65th ASH Annual Meeting & Exposition: 801. Gene Therapies: Poster III

Symposia: Gene Therapies Oral and Poster Abstracts
Poster

Halls G-H (San Diego Convention Center)

Safety and Efficacy of RM-001 (Autologous HBG1/2 Promoter-modified CD34+ Hematopoietic Stem and Progenitor Cells) in Patients with Transfusion-Dependent β-Thalassemia

Rongrong Liu¹^*, Li Wang²^*, Hui Xu, PhD³, Xiaolin Yin⁴^*, Junbin Liang³^*, Wenqiang Xie¹^*, Gaohui Yang¹^*, Yaoyun Li⁵^*, Yali Zhou⁵^*, Lei Shi³^*, Bin Xiao⁵^*, Lingling Shi¹^*, Zeyan Shi¹^*, Xuemei Zhou¹^*, Xiangmin Xu⁶^*, Jianpei Fang⁷^*, Yongrong Lai¹^*, Junjiu Huang⁸^* and Xinhua Zhang, MD²^*

¹Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
²923rd Hospital of the People's Liberation Army, Nanning, Guangxi, China
³Reforgene Medicine, Guangzhou, China
⁴Department of Hematology, 923rd Hospital of the People's Liberation Army, Nanning, China
⁵Department of Pediatrics, 923rd Hospital of the People's Liberation Army, Nanning, China
⁶Department of Medical Genetics, School of Basic Medical Sciences, Southern Medical University, Guangzhou, China
⁷Sun Yat-Sen Memorial Hospital of Sun Yat-sen University, Guangzhou, China
⁸School of Life Sciences, Sun Yat-sen University, Guangzhou, China

4995

Efficacy and Safety of Brl-101, CRISPR-Cas9-Mediated Gene Editing of the BCL11A Enhancer in Transfusion-Dependent β-Thalassemia

Biao Zheng, MD, PhD¹, Rongrong Liu²^*, Xinhua Zhang, MD³^*, Bin Fu, MD⁴^*, Yajing Xu, MD⁴^*, Jun Shi⁵^*, Xiaoqin Feng⁶^*, Li Wang³^*, Chris Wang, PhD¹^*, Rongmei Liang¹^*, Lingzhi Tan¹^*, Fei Wang, PhD¹^*, Dali Li, MD, PhD^1,7^*, Yuxuan Wu, PhD⁷^*, Mingyao Liu, MD, PhD^1,7^* and Yongrong Lai, MD, PhD²^*

¹BRL Medicine Inc., Shanghai, China
²Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China
³923rd Hospital of the People's Liberation Army, Nanning, Guangxi, China
⁴Xiangya Hospital, Central South University, Changsha, Hunan, China
⁵Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
⁶Department of Pediatrics, Nanfang Hospital, Guangzhou, China
⁷Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China

4996

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Rabi Hanna, MD¹, Haydar Frangoul, MD², Christopher McKinney, MD³^*, Luis Pineiro, MD⁴^*, Markus Mapara, MD⁵, Jignesh Dalal, MD⁶^*, Kai-Hsin Chang, PhD⁷, Michael Jaskolka, PhD⁷^*, Keunpyo Kim, PhD⁷^*, Daphne L. Farrington, MS⁷^*, Maha Wally, BS, MB⁷^*, Baisong Mei, MD, PhD⁷, Adebayo Lawal, MD, MSc, MBA⁷, Olubunmi O. Afonja, MD, MBA⁷ and Mark C. Walters, MD⁸

¹Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic, Cleveland, OH
²Sarah Cannon Research Institute at the Children's Hospital, Tristar Children's Hospital, Nashville, TN
³Children’s Hospital Colorado, University of Colorado Anschutz Medical Campus, Aurora, CO
⁴Blood and Marrow Transplant Program and Marrow Processing Laboratory, Baylor University Medical Center, Dallas, TX
⁵Blood and Marrow Stem Cell Transplantation Program, Division of Hematology & Oncology, Columbia University Irving Medical Center, New York, NY
⁶Division of Hematology and Bone Marrow Transplant, Rainbow Babies & Children’s Hospital, Cleveland, OH
⁷Editas Medicine, Inc., Cambridge, MA
⁸University of California, San Francisco Benioff Children’s Hospital, Oakland, CA

