Ashish O. Gupta, MBBS, MPH¹, Paul J. Orchard, MD^2*, Stuti Thakker^3*, Wilaiwan Durose^4*, Carina King^3* and Troy C. Lund, MD, PhD, MS²

¹Pediatric Blood and Marrow Transplant, University of Minnesota, Minneapolis, MN
²Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN
³University of Minnesota, Minneapolis
⁴University of Minnesota, Minneapolis, MN

Sung-Yun Pai, MD¹, Nisha Nagarsheth, PhD^2*, Susan Prockop, MD³, Myriam Armant, PhD^4*, Donald B. Kohn, MD⁵, Rebecca A. Marsh, MD^6*, Claire Booth, MBBS, PhD, MSc^7*, John K. Everett, PhD^8*, Jack Bleesing, MD, PhD^9*, Deepak Chellapandian, MD^10*, Colleen Dansereau, MSN, RN, CPN^11*, Satiro de Oliveira, MD^12*, Wendy B. London, PhD¹³, M. Angélica Marinovic, MD^14*, Theodore B. Moore, MD¹⁵, Matias Oleastro^16*, Grainne O'Toole^7*, Mark Vander Lugt, MD^17*, Luciano Urdinez, MD^18*, Kelly J. Walkovich, MD¹⁹, Jolan E. Walter, MD, PhD^20*, Axel Schambach, MD, PhD^21*, Adrian J. Thrasher, MBBS, PhD, FMedSci^22*, Frederic D. Bushman, PhD^8* and David A Williams, MD¹³

¹National Cancer Institute, Center for Cancer Research, NIH, Bethesda, MD
²National Cancer Institute, National Institutes of Health, Bethesda, MD
³Boston Children's Hospital/Dana Farber Cancer Institute, Boston, MA
⁴Boston Children's Hospital, Boston, MA
⁵Broad Institute of Harvard and MIT, University of California - Los Angeles, Los Angeles, CA
⁶Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁷Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom
⁸Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁹Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center; Cancer and Blood Diseases Institute, Cincinnati, OH
¹⁰Johns Hopkins All Children's Hospital, St Petersburg, FL
¹¹Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
¹²Division of Hematology/Oncology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA
¹³Boston Children's Hospital, Harvard Medical School, Boston, MA
¹⁴Clinica Santa Maria/Unidad de Inmunologia y Alergias, Santiago, Chile
¹⁵UCLA Division of Pediatric Hematology, University of California, Los Angeles, Los Angeles, CA
¹⁶Hospital Nacional de Pediatria Garrahan, Buenos Aires, BA, Argentina
¹⁷Division of Pediatric Hematology/Oncology; Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, MI
¹⁸Hospital Nacional de Pediatra Garrahan, Buenos Aires, Argentina
¹⁹Division of Hematology/Oncology, Department of Pediatrics, University of Michigan, Ann Arbor, MI
²⁰Division of Allergy and Immunology, Departments of Pediatrics, Johns Hopkins All Children's Hospital, St. Petersburg, FL
²¹Experimental Hematology, Hannover Medical School, Hannover, Germany
²²UCL Great Ormond Street Institute of Child Health, London, United Kingdom

Agnieszka Czechowicz, MD, PhD^1,2,3, Julián Sevilla, MD, PhD^4,5*, Claire Booth, MBBS, PhD, MSc^6*, Rajni Agarwal, MD^1,2,3, Josune Zubicaray, PhD^4*, Paula Río, PhD^5,7,8*, Susana Navarro, PhD^5,7,8*, Kritika Chetty, MBBS^6*, Grainne O'Toole^6*, Jinhua Xu-Bayford^6*, Philip Ancliff, MA, MRCP, MRCPath^6*, Elena Sebastián, MD^4*, Grace Choi^9*, Miriam Zeini, PhD^9*, Eileen Nicoletti, MD^9*, John E Wagner, MD¹⁰, Gayatri R. Rao, MD, JD^9*, Adrian J. Thrasher, MBBS, PhD, FMedSci^6*, Jonathan D. Schwartz, MD⁹, Maria Grazia Roncarolo, MD, PhD^1,2,3 and Juan A. Bueren, 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 School of Medicine, Stanford, CA
⁴Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
⁵Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
⁶UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁷Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
⁸Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain
⁹Rocket Pharmaceuticals, Inc., Cranbury, NJ
¹⁰University of Minnesota, Minneapolis, MN

Ryan Urak, PhD^1*, Stephen J Forman, MD² and Xiuli Wang, MD PhD³

¹T cell Therapeutics Research Laboratory, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA
²Department of Hematology/HCT, City of Hope National Medical Center, Duarte, CA
³Department of Hematology and Hematopoietic Cell Transplantation, City of Hope Comprehensive Cancer Center, Duarte, CA

