Asif Alavi, MD¹, Lakshmanan Krishnamurti, MD², Mehrdad Abedi, MD³, Isobelle Galeon, MD, MS, MPH^4*, David Reiner, MD^5*, Sharon E Smith, MD^4*, Lin Wang, PhD^4*, Anne Ramezi, MS^4*, Pablo Rendo⁴ and Mark C. Walters, MD⁶

¹Karmanos Cancer Institute, Wayne State University, Detroit, MI
²Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory Healthcare, Atlanta, GA
³University of Davis Medical Center, Sacramento, CA
⁴Sanofi, Cambridge, MA
⁵Sanofi, Bridgewater, NJ
⁶Division of Hematology, UCSF Benioff Children's Hospital Oakland, Oakland, CA

Cory Sago, PhD^1*, Elizabeth Campbell, Ph.D.^2*, Brianna Lutz, Ph.D.^2*, Neeraj Patwardhan, Ph.D.^2*, Gregory Hamilton, Ph.D.^2*, Courtney Wong^2*, Daniel Lee, M.S.^2*, Shayon Keating^2*, Kathryn Murray, M.S.^2*, Manmohan Singh, Ph.D.^1* and Giuseppe Ciaramella, Ph.D.^1*

¹Beam Therapeutics, Cambridge, MA
²Beam Therapeutics, Cambridge

Donald B. Kohn, MD¹, Claire Booth, MBBS, PhD, MSc^2*, Julián Sevilla, MD, PhD^3*, Gayatri R Rao, MD, JD^4*, Elena Almarza, PhD^4,5,6*, Dayna Terrazas, RN^1*, Eileen Nicoletti, MD^4*, Augustine Fernandes, PhD^1*, Caroline Kuo, MD^1*, Satiro de Oliveira, MD^1*, Theodore B. Moore, MD¹, Kenneth M Law, PhD^4*, Brian C Beard, PhD^4*, Grace Choi, BS^4*, Miriam Zeini, PhD^4*, Cristina Mesa-Núñez, PhD^5,6*, Adrian J. Thrasher, MBBS, PhD, FMedSci^2*, Juan A Bueren, PhD^5,6* and Jonathan D Schwartz, MD⁴

¹University of California, Los Angeles, Los Angeles, CA
²UCL Great Ormond Street Institute of Child Health, London, United Kingdom
³Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
⁴Rocket Pharmaceuticals, Inc., Cranbury, NJ
⁵Unidad de Innovación Biomédica, Centro 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

Rkia El Kharrag, PhD^*, Kurt Berckmueller^*, Margaret Cui^*, Ravishankar Madhu^*, Anai M Perez^*, Olivier Humbert, PhD^*, Hans-Peter Kiem, MD PhD and Stefan Radtke, PhD

Fred Hutchinson Cancer Research Center, Seattle, WA

Alessandra Magnani, MD, PhD^1,2*, Michaela Semeraro, MD, PhD^1,3*, Frédéric ADAM^4*, Claire Booth, MBBS, PhD, MSc^5,6,7*, Loic Dupre, PhD^8*, Emma C Morris^9*, Aurélie Gabrion^10*, Cecile Roudaut^2*, Delphine Borgel, PhD, PharmD^11*, Antoine Toubert, MD, PhD^12,13,14*, Emmanuel Clave, PhD^15*, Chrystelle Abdo, PharmD^16*, Guy Gorochov^17*, Rachel Petermann^18*, Mélanie Guiot^19*, Makoto Miyara, MD^20*, Despina Moshous, MD, PhD^1,21,22*, Elisa Magrin, PharmD^10*, Adeline Denis^23*, Felipe Suarez, MD, PhD²⁴, Chantal Lagresle-Peyrou^25,26,27*, Aoife Doto^28*, John K. Everett, PhD^29*, Amélie Trinquand^30*, Marianne Guisset^31*, Jin Hua Xu-Bayford^32*, Salima Hacein-Bey-Abina^33,34*, Alexandre Kauskot, PhD^35*, Reem Elfeky, MD, PhD^36*, Christine Rivat^6,7,37*, Sarah Abbas^38*, Bobby H Gaspar, MBBS, PhD, BS^6,39*, Elizabeth A. Macintyre, PhD, MD^40,41,42, Capucine Picard, MD, PhD^43,44,45,46*, Frederic D. Bushman, PhD^28,29*, Anne Galy, PharmD, PhD^47,48, Alain Fischer, MD, PhD^49,50,51,52, Emmanuelle Six, PhD^53,54*, Adrian J. Thrasher, MBBS, PhD, FMedSci^6,7,55,56* and Marina Cavazzana, MD, PhD^57,58,59,60