4997

Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Franco Locatelli, MD, PhD¹, Peter Lang, MD, PhD²^*, Selim Corbacioglu, MD, PhD³^*, Donna Wall, MD⁴, Amanda M Li, MD⁵, Josu de La Fuente, PhD⁶, Puja Kohli, MD, MMSc⁷^*, Nanxin Li, PhD, MBA⁷, Jaime Rubin, MA, MPH⁷^*, Siyu Zhang, PhD⁷^*, Lanju Zhang, PhD⁷^*, William Hobbs, MD, PhD⁷ and Haydar Frangoul, MD⁸

¹IRCCS, Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy
²University of Tübingen, Tübingen, Germany
³University of Regensburg, Regensburg, Germany
⁴SickKids, Toronto, Canada
⁵BC Children’s Hospital, University of British Columbia, Vancouver, Canada
⁶Imperial College Healthcare NHS Trust, St Mary's Hospital, London, United Kingdom
⁷Vertex Pharmaceuticals, Boston, MA
⁸Sarah Cannon Research Institute at The Children’s Hospital at TriStar Centennial, Nashville, TN

4998

Gene Therapy of Transfusion-Dependent β-Thalassemia Patients with Quick Engraftment of Reinfused Hematopoietic Stem Cells: An Investigator-Initiated Trial of KL003

Jinqi Huang¹^*, Yuming Zhang, M.D.¹^*, Liang Liang, M.D.¹^*, Jiayuan Wu, Ph.D.¹^*, Xiaoqian Li, M.D.¹^*, Zhonglu Ye¹^*, Bin Lin¹^*, Jingzhi Ma¹^*, Lingling Su²^*, Qing Ye²^*, Jianan Sun²^*, Zhengliang Wang²^*, Baozhen Sun²^*, Xiaodong Hu²^*, Peng Lv²^*, Jianjun Tao²^*, Ying Dang²^*, Depei Wu, M.D.¹, Youren Tong²^* and Haoquan Wu²^*

¹Department of Hematology, Affiliated Hospital of Guangdong Medical University, Zhanjiang, Guangdong, China
²Kanglin Biotech (Hangzhou) Co., Ltd., Hangzhou, China

4999

Improvements in Health-Related Quality of Life after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease

Akshay Sharma, MBBS¹, Haydar Frangoul, MD², Markus Mapara, MD³^*, Suzan Imren, MD⁴^*, Nanxin Li, PhD, MBA⁴, Tina Liu, PhD⁴^*, Jaime Rubin, MA, MPH⁴^*, Daoyuan Shi, PhD⁴^*, William Hobbs, MD, PhD⁴ and Stephan Grupp, MD, PhD⁵

¹St. Jude Children's Research Hospital, Memphis, TN
²Sarah Cannon Research Institute at The Children’s Hospital at TriStar Centennial, Nashville, TN
³Department of Medicine, Division of Hematology/Oncology, Columbia University, New York, NY
⁴Vertex Pharmaceuticals, Boston, MA
⁵Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA

5000

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

David C Shyr, MD¹, Robert Lowsky, MD²^*, Weston Miller, MD³^*, Mark A. Schroeder, MD⁴, Tonia Buchholz, PhD⁵^*, Kirstin Dougall, MBA⁶^*, Allison Intondi⁵^*, Alexandra Charles⁷^*, Josh Lehrer, MD, MPhil⁵^*, Ali Bouge⁷^*, Stacey Wolf⁷^*, Blair MacDonald⁷^*, Hena Din⁷^*, Alana Lerner, PhD⁵^*, Manal Amoury³^*, Sebastien Treusch, PhD³^*, Glen Chew⁸^*, Brian Silva⁸^*, Haider Mashhedi³^*, Vincent Siu³^*, Ian Perrone³^*, Twaritha Vijay³^*, Aayami Jain³^*, Kristina Krassovsky⁸^*, William Matern⁸^*, Premanjali Lahiri³^*, Ryan Rodriguez, PhD³^*, Jason Skowronski^3,9^*, Arpit Batish⁷^*, Dana Margittai⁷^*, Prachi Wani⁷^*, Timothy Van Horn⁷^*, Claudia Flautero⁷^*, Rosalva Flores³^*, Jade Lee³^*, Keri Tate¹⁰^*, Maria-Grazia Roncarolo, MD¹¹, Steven Feldman, PhD¹²^*, John DiPersio¹³ and Matthew Porteus, MD, PhD¹⁴

