-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

801. Gene Therapies: Poster II

Program: Oral and Poster Abstracts
Type: Poster
Sunday, December 12, 2021: 6:00 PM-8:00 PM
Hall B5, Level 1, Building B (Georgia World Congress Center)

Asif Alavi, MD1, Lakshmanan Krishnamurti, MD2, Mehrdad Abedi, MD3, Isobelle Galeon, MD, MS, MPH4*, David Reiner, MD5*, Sharon E Smith, MD4*, Lin Wang, PhD4*, Anne Ramezi, MS4*, 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, PhD1*, Elizabeth Campbell, Ph.D.2*, Brianna Lutz, Ph.D.2*, Neeraj Patwardhan, Ph.D.2*, Gregory Hamilton, Ph.D.2*, Courtney Wong2*, Daniel Lee, M.S.2*, Shayon Keating2*, 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, MSc2*, Julián Sevilla, MD, PhD3*, Gayatri R Rao, MD, JD4*, Elena Almarza, PhD4,5,6*, Dayna Terrazas, RN1*, Eileen Nicoletti, MD4*, Augustine Fernandes, PhD1*, Caroline Kuo, MD1*, Satiro de Oliveira, MD1*, Theodore B. Moore, MD1, Kenneth M Law, PhD4*, Brian C Beard, PhD4*, Grace Choi, BS4*, Miriam Zeini, PhD4*, Cristina Mesa-Núñez, PhD5,6*, Adrian J. Thrasher, MBBS, PhD, FMedSci2*, Juan A Bueren, PhD5,6* 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, PhD1,2*, Michaela Semeraro, MD, PhD1,3*, Frédéric ADAM4*, Claire Booth, MBBS, PhD, MSc5,6,7*, Loic Dupre, PhD8*, Emma C Morris9*, Aurélie Gabrion10*, Cecile Roudaut2*, Delphine Borgel, PhD, PharmD11*, Antoine Toubert, MD, PhD12,13,14*, Emmanuel Clave, PhD15*, Chrystelle Abdo, PharmD16*, Guy Gorochov17*, Rachel Petermann18*, Mélanie Guiot19*, Makoto Miyara, MD20*, Despina Moshous, MD, PhD1,21,22*, Elisa Magrin, PharmD10*, Adeline Denis23*, Felipe Suarez, MD, PhD24, Chantal Lagresle-Peyrou25,26,27*, Aoife Doto28*, John K. Everett, PhD29*, Amélie Trinquand30*, Marianne Guisset31*, Jin Hua Xu-Bayford32*, Salima Hacein-Bey-Abina33,34*, Alexandre Kauskot, PhD35*, Reem Elfeky, MD, PhD36*, Christine Rivat6,7,37*, Sarah Abbas38*, Bobby H Gaspar, MBBS, PhD, BS6,39*, Elizabeth A. Macintyre, PhD, MD40,41,42, Capucine Picard, MD, PhD43,44,45,46*, Frederic D. Bushman, PhD28,29*, Anne Galy, PharmD, PhD47,48, Alain Fischer, MD, PhD49,50,51,52, Emmanuelle Six, PhD53,54*, Adrian J. Thrasher, MBBS, PhD, FMedSci6,7,55,56* and Marina Cavazzana, MD, PhD57,58,59,60

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 BChir1*, Peng Hua, PhD1, Sachith Mettananda, DPhil, FRCP2*, Christopher Fisher3*, Noemi Roy, MD, PhD4, Siobhan Rice1*, Anindita Roy3*, Douglas R. Higgs, FRS3* 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, PhD1*, Waleed Hakami1*, Keyvan Keyvanfar, BS2*, Richard H. Smith, PhD1* 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 Mortellaro1*, Matteo Zoccolillo2*, Cristina Mesa Nuñez2*, Alessia Brix3*, Immacolata Brigida1*, Federica Barzaghi4*, Serena Scala2*, Raisa Jofra Hernández1*, Luca Basso-Ricci2*, Mariasilvia Colantuoni1,5*, Simone Cesaro6*, Francesca Conti7*, Andrea Pession8*, Fabio Benedetti9*, Marco Gattorno10*, Luigi Naldini2,5*, Maria Pia Cicalese4*, Anna Pistocchi3* and Alessandro Aiuti, MD, PhD2,4,5

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 Ramamurthy1*, Wen Ting Zheng2*, Sunil George, PhD1*, Meimei Wan1*, Yu Zhou, PhD1*, Baisong Lu, PhD1*, Colin E Bishop, PhD1*, Anthony Atala, M.D.1*, Christopher D Porada, PhD1* 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 Patel1*, Michael Pettiglio, BS1*, Christopher Cummins, BS1*, Jeff Pimentel1*, Anjali Kapuria1*, Azita Ghodssi1*, Meltem Isik, PhD1*, Alejandra Falla, PhD1*, Ravindra Amunugama, PhD1*, Jessica Lisle, PhD1*, Shu Wang2*, Michelle Lin, PhD1, John Lydeard, PhD1*, Gary Ge, PhD1*, Dane Hazelbaker, PhD1*, Elizabeth Paik, PhD1* and Tirtha Chakraborty, PhD1*

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

Christi Salisbury-Ruf, MS, PhD1*, Richard H. Smith, PhD2*, Fariba Chinian, MS3*, Daisuke Araki, MD4, Keyvan Keyvanfar5* 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, MSc1*, Tsai-Hua Chung, PhD1,2*, Min Zhou3* and Lung-Ji Chang, PhD1,2

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, PhD1*, Zach Frye1*, Andre DeGroot1*, Walter Flores1*, Kutlu Elpek, Ph.D.1*, Lauren Pepper1*, Adam Johnson, PhD1*, Jagesh V Shah, PhD1*, Aaron E. Foster, PhD2 and Terry J. Fry, MD1

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

*signifies non-member of ASH