Vera Bzhilyanskaya, BS^1*, Julie Brault, PhD, PharmD^1*, Siyuan Liu, BS^2*, Andres F. Zea Vera, MD, PhD^1*, Nikita Kozhushko, MSc^1*, Amanda Lawson, BA^1*, Uimook Choi, PhD^1*, Aaron B. Clark, BS^3*, Ronald J. Meis, PhD^3*, Michelle Ma, BS^1*, Cicera Lazzarotto, PhD^4*, Shengdar Tsai, PhD⁴, Xiaolin Wu, PhD^2*, Gary A. Dahl, PhD^3*, Jenna Bergerson, MD^1*, Alexandra F. Freeman, MD^1*, Benjamin Kleinstiver, PhD^5*, Harry L Malech, MD¹ and Suk See De Ravin, MD, PhD^1*

¹Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD
²Cancer Research Technology Program, Leidos Biomedical Research, Inc., Frederick, MD
³CELLSCRIPT, LLC, Madison, WI
⁴Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁵Center for Genomic Medicine, Department of Pathology, Massachusetts General Hospital, Charlestown, MA

Jesse Green^*, Adam Johnson, PhD^*, Brian Granger^*, Ouwen Liang^*, Jesus Moreno^*, Andrew Tucker^*, Hallee Wright^*, Brian Dolinski^*, Hanane Ennajdaoui^*, Semih Tareen^*, Neal van Hoeven, PhD^*, Jagesh Shah^*, Kutlu Elpek^*, Aaron Foster and Terry J. Fry, MD

Sana Biotechnology, Seattle, WA

Claire Booth, MBBS, PhD, MSc^1*, Donald B. Kohn, MD², Myriam Armant, PhD^3*, Susan Prockop, MD⁴, Karen Buckland, PhD^5*, Sharat Chandra, MBBS, MD^6*, Shanmuganathan Chandrakasan, MD⁷, Kritika Chetty, MBBS^8*, Colleen Dansereau, MSN, RN, CPN^9*, Satiro de Oliveira, MD^10*, John Everett^11*, Diego León Rico, PhD^8*, Wendy B. London, PhD¹², Jin Hua Xu-Bayford^13*, Theodore B. Moore, MD¹⁴, Nisha Nagarsheth, PhD^15*, Suhag Parikh, MD^16*, Dayna Terrazas, RN^17*, Shanna L White^18*, Axel Schambach, MD, PhD^19*, Frederic D. Bushman, PhD^20*, Adrian J. Thrasher, MBBS, PhD, FMedSci^21*, David A. Williams, MD²² and Sung-Yun Pai, MD²³

¹Great Ormond Street Hospital, London, ENG, United Kingdom
²Broad Institute of Harvard and MIT, University of California - Los Angeles, Los Angeles, CA
³Boston Children's Hospital, Boston, MA
⁴Boston Children's Hospital/Dana Farber Cancer Institute, Boston, MA
⁵University College London/Great Ormond Street Hospital, London, United Kingdom
⁶Cancer and Blood Disease Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁷Department of Pediatrics; Division of Hematology/Oncology, Emory University, Atlanta, GA
⁸UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁹Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA
¹⁰Division of Hematology/Oncology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA
¹¹University of Pennsylvania, Pennsylvania
¹²Boston Children's Hospital, Harvard Medical School, Boston, MA
¹³Great Ormond Street Hospital For Children NHS Trust, London, GBR
¹⁴UCLA Division of Pediatric Hematology, University of California, Los Angeles, Los Angeles, CA
¹⁵National Cancer Institute, National Institutes of Health, Bethesda, MD
¹⁶Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
¹⁷University of California, Los Angeles, Los Angeles, CA
¹⁸UCLA, Los Angeles
¹⁹Experimental Hematology, Hannover Medical School, Hannover, Germany
²⁰Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²¹UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom
²²Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Division of Hematology/Oncology, Boston, MA
²³National Cancer Institute, Center for Cancer Research, NIH, Bethesda, MD

Rofida Nofal, MD^1,2,3*, Yan Yi Chan^4*, Sushmita Sen^4*, Ulises Juarez Figueroa^5*, Hana Willner^4*, Matthias Felber^6*, Mark Krampf, MS^4*, Supawat Thongthip^4*, Grace Choi^7*, Eileen Nicoletti, MD^7*, Jonathan D. Schwartz, MD⁷, Kenneth I. Weinberg, MD⁸, Alfredo Rodriguez^9*, Rajni Agarwal, MD^1,3,10, Maria Grazia Roncarolo, MD, PhD^1,3,10 and Agnieszka Czechowicz, MD, PhD^1,2,3,11