¹Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
²Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France
³IMAGINE Institute, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
⁴INSERM, Le Kremlin-bicêTre, FRA
⁵Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom
⁶UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁷University College London/Great Ormond Street Hospital, London, United Kingdom
⁸CPTP, Inserm UMR 1043, Toulouse, France
⁹University College London, London, United Kingdom
¹⁰Biotherapy Department and Clinical Investigation Center, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France
¹¹INSERM, AP-HP, Paris, FRA
¹²Laboratoire d’Immunologie et d’Histocompatibilité, Hopital Saint-Louis - APHP, Paris, France
¹³Institut Universitaire d’Hématologie, INSERM UMR 1160, Université Paris Diderot, Sorbonne Paris Cité, Paris, France
¹⁴INSERM UMR 1160, Hopital Saint-Louis - APHP, Paris, France
¹⁵Institut Universitaire d’Hématologie, INSERM UMRS 1160, Université Paris Diderot, Sorbonne Paris Cité, Paris, France
¹⁶Hopital Necker enfants malades, Paris, FRA
¹⁷Hopital Pitie Salpetriere, Paris, FRA
¹⁸Institut National de la Transfusion Sanguine (INTS), Department of Platelet Immunology, F-75739, Paris, France
¹⁹Université Pierre Et Marie Curie, Paris, FRA
²⁰Immunochemistry department, Pitie Salpetriere Hospital, Paris, FRA
²¹INSERM UMR 1163, Laboratory of Genome Dynamics in the Immune System, Equipe Labellisée Ligue contre le Cancer, Paris, France
²²Imagine Institute, Genome Dynamics in the Immune System, INSERM U1163, Paris, France
²³INSERM UMR 1163, Paris, FRA
²⁴Service d'Hématologie adultes, Hôpital Universitaire Necker-Enfants Malades, AP-HP, Paris, France
²⁵Biotherapy Clinical Investigation Center, INSERM. Institut Imagine U1163. Université de Paris, Paris, France
²⁶Biotherapy Clinical Center, Assistance Publique - Hôpitaux de Paris, Paris, France
²⁷INSERM, Paris, France
²⁸University of Pennsylvania, Philadelphia, PA
²⁹Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³⁰Université Paris Descartes Sorbonne Cité, Institut Necker-Enfants Malades (INEM), INSERM U1151, and Laboratory of Onco-Hematology, Assistance Publique-Hôpitaux de Paris (AP-HP), Necker - Enfants Malades University Hospital, Paris, France, Paris, France
³¹Toulouse Institute for Infectious and Inflammatory Diseases (INFINITy), INSERM, CNRS, Toulouse III Paul Sabatier University, Toulouse, France, Toulouse, France
³²Great Ormond Street Hospital For Children NHS Trust, London, GBR
³³Université de Paris, CNRS, INSERM, UTCBS, Unité des technologies Chimiques et Biologiques pour la Santé, Paris, France, Paris, France
³⁴Clinical Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Le-Kremlin-Bicêtre, France, Paris, France
³⁵UMR-S Inserm 1176, Univ Paris Sud, Université Paris-Saclay, le Kremlin-Bicêtre, France
³⁶Department of Paediatric Immunology, Great Ormond Street Hospital, London, UK, London, GBR
³⁷Institute of Child Health, Molecular and Cellular Immunology Unit, University College London, London, United Kingdom
³⁸Généthon, Evry, France, Evry, France
³⁹Orchard Therapeutics, London, United Kingdom
⁴⁰Institut Necker Enfants Malades, Université de Paris (Descartes), Paris, France
⁴¹Université Paris Descartes Sorbonne Cité, Institut Necker-Enfants Malades (INEM), Institut national de recherche médicale (INSERM) U1151, Laboratory of Onco-Haematology, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades, Paris, Paris, France
⁴²Laboratory of Onco-Hematology, Necker University Hospital, Paris, France
⁴³Laboratory of lymphocyte activation and susceptibility to EBV, Imagine Institute UMR1163, Paris, France
⁴⁴Department of Pediatric Immunology, Hematology and Rheumatology, Necker Hospital, Paris, FRA
⁴⁵Study Center for Primary Immunodeficiencies, Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
⁴⁶Imagine Institute, University Paris Descartes-Sorbonne Paris Cité, Paris, France
⁴⁷GENETHON, EVRY, FRA
⁴⁸Genethon, Evry, France
⁴⁹INSERM, Paris, FRA
⁵⁰Department of Pediatric Immunology, Hematology and Rheumatology, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
⁵¹Collège de France, Paris, France
⁵²Imagine Institute, University Paris Descartes-Sorbonne Paris Cité, France, Paris, France
⁵³Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France
⁵⁴Imagine Institute, Paris Descartes–Sorbonne Paris Cité University, Paris, France
⁵⁵UCL Institute of Child Health, London, GBR
⁵⁶University College London; Great Ormond Street Hospital NHS Trust, London, United Kingdom
⁵⁷University of Paris, Imagine Institute, Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France
⁵⁸Smart Immune, Paris, France
⁵⁹IMAGINE Institute Sorbonne Paris Cité, Université Paris Descartes, Paris, France
⁶⁰Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Paris, France

