64th ASH Annual Meeting and Exposition: 801. Gene Therapies: Advances in Clinical Gene Therapy for Hematological Disorders

Symposia: Gene Therapies Oral and Poster Abstracts
Oral

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

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

781

Successful Ex Vivo Telomere Elongation with Exg-001 in a Patient with a Dyskeratosis Congenita

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

782

Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

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

783

Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011

Stacy E. Croteau, MD^1,2, 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, PhD^8,9, Jill Moormeier, MD, MPH¹⁰^*, Pantep Angchaisuksiri, MD^11,12, 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

784

Second Generation Adeno-Associated Viral Vector Using a Novel Factor VIII Variant with Improved Secretion Achieves Higher Factor VIII Expression in Non-Human Primates

Giang N. Nguyen, BS¹^*, Maria C. Seleme, PhD¹^*, Jonathan R. Lindgren, BS¹^*, Allysen C. Henriksen, BS¹^*, Amy Muehlmatt, MLAS^1,2^*, Melanie McFadden, DVM, MLAS^1,2^*, Geary R. Smith, DVM, MS^1,2^* and Denise E. Sabatino, PhD^1,3

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

785

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

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, MD^6,7^*, Francesca Tucci, MD^8,9^*, Maryam Omrani²^*, Francesca Dionisio, MSc¹⁰^*, Stefania Giannelli, PhD¹⁰^*, Sarah Marktel, MD^11,12^*, Valeria Calbi, MD^6,13^*, Francesca Ferrua, MD, PhD^13,14^*, Bernhard Gentner, MD, PhD^4,15, Fabio Ciceri, MD¹⁶^*, Luigi Naldini, MD, PhD^7,13^*, Giuliana Ferrari, PhD^7,17, Alessandro Aiuti, MD, PhD^4,9 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

786

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo, CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

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

See more of: Oral and Poster Abstracts

<< Previous Session | Next Session >>

^*signifies non-member of ASH

801.O1.6 801. Gene Therapies: Advances in Clinical Gene Therapy for Hematological Disorders