¹Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
²Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
³Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA
⁴Stanford University, Stanford, CA
⁵Laboratorio de Citogenética, Instituto Nacional de Pediatría, Mexico city, Mexico
⁶Universitäts-Kinderspital Zürich, Zurich, Switzerland
⁷Rocket Pharmaceuticals, Inc., Cranbury, NJ
⁸Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
⁹Laboratorio de Citogenética, Instituto Nacional de Pediatría, Mexico City, YU, MEX
¹⁰Lucile Packard Children’s Hospital, Palo Alto, CA
¹¹Lucile Packard Children’s Hospital, Stanford, CA

Claire Booth, MBBS, PhD^1*, Julián Sevilla, MD, PhD^2*, Gayatri R. Rao, MD, JD^3*, Maria Chitty Lopez, MD^3*, Elena Almarza, PhD^3*, Dayna Terrazas, RN^4*, Josune Zubicaray, PhD^2*, Marta González Vicent, MD, PhD^2*, Kritika Chetty, MD^1*, Grainne O'Toole^1*, Jinhua Xu-Bayford^1*, Eileen Nicoletti, MD^3*, Augustine Fernandes, PhD^4*, Caroline Kuo, MD^4*, Satiro de Oliveira, MD^4*, Theodore B. Moore, MD⁴, Grace Choi^3*, Miriam Zeini, PhD^3*, Cristina Mesa-Núñez, PhD^3,5,6*, Adrian J. Thrasher, MBBS, PhD, FMedSci^1*, Juan A. Bueren, PhD^5,6*, Jonathan D. Schwartz, MD³ and Donald B. Kohn, MD⁴

¹UCL Great Ormond Street Institute of Child Health, London, United Kingdom
²Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain
³Rocket Pharmaceuticals, Inc., Cranbury, NJ
⁴University of California, Los Angeles, Los Angeles, CA
⁵Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain
⁶Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain

Adam Giermasz, MD, PhD¹, Nathan Visweshwar, MD^2*, Thomas J. Harrington, MD^3*, Andrew D Leavitt, MD⁴, Barbara Konkle⁵, Jeremy Rupon, MD, PhD⁶, Gregory Di Russo, MD^7*, Li-Jung Tseng, PhD, MBA^8*, Maria de los Angeles Resa, PhD^8*, Florence Ganne^9*, Delphine Agathon, PhD^9*, Frank Plonski, RN, MA^6*, Didier Rouy, MD, PhD^10*, Bettina M. Cockroft, MD, MBA^11*, Annie Fang, MD, PhD^8* and Steven Arkin, MD⁷

¹University of California Davis, Sacramento, CA
²University of South Florida, Tampa, FL
³University of Miami Miller School of Medicine, Miami, FL
⁴University of California San Fransico, San Francisco, CA
⁵Bloodworks Northwest and the University of Washington, Seattle, WA
⁶Pfizer Inc., Collegeville, PA
⁷Pfizer Inc., Cambridge, MA
⁸Pfizer Inc., New York, NY
⁹Pfizer Inc., Paris, France
¹⁰Sangamo Therapeutics, Brisbane, CA
¹¹Sangamo Therapeutics, Inc., Brisbane, CA

Giulia Hardouin^1*, Panagiotis Antoniou, PhD^1*, Pierre Martinucci^1*, Tristan Felix, Ph.D.^2*, Marta Zoccheddu^3*, Anne Chalumeau, Ph.D.^2*, Giacomo Frati^3*, Sandra Manceau^4*, Laure Joseph, MD^5*, Oriana Romano^6,7*, Marina Cavazzana, MD, PhD⁸ and Annarita Miccio, PhD¹

¹Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, 75015, Paris, France
²Université Paris Cite', Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, 75015, Paris, France
³Université de Paris, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, 75015, Paris, France, Paris, France
⁴Hôpital Necker, APHP, Paris, FRA
⁵Biotherapy Department, French National Sickle Cell Referral Center, Hôpital Necker, Assistance-Publique Hôpitaux de Paris, Paris, DC, France
⁶Department of Life Sciences, University of Modena and Reggio Emilia, 41125, Modena, Italy, Modena, Italy
⁷Department of Molecular Medicine, University of Padova, 35131, Padova, Italy., Padova, Italy
⁸Biotherapy Department and Clinical Investigation Center, Assistance Publique Hopitaux de Paris, Inserm, Paris, France

Bhavya S Doshi, MD^1,2,3, Ben J Samelson-Jones, MD, PhD^1,2,4, Timothy C Nichols^5*, Elizabeth P Merricks, PhD^5*, Valder R Arruda, MD, PhD^1,2,4 and Mary Beth Callan, VMD^6*

¹Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Department of Pediatrics, University of Pennsylvania, Philadelphia, PA
⁴Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁵Department of Pathology and Laboratory Medicine and the UNC Blood Research Center, University of North Carolina, Chapel Hill, NC
⁶Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA

