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

Oral and Poster Abstracts
Poster

Anemias, Biological, Hemophilia, HSCs, sickle cell disease, Bleeding and Clotting, Diseases, thalassemia, Therapies, Adverse Events, Genetic Disorders, Biological Processes, Hemoglobinopathies, gene therapy, Immune Disorders, Technology and Procedures, Cell Lineage, gene editing, Study Population, Clinically relevant, immune mechanism, transplantation, stem cells

2446

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

José Luis López Lorenzo, MD^1,2^*, Susana Navarro, PhD^2,3,4^*, Ami J Shah, MD^5,6,7, Maria Grazia Roncarolo, MD^5,6,7, Julián Sevilla, MD, PhD⁸^*, Lucía Llanos, MD, PhD^1,2^*, Begoña Pérez Camino de Gaisse, MD^1,2^*, Sol Sanchez, MD^1,2^*, Bertil Glader, MD, PhD^7,9, May Chien, MD^7,9, Oscar Quintana Bustamante, PhD^2,3,4^*, Brian C Beard, PhD¹⁰^*, Kenneth M Law, PhD¹⁰^*, Miriam Zeini, PhD¹⁰^*, Grace Choi, BS¹⁰^*, Eileen Nicoletti, MD¹⁰^*, Juan A Bueren, PhD^2,3,4^*, Gayatri R Rao, MD, JD¹⁰^*, Jonathan D Schwartz, MD¹⁰ and José C Segovia, PhD^2,3,4^*

¹Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
²Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), Madrid, Spain
³Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
⁴Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain
⁵Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
⁶Center for Definitive and Curative Medicine, Stanford University, Stanford, CA
⁷Stanford Children's Hospital, Palo Alto, CA
⁸Hospital Infantil Universitario Niño Jesus, Madrid, Spain
⁹Department of Pediatrics, Division of Hematology/Oncology, Stanford University School of Medicine, Stanford, CA
¹⁰Rocket Pharmaceuticals, Inc., New York, NY

2447

Multiplex CRISPR/Cas9 Genome Editing Targeting the BCL11A/HBG Axis Maximizes Fetal Hemoglobin Reinduction but Generates Chromosomal Translocations Which Persist In Vivo

Clare Samuelson¹^*, Olivier Humbert, PhD¹^*, Stefan Radtke, PhD¹, Haiying Zhu²^*, Mallory J Llewellyn, BS¹^*, Emily Fields, BS¹^*, Savannah Cook, BS¹^*, Meei-Li W Huang²^*, Olivier Negre, PhD³^*, Keith Jerome, MD PhD^1,2^* and Hans-Peter Kiem, MD PhD^1,2

¹Fred Hutchinson Cancer Research Center, Seattle, WA
²University of Washington, Seattle, WA
³bluebird bio, inc., Cambridge, MA

2448

Highly Efficient Gene Editing of Human Hematopoietic Stem Cells to Treat X-Linked Sideroblastic Anemia

Riguo Fang, PhD¹^*, Yingchi Zhang, PhD²^*, Pengfei Yuan, PhD¹^*, Huihui Yang, Master¹^*, Lingling Yu, Bachelor¹^*, Yongjian Zhang, PhD¹^*, Jia Shi, PhD³^*, Guangzhen Ji, PhD²^*, Jingliao Zhang, MD²^*, Wei Qi, Master⁴^*, Xiaofan Zhu, MD², Dong Wei, PhD¹^* and Tao Cheng⁵

¹EdiGene Inc., Beijing, 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
³EdiGene (Guangzhou) Inc., Guangzhou, China
⁴Novogene Co, Ltd, Beijing, China
⁵State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College, Tianjin, China

2449

Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events

Martin Rodriguez, BSc¹, Brady Trevisan¹^*, Sunil George, PhD¹^*, Ritu M Ramamurthy¹^*, Andrew Rabah¹^*, Jordan E Shields, MS²^*, Denise Schwahn, DVM³^*, Jorge Figueroa, MD⁴^*, Diane Meares⁵^*, John Owen, MD, MBA, FRCPC⁵, Michael Gautreaux, Ph.D., D.ABHI⁶^*, Anthony Atala, M.D.¹^*, Christopher B Doering, PhD^7,8,9, H. Trent Spencer, Ph.D.^2,9,10^*, Christopher D Porada, PhD¹^* and M. Graca Almeida-Porada, MD, PhD¹

¹Fetal Research and Therapy Program, Wake Forest Institute for Regenerative Medicine, Winston Salem, NC
²Aflac Cancer and Blood Disorders Center, Emory University/Children's Healthcare of Atlanta, Atlanta, GA
³Zoetis Veterinary Medical Research and Development, Kalamazoo
⁴Center for Research in Obstetrics and Gynecology, Wake Forest School of Medicine, Winston Salem
⁵Section on Hematology and Oncology, Wake Forest School of Medicine, Winston Salem, NC
⁶HLA/Immunogenetics and Immunodiagnostics Laboratories, Wake Forest School of Medicine, Winston Salem
⁷Department of Pediatrics, Emory University, Atlanta, GA
⁸Department of Pediatrics, Emory University Emory Childrens Center, Atlanta, GA
⁹Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA
¹⁰Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

2450

Platelet-Targeted Gene Therapy Induces Robust Immune Tolerance Even in a Primed Model Via Peripheral Clonal Deletion of CD4 and CD8 T Cells and Expansion of Treg Cells

Jing Li, MD, PhD^*, Juan Chen, PhD^*, Jocelyn A. Schroeder^* and Qizhen Shi, MD, PhD

Medical College of Wisconsin, Blood Research Institute, Children's Research Institute, Milwaukee, WI

2451

Cross Priming of Transgene Product-Specific CD8⁺ T Cells in Hepatic AAV Gene Transfer Depends on IL-1 Receptor and XCR1⁺ Dendritic Cells but Not TLR9

Sandeep Kumar, PhD¹^*, Moanaro Biswas, PhD¹, Annie R Pineros, PhD¹^*, Ype P De Jong, MD²^* and Roland W Herzog, PhD¹

¹Pediatrics, Indiana University School of Medicine, Indianapolis, IN
²2Division of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY

2452

A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing

Lisa Spronk¹^*, Richard Logtenstein¹^*, Martin de Haan¹^*, Jaap Twisk²^*, Valerie Ferreira¹^*, Sander van Deventer¹^* and Liesbeth Heijink¹^*

¹uniQure Biopharma BV, Amsterdam, Netherlands
²uniQure biopharma B.V, Amsterdam, Netherlands

2453

AAV Vector Dose Determines TLR9 Dependence of CD8+ T Cell Response to Transgene Product

Ning Li¹^*, Thais Bertolini¹^* and Roland W Herzog, PhD²

¹Herman B Wells Center for Pediatric Research, IUPUI, Indianapolis, IN
²Pediatrics, Indiana University School of Medicine, Indianapolis, IN

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^*signifies non-member of ASH