-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.O1.6 801. Gene Therapies: Advances in Clinical Gene Therapy for Hematological Disorders Clinically Relevant Abstract

Symposia: Gene Therapies Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Sickle Cell Disease, Bleeding and Clotting, Biological therapies, bleeding disorders, Research, clinical trials, Bone Marrow Failure Syndromes, hemophilia, Inherited Marrow Failure Syndromes, Translational Research, Genetic Disorders, Thalassemia, Clinical Research, Hemoglobinopathies, pediatric, Diseases, Immune Disorders, immunodeficiency, Gene Therapy, Therapies, white blood cell disorders, computational biology, Technology and Procedures, Metabolic Disorders, gene editing, Study Population, Human, omics technologies
Monday, December 12, 2022: 10:30 AM-12:00 PM
220-222 (Ernest N. Morial Convention Center)
Moderators:
Ashish O. Gupta, MBBS, MPH, University of Minnesota and Erica B. Esrick, MD, Children's Hospital Boston
Disclosures:
No relevant conflicts of interest to declare.
10:30 AM

Kasiani C. Myers, MD1, Stella M Davies, MBBS, PhD, MRCP2, Carolyn Lutzko, PhD3*, Robin Wahle3*, Geraldine Aubert, PhD4*, Tomokazu Amano, PhD5*, Misa Amano5*, Hong Yu5* and Minoru S.H. Ko, MD, PhD5*

1Department of Pediatrics, Division of Bone Marrow Transplantation and Immune Deficieny, Cincinnati Children's Hospital, Cincinnati, OH
2Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
3Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
4Repeat Diagnostics Inc,, Vancouver, BC, Canada
5Elixirgen Therapeutics, Inc, Baltimore, MD

10:45 AM

Robert F Wynn, MD1, Jane L Kinsella, MBChB2*, Rebecca J Holley, BSc, PhD3*, Jane Potter, MBBS, MSc, BSc4*, Adrian J. Thrasher, MBBS, PhD, FMedSci5*, Claire Booth, MBBS, PhD, MSc6*, Karen Buckland, PhD7*, Natalia Izotova, PhD8*, Stewart Rust, B.Sc. (Hons), D.Clin.Psych, Pg.Dip., C.Psychol., AfBPS.9*, Daniel Weisberg, BSc, PhD, PGCert, ClinPsyD9*, Heather J Church, PhD10*, Karen L Tylee11*, Kathryn L Brammeier11*, Helena Lee12*, Laura Ford12*, Atusa Sadegholnejat12*, Simon A Jones, MBChB, BSc, MRCPCH11*, Stuart Ellison13* and Brian Bigger, PhD14*

1Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom
2Paediatric Bone Marrow Transplant, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom
3Stem Cell and Neuropathies, University of Manchester, Manchester, United Kingdom
4Royal Manchester Childrens Hospital, Manchester University Foundation Trust, Manchester, United Kingdom
5UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom
6Institute of Child Health, London, ENG, United Kingdom
7University College London/Great Ormond Street Hospital, London, United Kingdom
8Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
9Royal Manchester Children's Hospital, Manchester, United Kingdom
10Willink Biochemical Genetics Unit, Manchester Foundation Trust, Manchester, United Kingdom
11Willink Biochemical Genetics Unit, Manchester NHS Foundation Trust, Manchester, United Kingdom
12Transplantation Laboratory, Manchester NHS Foundation Trust, Manchester, United Kingdom
13Stem Cell and Neurotherapies Laboratory, University of Manchester, Manchester, United Kingdom
14Stem Cell and Neurotherapies, University of Manchester, Manchester, United Kingdom

11:00 AM

Stacy E. Croteau, MD1,2, M. Elaine Eyster, MD3, Huyen Tran, MD, PhD4*, Margaret V. Ragni, MD, MPH5, Benjamin J. Samelson‑Jones, MD, PhD6*, Lindsey George, M.D.6*, Spencer Sullivan, MD7, John E. J. Rasko, MBBS, PhD8,9, Jill Moormeier, MD, MPH10*, Pantep Angchaisuksiri, MD11,12, Jerome Teitel, MD13, Gili Kenet, MD14, Tung Wynn, MD15*, Kristen Jaworski, MSN, NP16*, Amy Macdougall, BSN16*, Savina Jaeger, PhD16*, Trupti Trivedi16*, Federico Mingozzi, PhD16, Tiffany Chang, MD, MAS16* and Gallia Levy, MD, PhD16*

