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

Oral and Poster Abstracts
Oral

Adult, ALL, Biological, CRS, HSCs, Leukemia, Diseases, neurotoxicity, CAR-Ts, Elderly, Therapies, Adverse Events, Pediatric, Biological Processes, immunodeficiency, white blood cells, cell expansion, gene therapy, immune cells, Immune Disorders, Technology and Procedures, Cell Lineage, immunotherapy, gene editing, infusion, Lymphoid Malignancies, Study Population, Clinically relevant, genomics, hematopoiesis, flow cytometry, immune mechanism, NGS

Moderators:

Susanne H.C. Baumeister, MD, Harvard Medical School, Boston Children's Hospital, Dana-Farber Cancer Institute and Amit C. Nathwani, MD, PhD, University College London

Disclosures:

No relevant conflicts of interest to declare.

2:00 PM

497

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Natalia Izotova¹^*, Christine Rivat¹^*, Cristina Baricordi, PhD²^*, Elena Blanco-Alvarez¹^*, Danilo Pellin²^*, Eleanor Watt³^*, Soragia Athina Gkazi, PhD¹^*, Stuart Adams, PhD³^*, Kimberly Gilmour³^*, Jinhua Xu Bayford³^*, Claire Booth, MBBS, PhD, MSc¹^*, Bobby H Gaspar, MBBS, PhD, BS¹^*, Adrian J. Thrasher, MBBS, PhD, FMedSci¹^* and Luca Biasco, PhD^1,2

¹UCL Great Ormond Street Institute of Child Health, London, United Kingdom
²Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
³Great Ormond Street Hospital, London, United Kingdom

2:15 PM

498

Deep and Durable Remissions of Relapsed Multiple Myeloma on a First-in-Humans Clinical Trial of T Cells Expressing an Anti-B-Cell Maturation Antigen (BCMA) Chimeric Antigen Receptor (CAR) with a Fully-Human Heavy-Chain-Only Antigen Recognition Domain

Lekha Mikkilineni, MD¹, Elisabet E. Manasanch, MD², Danielle Vanasse, BS³^*, Jennifer N. Brudno, MD⁴, Jennifer Mann, CRNP⁴^*, Richard Sherry, MD⁴^*, Stephanie L Goff, MD⁴^*, James C Yang, MD⁵^*, Norris Lam, BS³^*, Irina Maric, MD, MSci⁶, Maryalice Stetler-Stevenson, MD, PhD⁷^*, Hao-Wei Wang, MD, PhD⁸, Constance M. Yuan, MD, PhD⁷^*, David F. Stroncek, MD⁹, Steven L Highfill, PhD¹⁰^*, Vicki Fellowes¹¹^*, Micaela Ganadan, BSN, RN¹^*, Rashmika Patel¹²^*, Steven A Rosenberg, MD, PhD¹³^* and James N. Kochenderfer, MD^14,15

¹Surgery Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD
²Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
³Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD
⁴Surgery Branch, National Cancer Institute, Bethesda, MD
⁵Surgery Branch, National Cancer Institute, Maryland
⁶Department of Laboratory Medicine, National Institutes of Health, Bethesda, MD
⁷Laboratory of Pathology, National Cancer Institute, NIH, Bethesda, MD
⁸Department of Laboratory Medicine, National Cancer Institute, National Institute of Health, Bethesda, MD
⁹Center for Cellular Engineering, National Institutes of Health, Bethesda, MD
¹⁰Cell Processing Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MD, USA, National Institutes of Health, Bethesda, MD
¹¹Cell Processing Section, Department of Transfusion Medicine, National Cancer Institute, Bethesda, MD
¹²Center for Cancer Research, National Cancer Institute, Bethesda
¹³Surgery Branch, National Cancer Institute, Bethesda
¹⁴Surgery Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD
¹⁵Surgery Branch, National Cancer Institute/National Institutes of Health, Bethesda, MD

2:30 PM

499

The Safety and Efficacy of a CRISPR/Cas9-Engineered Universal CAR-T Cell Product (CTA101) in Patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

Yongxian Hu, MD¹^*, Yali Zhou²^*, Mingming Zhang, MD¹^*, Wengang Ge²^*, Yi Li, MD¹^*, Li Yang, MD¹^*, Guoqing Wei, MD¹^*, Lu Han²^*, Hao Wang²^*, Xingwang Zhang, PhD²^*, Jiangtao Ren, PhD²^* and He Huang, MD, PhD¹

¹Bone Marrow Transplantation Center, The First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, China
²Nanjing Bioheng Biotech Co., Ltd, Nanjing, China

2:45 PM

500

Long-Term Outcomes Following Donor-Derived Anti-CD19 CAR-T Cell Therapy for B-Cell Acute Lymphoblastic Leukemia Patients Relapsed after Allogenic Stem Cell Transplantation

Rongli Zhang, MD¹^*, Ying Wang, MD², Dehui Zou, MD³^*, Erlie Jiang²^*, Yi He¹^*, Donglin Yang¹^*, Sizhou Feng, MD¹^*, Jianxiang Wang, MD¹ and Ming-Zhe Han¹

¹National Clinical Research Center for Blood Diseases, State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
³lymphoma and myeloma center, National Clinical Research Center for Blood Diseases, State Key Laboratory of Experimental Hematology, Blood Diseases Hospital & Institute of Hematology, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China

3:00 PM

501

Novel CoupledCAR^TM Technology for Treating Colorectal Cancer

Lei Xiao, PhD¹, Song Li²^*, Chengfei Pu¹^*, Zhiyuan Cao¹^*, Xinyi Yang³^*, Ning Li³^*, Youli Luo⁴^*, Haiyan Zhao⁵^*, Hang Yang¹^*, Xi Huang¹^*, Xiaogang Shen¹^*, Xiuwen Wang²^*, Yongping Song³^*, Junjie Mao⁴^*, Pengfei Pang⁴^*, Zhao Wu¹^*, Victor Lu, PhD⁶^* and Qun Hu⁵^*

¹Innovative Cellular Therapeutics, Shanghai, China
²Shandong University, Jinan, China
³Zhengzhou University, Zhengzhou, China
⁴SUN YAT-SEN University, Guangzhou, China
⁵Inner Mongolia Medical University, Neimenggu, China
⁶Innovative Cellular Therapeutics, Inc, Rockville, MD

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