Jie Gong, PhD^1*, Yuchen Liu, MSc^2*, Min Zhou, MD^3* and Lung-Ji Chang, PhD^2,4

¹School of Medicine, Chengdu Women's and Children's Central Hospital, University of Electronic Science and Technology of China, Chengdu, China
²Geno-Immune Medical Institute, Shenzhen, China
³Chengdu Women's and Children's Central Hospital, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China
⁴School of Medicine, University of Electronic Science and Technology of China, Chengdu, China

Jun Shi^1*, Riguo Fang, PhD^2*, Zhen Gao^1*, Zhongyu Shi, PhD^2*, Zhexiang Kuang^1*, Yongjian Zhang, PhD^2*, Lele Zhang^1*, Huihui Yang, Master^2*, Yingchi Zhang, PhD^1*, Dehui Zou, M.D., Ph.D.^1*, Liwei Fang^1*, Jiali Sun^1*, Qiang Li^1*, Yao Zou, MD^1*, Hong Pan^1*, Jingyu Zhao^1*, Liyun Li^1*, Xiao Yu^1*, Jing Xu, PhD^1*, Chun Xu^1*, Yanjie Liu^1*, Weiwei Wang^1*, Jia Shi, PhD^3*, Jie Liu, Master^3*, Jia Wang^3*, Nian Shi^3*, Lei Yang^2*, Weiqiang Wang^2*, Suyi Li^3*, Moye Shi^2*, Xiaoshuang Zhang^2*, Pengfei Yuan, PhD^2*, Wensheng Wei^4*, Dong Wei, PhD^2* and Tao Cheng¹

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 & Peking Union Medical College, Tianjin, China
²EdiGene Inc., Beijing, China
³EdiGene (Guangzhou) Inc., Guangzhou, China
⁴Biomedical Pioneering Innovation Center, Beijing Advanced Innovation Center for Genomics, Peking-Tsinghua Center for Life Sciences, Peking University Genome Editing Research Center, State Key Laboratory of Protein and Plant Gene Research, Beijing, China

Matthew S. Evans, MD¹, Witold B. Rybka, MD FRCPC¹, Stacy E. Croteau, MD², Huyen Tran, MD, PhD^3*, John E. J. Rasko, MBBS, PhD⁴, Kristen Jaworski, MSN, NP^5*, Amy MacDougall, BSN^5*, Savina Jaeger, PhD^5*, Federico Mingozzi, PhD⁵, Tiffany Chang, MD, MAS^5* and Gallia Levy, MD, PhD^5*

¹Penn State Health Milton S. Hershey Medical Center, Hershey, PA
²Boston Children's Hospital, Boston, MA
³Australian Centre for Blood Diseases, Monash University, Melbourne, Australia
⁴Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney, Camperdown, Australia
⁵Spark Therapeutics, Philadelphia, PA

Miguel Escobar, MD^1*, Barbara Konkle², Jonathan Ducore³, Kavitha Rajavel^4*, Kefeng Sun^4*, Brian Yan^4* and John Chapin⁴

¹McGovern Medical School and Gulf States Hemophilia and Thrombophilia Center, University of Texas Health Science Center, Houston Medical School, Houston, TX
²Division of Hematology, University of Washington School of Medicine, Seattle, WA
³University of California Davis Medical Center, Sacramento, CA
⁴Takeda Development Center Americas, Inc, Cambridge, MA

Tirthadipa Pradhan-Sundd, PhD¹, Tomasz W. Kaminski, PharmD², Rikesh K K. Dubey, PhD², Shweta Gudapati^3*, Omika Katoch, PhD^2*, Jesús Tejero^4*, Enrico M. Novelli, MD⁵, Margaret V. Ragni, MD, MPH⁶ and Prithu Sundd, PhD²

¹Hematology-Oncology, University of Pittsburgh, Pittsburgh, PA
²Pittsburgh Heart, Lung and Blood Vascular Medicine Institute, University of Pittsburgh School of Medicine, Pittsburgh, PA
³University of Pittsburgh, Pittsburgh, PA
⁴Division of Pulmonary, Allergy, and Critical Care Medicine, University of Pittsburgh, Pittsburgh, PA
⁵Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute, University of Pittsburgh, Pittsburgh, PA
⁶Department of Medicine, University of Pittsburgh, Pittsburgh, PA

Georgios E. Christakopoulos, MD¹, Thiyagaraj Mayuranathan^2*, Alfonso G. Fernandez, PhD^3*, Kalin Mayberry, BS^3*, Yu Yao, MD^4*, Rahul Telange, MSc, BS^3*, Michael Dudley^3*, Rachel Levine, PhD^2*, Erin Dempsey, BS^2*, David R. Liu, PhD^5,6,7*, Jonathan S Yen, PhD^2,8,9 and Mitchell J. Weiss, MD, Ph.D¹⁰