¹Stanford University, Palo Alto, CA
²Stanford Univ. Med. Ctr., Stanford, CA
³Graphite Biologics, South San Francisco
⁴Siteman Cancer Center, Washington University, Saint Louis, MO
⁵Graphite Biologics, South San Francisco, CA
⁶Stanford University School of Medicine, Palo Alto, CA
⁷Stanford University, Stanford
⁸Graphite Bio, South San Francisco, CA
⁹Kamau Therapeutics, South San Francisco
¹⁰Stanford University, Stanford, CA
¹¹Stanford School of Medicine, Stanford, CA
¹²Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University, Stanford, CA
¹³Washington University School of Medicine, Saint Louis, MO
¹⁴Stanford Medical School, Stanford, CA

5001

Prime Editing of the α-Thalassemia Hb Constant Spring Mutation

Congwen Shao¹^*, Qing Liu²^*, Jinchao Xu³^*, Ye Xin⁴^*, Jianxiang Zhang⁴^*, Yuhua Ye¹^*, Hualei Luo²^*, Xinhua Zhang, MD⁵^*, Xiangmin Xu⁶^* and Peng Xu, PhD⁷

¹Southern Medical University, Guangzhou, CHN
²Southern Medical University, Guangzhou, China
³Soochow University, suzhou, China
⁴Soochow University, Suzhou, China
⁵923rd Hospital of the People's Liberation Army, Nanning, Guangxi, China
⁶Department of Medical Genetics, School of Basic Medical Sciences, Southern Medical University, Guangzhou, China
⁷Hematology Center, Cyrus Tang Medical Institute, Soochow University, Suzhou, Jiang Su, China

5002

Optimization of Lentiviral ß-Globin Vectors in a Forward Orientation to Enhance Therapeutic Effectiveness for Sickle Cell Disease

Jennifer Okalova¹^*, Jordan Shields Alexander, BS¹^*, Christopher Chambliss, PhD¹, Zhongyu Zhu, PhD²^*, Yanping Xie, PhD²^*, Matthew Addington-Hall, BS²^*, Oxana Slessareva, MS²^*, Tony Luo, PhD²^*, Beatrix Ferencz, PhD²^*, Ibeawuchi Oparaocha, MS²^*, Naoya Uchida, MD, PhD³, John F. Tisdale, MD⁴, Boro Dropulic, PhD, MBA²^*, H. Trent Spencer, PhD⁵^*, Rimas J Orentas, PhD² and David R Archer, PhD⁶

¹Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA
²Caring Cross, Gaithersburg, MD
³Cellular and Molecular Therapeutics Branch, NHLBI/NIDDK, NIH, NHLBI, MCHB, Bethesda, MD
⁴Cellular and Molecular Therapeutics Branch, NHLBI/NIDDK, NIH, Bethesda, MD
⁵Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA
⁶Aflac Cancer & Blood Disorders Center, Emory University, Atlanta, GA

5003

Using Human Genetics to Develop Strategies to Increase Erythropoietic Output from Genome-Edited Hematopoietic Stem and Progenitor Cells

Sofia E Luna, BA¹, Joab Camarena, BS²^*, Jessica P Hampton, BS², Kiran R Majeti, BS²^*, Carsten T Charlesworth²^*, Eric Soupene, PhD³^*, Vivien A. Sheehan, MD, PhD⁴, Kyle Cromer, PhD, MPhil, BS⁵ and Matthew Porteus, MD, PhD⁶

¹Stanford University School of Medicine, Stanford, CA
²Stanford University, Stanford, CA
³Children’s Hospital of Oakland Research Institute, Oakland, CA
⁴Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
⁵Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA
⁶Department of Pediatrics, Stanford University, Stanford, CA

5004

Phase I Trial of Systemic Administration of Vesicular Stomatitis Virus Genetically Engineered to Express NIS and Human Interferon, in Patients with Relapsed or Refractory Multiple Myeloma, Lymphomas, or Histiocytic/Dendritic Cell Neoplasms

N. Nora Bennani, MD¹, Joselle Cook, MBBS², Susan M. Geyer, PhD³, Brenda F Ginos¹^*, Lianwen Zhang¹^*, Javier Munoz, MD⁴^*, Stephen Broski¹^*, Stephen Russell, MD, PhD^1,5, Kah-Whye Peng, PhD¹^* and Martha Lacy, MD¹^*

¹Mayo Clinic, Rochester, MN
²Division of Hematology, Mayo Clinic, Rochester, MN
³Alliance Statistics and Data Management Center, Mayo Clinic, Rochester, MN
⁴Mayo Clinic, Phoenix
⁵Vyriad, Rochester, MN

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