Akshay Sharma, MBBS¹, Jane S Hankins, MD, MS¹, Jaap Jan Boelens, MD², Maria Cancio^2*, Radhika Peddinti, MD, MBBS³, Lawrence Rispoli, BA^4*, Sarah Sloan^5*, Sarah DiDominick Costa^4*, Sharon Peled^4*, Andrea Wiethoff^4* and James LaBelle, MD, PhD³

¹St. Jude Children's Research Hospital, Memphis, TN
²Memorial Sloan Kettering Cancer Center, New York, NY
³University of Chicago Medicine, Chicago
⁴Novartis Biomedical Research, Cambridge, MA
⁵Novartis Pharma AG, Basel, Switzerland

Haydar Frangoul, MD¹, Franco Locatelli, MD², Akshay Sharma, MBBS³, Monica Bhatia, MD^4*, Markus Y Mapara, MD⁵, Robert I. Liem, MD⁶, Donna Wall, MD⁷, Lyndsay Molinari, MD⁸, Laurence Dedeken, MD⁹, Kevin H.M. Kuo, MD, MSc, FRCPC¹⁰, Paul Telfer, MD, FRCP^11*, Ami J. Shah, MD¹², Marina Cavazzana, MD, PhD¹³, Selim Corbacioglu, MD, PhD¹⁴, Michael J. Eckrich¹⁵, Damiano Rondelli, MD^16*, Roland Meisel, MD^17*, Stephan Lobitz, MD, MSc^18*, Mariane De Montalembert, MD, PhD¹³, Martin H. Steinberg, MD¹⁹, Mark C. Walters, MD^20*, Laura Bower, MD^21*, Suzan Imren, MD^21*, Tina Liu, PhD^21*, Weiyu Zhou, PhD^21*, William Hobbs, MD, PhD²¹ and Stephan A. 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
³Department of Bone Marrow Transplantation and Cellular Therapy, St. 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
⁵Department of Medicine, Division of Hematology & Oncology, Columbia University, New York
⁶Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁷The Hospital for Sick Children/University of Toronto, Toronto, Canada
⁸Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children’s Hospital, San Antonio, TX
⁹Hôpital Universitaire des Enfants Reine Fabiola, Brussels, BEL
¹⁰Division of Hematology, University of Toronto, Toronto, ON, Canada
¹¹Royal London Hospital, Barts Health NHS Trust, London, United Kingdom
¹²Stanford University, Palo Alto, CA
¹³Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris, Paris, France
¹⁴University of Regensburg, Regensburg, Germany
¹⁵Atrium Health in Charlotte, Charlotte, NC
¹⁶Division of Hematology/Oncology, University of Illinois College of Medicine, Chicago, IL
¹⁷Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany
¹⁸Pädiatrische Hämatologie und Onkologie, Gemeinschaftsklinikum Mittelrhein, Koblenz, Germany
¹⁹Boston University Chobanian & Avedisian School of Medicine, Boston, MA
²⁰UCSF Benioff Children’s Hospital Oakland, Oakland, CA
²¹Vertex Pharmaceuticals Incorporated, Boston, MA
²²Children’s Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

Rabi Hanna, MD¹, Haydar Frangoul, MD², Christopher McKinney, MD³, Luis Pineiro, MD^4*, Markus Mapara, MD, PhD^5*, Jignesh Dalal, MD^6*, Hemalatha Rangarajan, MD⁷, Harold Atkins, MD^8*, Kai-Hsin Chang, PhD⁹, Baisong Mei, MD, PhD^9*, Olubunmi Afonja, MD, MBA^9* and Mark C. Walters, MD^10*

¹Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland, OH
²Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial, Nashville, TN
³Children’s Hospital of Colorado, Department of Pediatrics, University of Colorado Anschutz Medical Campus, Aurora, CO
⁴Blood and Marrow Transplant Program and Marrow Processing Laboratory, Baylor University Medical Center, Dallas, TX
⁵Bone Marrow Transplantation and Cell Therapy Program, Columbia University Irving Medical Center, New York City, NY
⁶Rainbow Babies & Children’s Hospital, Cleveland, OH
⁷Department of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Nationwide Children's Hospital, Columbus, OH
⁸Transplant and Cell Therapy Program, Department of Medicine, The Ottawa Hospital, Ottawa, ON, Canada
⁹Editas Medicine, Inc., Cambridge, MA
¹⁰School of Medicine, University of California, San Francisco and Benioff Children’s Hospital, Oakland, CA

Britney Hernandez, BS^1*, Ashwin P. Patel, MD, PhD, MPH², Erica N Evans^1*, Jordan Zgodny, BA^1*, Stephen Gottschalk, MD³, Brandon M Triplett, MD^3*, Serena De Vita, MD, PhD^4*, Kirby Wallace, PhD^5*, David C Shyr, MD⁶, Elizabeth O Stenger, MD, MSc⁷, Suhag Parikh, MD^7*, Akshay Sharma, MBBS⁸ and Vivien Sheehan, MD, PhD⁹

¹Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
²Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
³Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN
⁴Novartis Biomedical Research, Cambridge, MA
⁵Kamau Therapeutics, South San Francisco, CA
⁶Department of Pediatrics/ Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
⁷Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA
⁸Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN
⁹Emory University School of Medicine, Atlanta, GA