1Department of Pediatrics, Harvard Medical School, Boston, MA
2Division of Hematology and Oncology, Boston Children’s Hospital, Boston, MA
3Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey, PA
4Australian Centre for Blood Diseases, Monash University, Melbourne, VIC, Australia
5Department of Medicine, University of Pittsburgh, Pittsburgh, PA
6Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia, PA
7Mississippi Center for Advanced Medicine, Madison, MS
8Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, Sydney, Australia
9The Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney, Sydney, Australia
10Department of Medicine, UMKC School of Medicine, Kansas City, MO
11Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
12University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC
13St Michael's Hospital Haemophilia Treatment Centre, University of Toronto, Toronto, ON, Canada
14Israel National Hemophilia Center and Thrombosis Institute, Sheba Medical Center, Tel Hashomer, Israel
15Division of Hematology & Oncology, Department of Pediatrics, University of Florida, Gainesville, FL
16Spark Therapeutics, Philadelphia, PA

11:15 AM

Giang N. Nguyen, BS1*, Maria C. Seleme, PhD1*, Jonathan R. Lindgren, BS1*, Allysen C. Henriksen, BS1*, Amy Muehlmatt, MLAS1,2*, Melanie McFadden, DVM, MLAS1,2*, Geary R. Smith, DVM, MS1,2* and Denise E. Sabatino, PhD1,3

1The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA
2Comparative Medicine Services Core, The Children's Hospital of Philadelphia, Philadelphia, PA
3Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

11:30 AM

Andrea Calabria, PhD1*, Giulio Spinozzi2*, Daniela Cesana, PhD2*, Fabrizio Benedicenti3*, Giulia Pais4*, Serena Scala, PhD3*, Maria Rosa Lidonnici, PhD5*, Samantha Scaramuzza, PhD3*, Alessandra Albertini2*, Simona Esposito2*, Fabiola De Mattia3*, Daniele Canarutto, MD6,7*, Francesca Tucci, MD8,9*, Maryam Omrani2*, Francesca Dionisio, MSc10*, Stefania Giannelli, PhD10*, Sarah Marktel, MD11,12*, Valeria Calbi, MD6,13*, Francesca Ferrua, MD, PhD13,14*, Bernhard Gentner, MD, PhD4,15, Fabio Ciceri, MD16*, Luigi Naldini, MD, PhD7,13*, Giuliana Ferrari, PhD7,17, Alessandro Aiuti, MD, PhD4,9 and Eugenio Montini, PhD1*

1San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milano, Italy
2San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, ITA
3San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy
4San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy
5San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, MI, Italy
6Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
7Vita-Salute San Raffaele University, Milan, Italy
8San Raffaele Telethon Institute for Gene Therapy, Milano, Italy
9Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
10San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), San Raffaele Scientific Institute, Milan, Italy
11Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
12Pediatric Immunohematology and Bone Marrow Transplantation Unit, Milano, ITA
13San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
14Pediatric Immunohematology and Stem Cell Programme, San Raffaele Scientific Institute, Milan, ITA
15Translational Stem Cell and Leukemia Research Unit, San Raffale Scientific Institute, Milano, Italy
16Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy
17San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy

11:45 AM

Akshay Sharma, MBBS1, Jaap Jan Boelens, MD, PhD2, Maria I. Cancio, MD2, Jane S Hankins, MD, MS3, Prafulla A Bhad, MSc4*, Andrew Lewandowski, PhD4*, Xiaojun Zhao, PhD4*, Shripad Chitnis, PhD4*, Radhika Peddinti, MD5, Yan Zheng, MD, PhD, MS1*, Neena Kapoor, MD6*, Fabio Ciceri, MD7*, Jianping Yuan, PhD4*, Vionnie W. Yu, PhD4, Susan C. Stevenson4*, Serena De Vita, MD, PhD4 and James LaBelle, MD, PhD5

1St. Jude Children's Research Hospital, Memphis, TN
2Memorial Sloan Kettering Cancer Center, New York, NY
3Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
4Novartis Institutes for BioMedical Research, Cambridge, MA
5The University of Chicago Medicine, Chicago, IL
6Children's Hospital Los Angeles, Los Angeles, CA
7Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy

*signifies non-member of ASH