¹Department of Oncology, St Jude Children's Research Hospital, Memphis, TN
²Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
³Department of Hematology, St Jude Children's Research Hospital, Memphis, TN
⁴Department of Hematology, St Jude Children’s Research Hospital, Memphis, TN
⁵Howard Hughes Medical Institute, Harvard University, Cambridge, MA
⁶Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA
⁷Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA
⁸Therapeutic Genome Engineering, Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁹Beam Therapeutics, Cambridge, MA
¹⁰Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN

Huyen Tran, MD, PhD^1*, M. Elaine Eyster, MD², Stacy E. Croteau, MD³, Margaret V. Ragni, MD, MPH⁴, Benjamin J. Samelson‑Jones, MD, PhD^5*, Spencer Sullivan, MD⁶, John E. J. Rasko, MBBS, PhD^7,8,9, Kristen Jaworski, MSN, NP^10*, Amy MacDougall, BSN^10*, Savina Jaeger, PhD^10*, Magdalena Hofer, PhD^10*, Charlie Li, PhD^10*, Federico Mingozzi, PhD¹⁰, Tiffany Chang, MD, MAS^10* and Gallia Levy, MD, PhD^10*

¹Australian Centre for Blood Diseases, Monash University, Melbourne, VIC, Australia
²Penn State Health Milton S. Hershey Medical Center, Hershey, PA
³Boston Children’s Hospital, Boston Hemophilia Center, Boston, MA
⁴University of Pittsburgh Medical Center, Pittsburgh, PA
⁵Children’s Hospital of Philadelphia, Philadelphia, PA
⁶Mississippi Center for Advanced Medicine, Madison, MS
⁷Gene and Stem Cell Therapy Program Centenary Institute, The University of Sydney, Camperdown, NSW, Australia
⁸Faculty of Medicine and Health, The University of Sydney, Sydney, NSW, Australia
⁹Cell & Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, Australia
¹⁰Spark Therapeutics, Inc., Philadelphia, PA

Erica B. Esrick, MD^1,2, Amy Federico, CPNP^3*, Daniela Abriss^4*, Myriam Armant, PhD^4*, Kari Boardman^4*, Christian Brendel, PhD^1,5*, Marioara-Felicia Ciuculescu, MD^4*, Heather Daley^6*, Colleen Dansereau, MSN, RN, CPN^3*, Augustine Fernandes, PhD^7*, Matthew M. Heeney, MD^2,8, David G. Justus, MD^9*, Pei-Chi Kao^1*, Annette S. Kim, MD, PhD¹⁰, Donald B. Kohn, MD⁷, Leslie E. Lehmann, MD^1,2, Donghui Liu^4*, Wendy B. London, PhD^11,12, John P Manis, MD⁹, Theodore B. Moore, MD⁷, Emily Morris, MPH^3*, Danilo Pellin, PhD^13*, Ellen Proeung, PA^1*, Shanna Richard^6*, Gavin D. Roach, MD, MS⁷, Kit L. Shaw, PhD^14*, Dayna Terrazas, RN^7*, Shanna L White, MD^7* and David A. Williams, MD^1,2

¹Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
²Department of Pediatrics, Harvard Medical School, Boston, MA
³Gene Therapy Program, Boston Children's Hospital, Boston, MA
⁴TransLab, Boston Children's Hospital, Harvard Medical School, Boston, MA
⁵Harvard Stem Cell Institute, Harvard Medical School, Boston, MA
⁶Dana-Farber Cancer Institute, Cell Manipulation Core Facility, Boston, MA
⁷Division of Pediatric Hematology/Oncology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA
⁸Pediatric Hematology-Oncology, Children's Hospital - Boston, Boston, MA
⁹Department of Laboratory Medicine, Boston Children’s Hospital, Boston, MA
¹⁰Department of Pathology, Brigham and Women's Hospital, Boston, MA
¹¹Dana-Farber Boston Children’s Cancer and Blood Disorders Center, Boston, MA
¹²Harvard Medical School, Boston, MA
¹³Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
¹⁴Dana-Farber Cancer Institute, Boston, MA

Professor John Rasko, BSc (Med), MBBS (Hons), PhD^1,2,3, George Bashirians^4*, Christos Petropoulos^5*, Terri Wrin^6*, Yuvika Paliwal^7*, Suryanarayan Somanathan, PhD^4*, Michelle Casey^7*, Candida da Fonseca Pereira, PhD, MBA^8*, Ian Winburn, MBBS, PhD, MRCS^9* and Amit Chhabra, MBBS, MPH¹⁰