Qian Liang, M.D.^1*, Ziqi Zhang^2*, Xuefeng Wang, M.D.^3* and Wenman WU, MD^4*

¹Ruijin Hospital, Shanghai Jiaotong University School of Medicine, shanghai, China
²Ruijin Hospital affiliated to Shanghai Jiaotong University School of Medicine, Shanghai, China
³Faculty of Laboratory Medicine, Ruijin hospital affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, CHN
⁴Department of Laboratory Medicine, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China, shanghai, PA, China

Thiyagaraj Mayuranathan^1*, Gregory A. Newby, PhD^2,3,4,5*, Ruopeng Feng, PhD¹, Yu Yao, MD^6*, Kalin Mayberry, BS^6*, Guolian Kang, PhD, MS⁷, Cicera Lazzarotto, PhD^1*, Yichao Li, Ph.D^1*, Rachel Levine, PhD^1*, Erin Dempsey, BS^1*, Shaina N. Porter, PhD^8*, Phillip A Doerfler, Ph.D⁹, Jingjing Zhang, MS^9*, Yoonjeong Jang, DVM, PhD¹, Senthil Bhoopalan, MBBS, PhD^10*, Nikitha Nimmagadda, MSc^1*, Akshay Sharma, MD^11*, John Tisdale, MD¹², Shondra Miller^8*, Yong Cheng, PhD¹, Shengdar Tsai, PhD¹, Mitchell J. Weiss, MD, Ph.D⁹, David R. Liu, PhD^4,5,13* and Jonathan S Yen, PhD¹

¹Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
²Broad Institute of MIT and Harvard, Cambridge, MA
³Broad Institute of Harvard and MIT, Harvard University, Cambridge, MA
⁴Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA
⁵Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA
⁶Department of Hematology, St Jude Children’s Research Hospital, Memphis, TN
⁷Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN
⁸Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN
⁹Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
¹⁰Department of Hematology, St Jude Children's Research Hospital, Memphis, TN
¹¹Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis
¹²Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
¹³Howard Hughes Medical Institute, Harvard University, Cambridge, MA

Boya Liu, PhD¹, Kevyn Hart^2*, Christian Brendel, PhD^3*, Donald B. Kohn, MD^4,5 and David A. Williams, MD^6,7,8

¹Department of Hematology/Oncology, Boston Children's Hospital, Boston, MA
²Department of Human Genetics Human Genetics, University of California, Los Angeles, Los Angeles, CA
³Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
⁴Department of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, CA
⁵University of California - Los Angeles, Department of Pediatrics, Los Angeles, CA
⁶Harvard Medical School, Boston, MA
⁷Dana-Farber/Boston Children’s Cancer & Blood Disorders Center, Division of Hematology/Oncology, Boston, MA
⁸Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA

Panagiotis Antoniou, PhD^1*, Mathieu von Joest, PhD^1*, Letizia Fontana^1*, Giulia Hardouin^1,2*, Pierre Martinucci^1*, Marina Cavazzana, MD, PhD^2,3 and Annarita Miccio, PhD¹

¹Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, 75015, Paris, France
²Biotherapy Clinical Investigation Center, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France
³Biotherapy Department, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France

Sebastian Treusch, PhD^*, Twaritha Vijay, MS^*, William M. Matern, PhD^*, Kristina Krassovsky, PhD^*, Vincent Siu, MS, MBA^*, Ian Perrone, MS^*, Glen M. Chew, PhD^*, Jane Grogan, PhD^* and Alana Lerner, PhD^*

Graphite Bio, Inc., South San Francisco, CA

Aysha Ansari, MEng^1*, Remant Bahadur KC, PhD^1*, Mohammad Nasrullah, MSc, BPharm², Daniel NM Sundaram, PhD^1*, Luis Morales Burbano, PhD^1*, Joseph Brandwein, MD³, Xiaoyan Jiang, MD, PhD⁴ and Hasan Uludag, PhD^1,2,5*

¹Department of Chemical and Materials Engineering, University of Alberta, Edmonton, AB, Canada
²Faculty of Pharmacy and Pharmaceutical Sciences, University of Alberta, Edmonton, AB, Canada
³Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada
⁴Terry Fox Laboratory, British Columbia Cancer Agency and Department of Medical Genetics, University of British Columbia, British Columbia, Canada
⁵Department of Biomedical Engineering, Faculty of Medicine & Dentistry, University of Alberta, Edmonton, AB, Canada

Alex Alex Pendergast, BSc^1*, So Hyun Park, PhD², Loren A. Binns^1*, Celeste K. Kanne^1*, Cecile Karsenty, MD^3,4, Britney N. Hernandez^1*, Byoungyong Yoo^2*, Yankai Zhang, PhD¹, Gang Bao, PhD^2* and Vivien A. Sheehan, MD, PhD¹

¹Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
²Department of Bioengineering, Rice University, Houston, TX
³Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX
⁴Baylor College of Medicine, Houston, TX