62nd ASH Annual Meeting and Exposition: 801. Gene Editing, Therapy and Transfer: Poster I

Oral and Poster Abstracts
Poster

Adult, Biological, Hemophilia, HSCs, sickle cell disease, Animal models, bioengineering, Bleeding and Clotting, Diseases, Lymphoma (any), sickle cell trait, CAR-Ts, Therapies, Genetic Disorders, red blood cells, Biological Processes, Hemoglobinopathies, immunodeficiency, cell expansion, gene therapy, immune cells, Immune Disorders, T-Cell Lymphoma, Technology and Procedures, Cell Lineage, immunotherapy, gene editing, infusion, Lymphoid Malignancies, Study Population, Clinically relevant, genomics, hematopoiesis, transplantation, stem cells

1539

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD¹, Francesca Ferrante, MD²^*, Muriel Reis³^*, Sara Wiegmann⁴^*, Claudia Lange⁵^*, Manuela Braun⁵^* and Lisa A Michaels⁶^*

¹Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI
²Bayer, Basel, Switzerland
³Bayer, Sao Paulo, Brazil
⁴Bayer, Wuppertal, Germany
⁵Bayer, Berlin, Germany
⁶Bayer, Whippany, NJ

1540

Robust Pre-Clinical Results and Large-Scale Manufacturing Process for EDIT-301: An Autologous Cell Therapy for the Potential Treatment of SCD

Edouard De Dreuzy, PhD¹, Jack Heath¹^*, Patricia Sousa¹^*, Tusneem Janoudi¹^*, Harry An, PhD¹^*, Scott Hansen²^*, David K. Wood, PhD²^*, Charles F Albright, PhD¹^*, Sandra Teixeira, PhD¹^*, Tamara Monesmith¹^*, Kate Zhang, PhD¹ and Kai-Hsin Chang, PhD¹

¹Editas Medicine, Cambridge, MA
²Biomedical Engineering, University of Minnesota, Minneapolis, MN

1541

CoupledCAR^TM Technology for Treating Thyroid Cancer

Xingchen Liu¹^*, Lei Xiao, PhD², Keshu Zhou¹^*, Yu Liu³^*, Yong Huang³^*, Chengfei Pu²^*, Zhiyuan Cao²^*, Ruihong Zhu²^*, Haiyang Tang²^*, Zhipeng Huang²^*, Hang Yang²^*, Xi Huang²^*, Yongping Song¹^*, Renbin Liu³^* and Zhao Wu²^*

¹Zhengzhou University, Zhengzhou, China
²Innovative Cellular Therapeutics, Shanghai, China
³SUN YAT-SEN University, Guangzhou, China

1542

In Vivo HSC Gene Therapy for Hemoglobinopathies: A Proof of Concept Evaluation in Rhesus Macaques

Chang Li¹^*, Hongjie Wang, PhD¹^*, Sucheol Gil²^*, Afrodite Georgakopoulou, PhD student³^*, Stefan Radtke, PhD⁴, Evangelia Yannaki, MD^5,6^*, Thalia Papayannopoulou, MD, DrSci⁷, Hans-Peter Kiem, MD PhD⁴ and Andre Lieber, MD, PhD²

¹Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, WA
²University of Washington, Seattle, WA
³George Papanicolaou Hospital, Thessaloniki, Greece
⁴Fred Hutchinson Cancer Research Center, Seattle, WA
⁵G. PAPANICOLAOU HOSPITAL, THESSALONIKI, Greece
⁶Hematology Department and HCT Unit, George Papanikolaou General Hospital, Thessaloniki, Greece
⁷Division of Hematology, Department of Medicine, University of Washington, Seattle, WA

1543

Adenine Base Editing of the Sickle Allele in CD34⁺ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

S. Haihua Chu, PhD^*, Daisy Lam^*, Michael S. Packer, Ph.D.^*, Jennifer Olins^*, Alexander Liquori^*, Jeffrey Marshall, PhD^*, Calvin Lee^*, Bo Yan, PhD^*, Jeremy Decker^*, Bob Gantzer, M.S.^*, Scott J. Haskett, M.S.^*, Tanggis Bohnuud, PhD^*, David Born, PhD^*, Luis Barrera, PhD^*, Ian Slaymaker, PhD^*, Nicole M. Gaudelli, Ph.D.^*, Adam J. Hartigan, PhD^* and Giuseppe Ciaramella, Ph.D., B.Sc.^*

Beam Therapeutics, Cambridge, MA

1544

Human Orthogonal IL-2/IL-2Rβ As a Tunable Approach to Enhance CD19-Specific CAR-T Cell Antitumor Activity

Qian Zhang, MD PhD¹^*, Morgan Hresko²^*, Michael Jacob Hollander³^*, Lora Picton⁴^*, Leon Su⁴, Selene Nunez-Cruz⁵^*, Jonathan T Sockolosky³^*, K.Christopher Garcia, PhD⁶^* and Michael C. Milone⁷

¹Pathology and Lab medicine, University of Pennsylvania, Philadelphia, PA
²University of Pennsylvania, Piladelphia, PA
³Stanford University School of Medicine, Stanford, CA
⁴Stanford University Medical School, Stanford, CA
⁵University of Pennsylvania, Philadelphia, PA
⁶Howard Hughes Medical Institute, Stanford University School of Medicine, Stanford, CA
⁷Center for Cellular Immunotherapies, Perelman School of Medicine At the Univ. of Pennsylvania, Philadelphia, PA

1545

Adenine Base Editing of Gamma Globin Gene Promoters Shows No Detectable Off-Target RNA or DNA Editing

Ling Lin, PhD^*, Lauren E. Young, MS^*, Jennifer Olins^*, Jeremy Decker^*, Alexander Liquori^*, Yingying Zhang, PhD^*, Haihua Chu^*, Daisy Lam^*, Conrad Rinaldi, M.S.^*, Adrian P. Rybak, Ph.D.^*, Michael S. Packer, Ph.D.^*, Nicole M. Gaudelli, Ph.D.^*, Luis Barrera, PhD^*, Jeffrey Marshall, PhD^*, Matt Humes, PhD^*, Bob Gantzer, M.S.^*, Brian Cafferty, PhD^*, Aaron Larson, PhD^*, Carlo Zambonelli^*, Yeh-Chuin Poh, Ph.D.^*, Manmohan Singh, Ph.D.^*, Adam J. Hartigan, PhD^* and Giuseppe Ciaramella, Ph.D., B.Sc.^*

Beam Therapeutics, Cambridge, MA

1546

Defined Expansion Protocol Supports Ex Vivo Expansion of Gene Edited Hematopoietic Stem Cells to the Single Cell Level

Hans Jiro Jiro Becker¹^*, Masatoshi Sakurai, MD, PhD² and Satoshi Yamazaki, PhD^3,4^*

¹Institute of Medical Science, Division of Stem Cell Biology, The University of Tokyo, Tokyo, TKY, Japan
²Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan
³Institute of Medical Science, Division of Stem Cell Biology, The University of Tokyo, Tokyo, Japan
⁴Laboratory of Stem Cell Therapy, Faculty of Medicine University of Tsukuba, Ibaraki, Japan

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^*signifies non-member of ASH

801.P1.30 801. Gene Editing, Therapy and Transfer: Poster I