Priya S. Chockalingam, PhD^1*, Ling Lin, PhD^1*, Guo Chen, PhD^1*, Alex C. Minella, MD¹, Yinzhong Chen, PhD^1*, Vivien Sheehan, MD, PhD², Nan Zhang, MD, PhD^3*, Myriam Armant, PhD^4*, Aliya U. Zaidi, PhD^5*, Robert Goodrich, BSc^6*, Patrick C. Hines, MD, PhD⁵, Sunita Goyal, MD^1* and Amy Simon, MD^1*

¹Beam Therapeutics Inc., Cambridge, MA
²Emory University School of Medicine, Atlanta, GA
³Frontage Laboratories, Inc - Exton, PA, Exton, PA
⁴Translab, Boston Children's Hospital, Boston, MA
⁵Functional Fluidics Inc, Detroit, MI
⁶Functional Fluidics Inc., Detroit, MI

Sandra Santos^1*, Tara M. Robinson, MD, PhD² and David Trueman^3*

¹BioMarin UK Ltd., London, United Kingdom
²BioMarin Pharmaceutical Inc., Novato, CA
³Source Health Economics, London, United Kingdom

Li Wang, MD^1*, Hui Xu, PhD², Junbin Liang^2*, Yaoyun Li^3*, Lin Cheng^2*, Ying Luo^2*, Liancheng Huang^2*, Lei Shi^2*, Lihong Zeng^4*, Rongrong Luo^3*, Bin Xiao^3*, Meiping Liu^3*, Liping Huang^3*, Zhendong Xiao^5*, Xinhua Zhang, MD^1* and Junjiu Huang, PhD^6*

¹923rd Hospital of the People's Liberation Army, Nanning, Guangxi, China
²Reforgene Medicine, Guangzhou, China
³Department of Pediatrics, 923rd Hospital of the People's Liberation Army, Nanning, China
⁴Department of Blood Transfusion, 923rd Hospital of the People's Liberation Army, Nanning, China
⁵Biotherapy Center, The Third Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China
⁶School of Life Sciences, Sun Yat-sen University, Guangzhou, China

Rongrong Liu^1*, Li Wang^2*, Hui Xu, PhD³, Jianpei Fang^4*, Sixi Liu^5*, Xiaolin Yin^6*, Junbin Liang^3*, Xinyu Li^4*, Yue Li^5*, Gaohui Yang^1*, Yaoyun Li^2*, Yali Zhou^6*, Lei Shi^3*, Yongrong Lai, MD^1*, Junjiu Huang, PhD^7* and Xinhua Zhang, MD^8*

¹Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
²Department of Pediatrics, 923rd Hospital of the People's Liberation Army, Nanning, China
³Reforgene Medicine, Guangzhou, China
⁴Department of Pediatrics, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou, China
⁵Department of Pediatrics, Shenzhen Children’s Hospital, Shenzhen, China
⁶Department of Hematology, 923rd Hospital of the People's Liberation Army, Nanning, China
⁷School of Life Sciences, Sun Yat-sen University, Guangzhou, China
⁸923rd Hospital of the People's Liberation Army, Nanning, Guangxi, China

Ruonan Li^1,2,3*, Zhen Gao^1,2,3*, Yucan Shen^1,2,3*, Lele Zhang^1,2,3*, Ke Huang^1,2,3*, Hong Pan^1,3,4*, Weiwang Li^1,2,5*, Linzhu Tian^1,2,3*, Jingyu Zhao, MSc^1,2,3*, Liyun Li^1,2,3*, Xiao Yu^1,2,3*, Jing Xu^1,2,3*, Chun Xu^1,2,3*, Yanjie Liu^1,2,3*, Weiwei Wang^1,2,3*, Zhexiang Kuang^1,2,6*, Weiping Yuan^1,7 and Jun Shi, MD^1,2,3*

¹Tianjin Institutes of Health Science, Tianjin, China
²Red Blood Cell Diseases Center & Regenerative Medicine Clinic, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
³State 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
⁴Red Blood Cell Diseases Center & Regenerative Medicine Clinic, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, AL, China
⁵State 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, AL, China
⁶State 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, AL, China
⁷State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, National Clinical Research Center for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China

Yongrong Lai, MD^1*, Rongrong Liu, Doctor^1*, Lijie Wang, PhD^2,3*, Xu-Kai Ma, PhD^4,5,6*, Yaliang Li, MSc^7*, Gaohui Yang^1*, Lingling Shi, MD^1*, Jia Wei^4,5,6*, Zhenbin Wei^1*, Xuemei Zhou^1*, Kewei Lan^2*, Yan Zhang, PhD^2*, Xiaodun (Susan) Mou, PhD^2*, Li Yang, PhD^4,5,6* and Jia Chen, PhD^3,8

¹Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
²CorrectSequence Therapeutics Co., Ltd, Shanghai, China
³Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai, China
⁴Center for Molecular Medicine, Children’s Hospital of Fudan University and Shanghai Key Laboratory of Medical Epigenetics, Shanghai, China
⁵International Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Shanghai, China
⁶Institutes of Biomedical Sciences, Fudan University, Shanghai, China
⁷CorrectSequence Therapeutics Co., Ltd, Shanghai, AL, China
⁸Shanghai Clinical Research and Trial Center, Shanghai, China