Alexis Thompson, MD, MPH^1,2, Janet L. Kwiatkowski, MD, MSCE^1,2, Jennifer Schneiderman, MD, MS³, Isabelle Thuret, MD^4*, Andreas E. Kulozik, MD, PhD⁵, Evangelia Yannaki, MD^6*, Marina Cavazzana, MD, PhD^7,8,9, Suradej Hongeng, MD^10*, Timothy S. Olson, MD, PhD^1,2, Martin G. Sauer, MD¹¹, Adrian J. Thrasher, MBBS, PhD^12*, Ashutosh Lal, MD¹³, John E. J. Rasko, MBBS, PhD^14,15,16, Shamshad Ali, MS, MStat^17*, Ge Tao, PhD^17*, Himal L. Thakar, MD^17*, Ami Deora, PhD^17*, Clark Paramore, MPH^17*, Niki Witthuhn^17*, Mark C. Walters, MD^18* and Franco Locatelli, MD^19,20

¹Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Children's Hospital of Philadelphia, Philadelphia, PA
³Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL
⁴Hospital Timone, Marseille, France
⁵University of Heidelberg and DKFZ, Heidelberg, Germany
⁶G. Papanikolaou General Hospital, Thessaloniki, Greece
⁷Inserm Assistance Publique Hôpitaux de Paris, Paris, France
⁸Imagine Institute, Paris, France
⁹Hospital Necker, University Paris Descartes, Paris, France
¹⁰Ramathibodi Hospital, Mahidol University, Bangkok, THA
¹¹Hannover Medical School, Hannover, Germany
¹²UCL Great Ormond St Institute of Child Health, London, United Kingdom
¹³UCSF Benioff Children's Hospital Oakland, Oakland, CA
¹⁴Royal Prince Alfred Hospital, Sydney, NSW, AUS
¹⁵Centenary Institute, Camperdown, Australia
¹⁶The University of Sydney, Sydney, Australia
¹⁷bluebird bio, Inc., Somerville, MA
¹⁸UCSF Benioff Children’s Hospital Oakland, Oakland, CA
¹⁹IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
²⁰Catholic University of the Sacred Heart, Rome, Italy

Carlo Castruccio Castracani, PhD, PharmD^1*, Laura Breda, PhD^2*, Tyler E. Papp^3*, Amaliris Guerra, PhD^4*, Enrico Radaelli, DVM, PhD, DECVP^5*, Charles-Antoine Assenmacher, DVM, MSc, DACVP^5*, Giovanni Finesso, DVM^5*, Barbara Mui^6*, Ying K. Tam^6*, Simona Fontana^7*, Chiara Riganti, MD^7*, Veronica Fiorito, PhD^8*, Sara Petrillo, PhD^9*, Emanuela Tolosano, PhD^10*, Hamideh Parhiz, PhD, PharmD^11* and Stefano Rivella, PhD^{12,13,14,15,16}

¹Dpt. of Pediatrics/Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelhia, Philadelphia, PA
³Perelman School of Medicine, Division of Infectious Diseases, University of Pennsylvania, Philadelphia, PA
⁴Children's Hospital of Philadelphia, Philadelphia, PA
⁵Comparative Pathology Core, Department of Pathobiology, University of Pennsylvania, School of Veterinary Medicine, Philadelphia, PA
⁶Acuitas Therapeutics, Vancouver, BC, Canada
⁷Department of Oncology, University of Turin, Turin, ITA
⁸Molecular Biotechnology Center "Guido Tarone", Department of Molecular Biotechnology and Health Sciences, University of Turin, Turin, ITA
⁹Molecular Biotechnology Center "Guido Tarone", Department of Molecular Biotechnology and Health Sciences, University of Turin, Turin, Italy
¹⁰Molecular Biotechnology Center "Guido Tarone", Department of Molecular Biotechnology and Health Sciences, University of Turin, Torino, ITA
¹¹Division of Infectious Diseases, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA
¹²Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
¹³University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA
¹⁴Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
¹⁵Penn Center for Musculoskeletal Disorders, Children's Hospital of Philadelphia, Philadelphia, PA
¹⁶Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia, PA

Mariliis Ott^1*, Hadega Aamer^1*, Daryl Humes^1*, Reuben Burch^1*, Christina Proano^2*, Brian Dolinski^2*, Diana Vedenova^1*, Ashley Benson^1*, Ryan White^1*, Semih Tareen^1*, Jesse Green, PhD^1*, Kutlu G Elpek, PhD^2*, Neal van Hoeven^1* and Terry J Fry, MD¹

¹Sana Biotechnology, Seattle, WA
²Sana Biotechnology, Cambridge, MA

Michael T. Monte, MS^*, Lorenzo Tozzi, PhD^*, Rahul Palchaudhuri, PhD^*, Apiwat Wangweerawong, PhD^*, Yaser Heshmati, PhD, Giulia Schiroli, PhD^*, Han Gu, PhD^*, Yu Cao, PhD^*, William E. Salomon, PhD^*, Anne Bothmer, PhD^*, Jason Rodriguez^*, Mohit Gupta, PhD^*, James B. Rottman, PhD, DVM^*, William Querbes, PhD^*, Cecilia Cotta-Ramusino, PhD^*, Michael C Holmes, PhD^*, Hari Pujar, PhD^* and Z. Jane Wang, PhD^*

