PhD¹

¹Department of Pediatrics, IUPUI, Indianapolis, IN
²Pediatrics, Indiana University School of Medicine, Indianapolis, IN
³Division of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY

Lucila Kerbauy, MD, PhD¹, Larissa Leggieri Coa^1*, Micheli Severo Sielski^2*, Juliana Aparecida Preto Godoy, PhD^1*, Andrea Tiemi Kondo, MD^3*, Julia Teixeira Cottas Azevedo^2*, Augusto Barbosa Jr.^2*, Oswaldo Keith Okamoto, PhD^4*, Jose Mauro Kutner, MD, PhD⁵, Nelson Hamerschlak, MD, PhD⁶ and Raquel de Melo Alves Paiva, PhD^7*

¹Hospital Israelita Albert Einstein, Sao Paulo, Brazil
²Cell Therapy Department, Hospital Israelita Albert Einstein, Sao Paulo, Brazil
³Hospital Israelita Albert Einstein, Sao Paulo, SE, Brazil
⁴Human Genome and Stem Cell Research Center, Department of Genetics and Evolutionary Biology, Biosciences Institute, Universidade de Sao Paulo, Sao Paulo, Brazil
⁵Hospital Israelita Albert Einstein, Sao Paulo, BRA
⁶Hospital Israelita Albert Einstein, Sao Paolo, Brazil
⁷Hospital Israelita Albert Einstein, São Paulo, AL, Brazil