Mohsin Badat, MB BChir^1*, Peng Hua, PhD¹, Sachith Mettananda, DPhil, FRCP^2*, Christopher Fisher^3*, Noemi Roy, MD, PhD⁴, Siobhan Rice^1*, Anindita Roy^3*, Douglas R. Higgs, FRS^3* and James Davies, PhD³

¹MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, ENG, United Kingdom
²Department of Paediatrics, University of Kelaniya, Ragama, AL, LKA
³MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, United Kingdom
⁴Department of Haematology, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom

Daisuke Araki, MD¹, Christi Salisbury-Ruf, MS, PhD^1*, Waleed Hakami^1*, Keyvan Keyvanfar, BS^2*, Richard H. Smith, PhD^1* and Andre Larochelle, MD, PhD¹

¹Laboratory of Regenerative Therapies for Inherited Blood Disorders, Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
²Clinical Flow Core Facility, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Alessandra Mortellaro^1*, Matteo Zoccolillo^2*, Cristina Mesa Nuñez^2*, Alessia Brix^3*, Immacolata Brigida^1*, Federica Barzaghi^4*, Serena Scala^2*, Raisa Jofra Hernández^1*, Luca Basso-Ricci^2*, Mariasilvia Colantuoni^1,5*, Simone Cesaro^6*, Francesca Conti^7*, Andrea Pession^8*, Fabio Benedetti^9*, Marco Gattorno^10*, Luigi Naldini^2,5*, Maria Pia Cicalese^4*, Anna Pistocchi^3* and Alessandro Aiuti, MD, PhD^2,4,5

¹San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, ITA
²San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
³Department of Medical Biotechnology and Translational Medicine, Università degli Studi di Milano, Milan, Italy
⁴Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
⁵Vita-Salute San Raffaele University, Milan, Italy
⁶Pediatric Hematology-Oncology, Ospedale della Donna e del Bambino, Verona, Italy
⁷Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, ITA
⁸Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
⁹Hematology and Stem Cell Transplantation, Azienda Ospedaliera Universitaria di Verona, Verona, Italy
¹⁰Centro Malattie Autoinfiammatorie e Immunodeficienze, IRCCS Istituto Giannina Gaslini, Genoa, GE, ITA

Ritu M Ramamurthy^1*, Wen Ting Zheng^2*, Sunil George, PhD^1*, Meimei Wan^1*, Yu Zhou, PhD^1*, Baisong Lu, PhD^1*, Colin E Bishop, PhD^1*, Anthony Atala, M.D.^1*, Christopher D Porada, PhD^1* and M. Graca Almeida-Porada, MD³

¹Fetal Research and Therapy Program, Wake Forest Institute for Regenerative Medicine, Winston Salem, NC
²Massachusetts Institute of Technology, Cambridge, MA
³Fetal Research and Therapy Program, Wake Forest Institute For Regenerative Medicine, Winston-Salem, NC

Nipul Patel^1*, Michael Pettiglio, BS^1*, Christopher Cummins, BS^1*, Jeff Pimentel^1*, Anjali Kapuria^1*, Azita Ghodssi^1*, Meltem Isik, PhD^1*, Alejandra Falla, PhD^1*, Ravindra Amunugama, PhD^1*, Jessica Lisle, PhD^1*, Shu Wang^2*, Michelle Lin, PhD¹, John Lydeard, PhD^1*, Gary Ge, PhD^1*, Dane Hazelbaker, PhD^1*, Elizabeth Paik, PhD^1* and Tirtha Chakraborty, PhD^1*

¹Vor Biopharma, Cambridge, MA
²Vor Biopharma, Cambridge

Christi Salisbury-Ruf, MS, PhD^1*, Richard H. Smith, PhD^2*, Fariba Chinian, MS^3*, Daisuke Araki, MD⁴, Keyvan Keyvanfar^5* and Andre Larochelle, MD, PhD³

¹Laboratory of Regenerative Therapies for Inherited Blood Disorders, Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, NIH, NHLBI, Bethesda, MD
²Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, Bethesda, MD
³Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
⁴Clinical Research Center, Room 5-5248, National Institutes of Health, Bethesda, MD
⁵Flow Cytometry Core, NIH, NHLBI, Bethesda, MD

Jie Gong, MSc^1*, Tsai-Hua Chung, PhD^1,2*, Min Zhou^3* 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
³Chengdu Women's and Children's Central Hospital, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China

Christie Ciarlo, PhD^1*, Zach Frye^1*, Andre DeGroot^1*, Walter Flores^1*, Kutlu Elpek, Ph.D.^1*, Lauren Pepper^1*, Adam Johnson, PhD^1*, Jagesh V Shah, PhD^1*, Aaron E. Foster, PhD² and Terry J. Fry, MD¹

¹Sana Biotechnology, Seattle
²Sana Biotechnology, Seattle, WA