¹Sydney Medical School, University of Sydney, Camperdown, Australia
²Head of Department, Cell & Molecular Therapies, Royal Prince Alfred Hospital, Camperdown, Australia
³Gene and Stem Cell Therapy Program, Centenary Institute, Camperdown, Australia
⁴Pfizer Inc., Cambridge, MA
⁵Labcorp, South San Francisco, CA
⁶Labcorp, San Francisco, CA
⁷Pfizer Inc., Collegeville, PA
⁸Pfizer Australia, Sydney, Australia
⁹Pfizer Ltd, Walton on the Hill, Surrey, United Kingdom
¹⁰Pfizer Inc., New York, NY

Jing Zeng, MD^1*, My Anh Nguyen, MS^1*, Marioara-Felicia Ciuculescu, MD^2*, Esther Mintzer^3*, Pengpeng Liu, PhD^4*, Linda Yingqi Lin^1*, Tolulope O Rosanwo, BA⁵, Archana Verma^6*, Nola Neri^6*, Stacy A. Maitland^4*, Alden Richter^4*, David G. Justus, MD^7*, Kendell Clement, PhD^8*, Christian Brendel, PhD^{9,10,11,12,13*}, Luca Pinello, PhD^14*, John P Manis, MD¹⁵, Myriam Armant, PhD^16*, Scot A. Wolfe, PhD^17* and Daniel E. Bauer, MD, PhD¹⁸

¹Department of Pediatric Hematology/Oncology, Boston Children’s Hospital, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Harvard Medical School, Boston, MA
²Boston Children's Hospital, Harvard Medical School, Boston, MA
³University of Massachusetts Medical School, Worcester,
⁴University of Massachusetts Medical School, Worcester
⁵Division of Hematology/Oncology, Stem Cell Program, Boston Children's Hospital, Boston, MA
⁶Boston Children's Hospital, Harvard Medical School, Boston
⁷Department of Laboratory Medicine, Boston Children’s Hospital, Harvard Medical School, Boston, MA
⁸Massachusetts General Hospital, Harvard Medical School, Boston, MA
⁹Department of Pediatrics, Harvard Medical School, Boston, MA
¹⁰Hematology/Oncology, Harvard Medical School, Boston, MA
¹¹Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
¹²Harvard Stem Cell Institute, Harvard Medical School, Boston, MA
¹³Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
¹⁴Molecular Pathology Unit, Center for Cancer Research, Department of Pathology, Massachusetts General Hospital, Harvard Medical School, Boston, MA
¹⁵Children's Hospital Boston, Boston, MA
¹⁶Boston Children's Hospital, Boston, MA
¹⁷Department of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA
¹⁸Division of Hematology and Oncology, Boston Children's Hospital / Dana-Farber Cancer Institute, Harvard Medical School, Cambridge, MA

Lucy Y Ghoda, PhD^1*, Ebtesam Nafie, PhD^2*, Elizabeth Bloom-Saldana, PhD^3*, Patrick T. Fueger, PhD^4*, Timothy Synold, PhD^5*, James Simpson^4*, Piotr Swiderski, PhD^6*, Kokilah Muthaiyah, PhD^7*, Bin Zhang, PhD^8*, Marcin Kortylewski, PhD^9*, Steven Vonderfecht, Ph.D., DVM^10*, Le Xuan Truong Nguyen, PhD^11*, Jianying Zhang, PhD^12*, Joycelynne Palmer, PhD^12* and Guido Marcucci, MD¹³

¹Department of Hematologic Malignancies Translational Science, Gehr Family Center for Leukemia Research, Beckman Research Institute, City of Hope, Duarte, CA
²Department of Hematological Sciences Translational Research, Gehr Family Center for Leukemia Research, Beckman Research Institute, City of Hope, Duarte, CA
³City of Hope, Duarte, CA
⁴Beckman Research Institute, City of Hope, Duarte, CA
⁵Department of Cancer Biology, Beckman Research Institute, City of Hope, Duarte, CA
⁶DNA/RNA Synthesis Core Facility, Beckman Research Institute, City of Hope, Duarte, CA
⁷DNA/RNA Synthesis Facility, Beckman Research Institute, City of Hope, Duarte, CA
⁸Hematogic Malignancies Translational Science, Gehr Family Center for Leukemia Research, City of Hope Medical Center and Beckman Research Institute, Duarte, CA
⁹Department of Immuno-Oncology, Beckman Research Institute of City of Hope, City of Hope, Duarte, CA
¹⁰Animal Resource Center, City of Hope, Duarte, CA
¹¹Dept. of Hematological Malignancies Translational Medicine, Beckman Research Institute, City of Hope, Duarte, CA
¹²Department of Computational and Quantitative Medicine, Beckman Research Institute, City of Hope, Duarte, CA
¹³Department of Hematologic Malignancies Translational Science, Beckman Research Institute, City of Hope, Duarte, CA