Tessera Therapeutics, Somerville, MA

Sravya Kattula, PhD^1*, Gaurav M. Rajani, MS^1*, Vemika Chandra, PhD^2*, Mike M. Martinez, MS^1*, Raissa Eluere, PhD^1*, Patrick Au, PhD, DABT^1*, Robert T. Peters, PhD² and Joe Salas, PhD²

¹Ensoma, Boston, MA
²Ensoma, Inc., Boston, MA

Luis Daniel Daniel Mata Casimiro^1*, Manisha Du Plessis^2*, Dennis Unthan^3*, Heyang Zhang^3*, Matthias Barz, PhD^3*, Raymond M. Schiffelers, PhD^4* and Olaf Heidenreich, PhD^2,5

¹Princess Máxima Center for Pediatric Oncology, Utrecht, AL, Netherlands
²Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands
³Leiden Academic Centre for Drug Research (LACDR), Leiden University, Leiden, Netherlands
⁴CDL Research, University Medical Center Utrecht, Utrecht, Netherlands
⁵Wolfson Childhood Cancer Research Centre, Newcastle University, Newcastle upon Tyne, United Kingdom

Nils Wellhausen, BSc¹, Yuqi Zhou^2*, Michael S Bowman, PhD^3*, Kusala Anupindi, BS^2*, Abhimanyu Gowda^2*, Nils Engel, MD^2*, Carl H. June, MD⁴, Robert L. Bowman, PhD³, James Riley, PhD^2* and Saar Gill, MD, PhD⁵

¹Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, PA
²University of Pennsylvania, Philadelphia
³Department of Cancer Biology, University of Pennsylvania School of Medicine, Philadelphia, PA
⁴Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA
⁵University of Pennsylvania, Philadelphia, PA

Jean-Sebastien Diana, MD^1*, Despina Moshous, MD, PhD^2*, Martin Castelle, MD^2*, Boris Bessot, B.S.^3*, Elisa Magrin, PhD, PharmaD^4*, Marwa Chbihi, MD^5*, Paul Bastard, MD, Ph.D^6*, Alice Girardot^4*, Cecile Roudaut, B.S.^7*, Chloe Mollet, Ph.D^2*, Clotilde Aussel, PhD, PharmD^8*, Caroline Tuchmann-Durand^4*, Eden Schwartz, PharmD^4*, Olaf Neth, MD, Ph.D^9*, Peter Olbrich, MD, Ph.D^10*, Irene d'Alba, MD^11*, Jacques Riviere, MD^12*, Medhi Oualha, MD, Ph.D^8*, Laure Joseph, MD^13*, Benedicte Neven, MD, Ph.D^14*, Jean-Pierre De Villartay, Ph.D^3*, Anne Galy, PharmD, PhD^15*, Chantal Lagresle-Peyrou, PhD^3* and Marina Cavazzana, MD, PhD⁴

¹Necker Hospital, Biotherapy Department, PARIS, PARIS, France
²Necker Hospital (APHP), Paris, France
³Institut Imagine, Inserm, Paris, France
⁴Necker Hospital, Biotherapy Department, Paris, France
⁵APHP, Hopital Necker, Paris, FRA
⁶Hopital Necker, APHP, Paris, France
⁷Hopital Necker (APHP), Paris, France
⁸Hopital Necker (APHP), Paris, France
⁹Pediatric Infectious Diseases, Rheumatology and Immunology Unit, Hospital Universitario Virgen del Rocío, Institute of Biomedicine of Seville (IBIS)/ Universidad de Sevilla/CSIC, Red de Investigación Traslacional en Infectología Pediátrica RITIP, Sevilla, Spain
¹⁰Hospital Infantil Virgen Del Rocio, Sevilla, ESP
¹¹Maternal Infant Hospital "G. Salesi", Ancona, Italy
¹²Hospital Vall D´Hebron, Barcelona, Spain
¹³Biotherapy Department, French National Sickle Cell Referral Center, Hopital Necker, AP-HP, Paris, France
¹⁴BMT unit, Hopital Necker (APHP), Paris, France
¹⁵Genethon, Evry, France

Linah Omer, MD¹, William Johnston, MD^2*, Sarah Adams, BS^1*, Omar Banda, PhD^1*, Elena Sofia Vivona, MD^1*, Stefano Rivella, PhD^3,4, Mohamad-Gabriel Alameh, phD^5*, William Peranteau, MD^6* and Peter Kurre, MD^7,8

¹Comprehensive Bone Marrow Failure Center, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Department of medicine, University of Pennsylvania, Philadelphia
³Penn Institute for RNA Innovation, University of Pennsylvania, Philadelphia, PA
⁴Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
⁵Department of Medicine, University of Pennsylvania, Philadelphia, PA
⁶The Children's Hospital of Philadelphia, Philadelphia, PA
⁷Comprehensive Bone Marrow Failure Center, Children's Hospital of Philadelphia, Philadelphia, PA
⁸Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA