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

Oral and Poster Abstracts
Oral

Adult, Anemias, apheresis, Biological, Hemophilia, HSCs, sickle cell disease, bioengineering, Bleeding and Clotting, Diseases, Bone Marrow Failure, Therapies, Fanconi Anemia, Genetic Disorders, Pediatric, red blood cells, Biological Processes, Hemoglobinopathies, DNA repair, gene therapy, Technology and Procedures, Cell Lineage, erythropoiesis, gene editing, Study Population, Clinically relevant, hematopoiesis, transplantation, stem cells

Moderators:

Akshay Sharma, MBBS, St. Jude Children's Research Hospital and Donald B. Kohn, MD, University of California - Los Angeles, Department of Pediatrics

Disclosures:

Sharma: Spotlight Therapeutics: Consultancy; CRISPR Therapeutics: Other: Clinical Trial PI, Research Funding; Novartis: Other: Clinical Trial PI, Research Funding; Vertex Pharmaceuticals: Other: Clinical Trial PI, Research Funding.

11:30 AM

671

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

Andrew D. Leavitt, MD¹, Barbara A. Konkle, MD^2,3, Kimo Stine, MD⁴^*, Nathan Visweshwar, MD⁵^*, Thomas J. Harrington, MD⁶^*, Adam Giermasz, MD, PhD⁷, Steven Arkin, MD⁸, Annie Fang, MD, PhD⁹^*, Frank Plonski, RN, MA⁸^*, Lynne Smith, MBA¹⁰^*, Li-Jung Tseng, PhD, MBA⁹^*, Gregory Di Russo, MD⁸^*, Bettina M Cockroft, MD¹¹^*, Jeremy Rupon, MD¹⁰ and Didier Rouy, MD, PhD¹¹^*

¹University of California, San Francisco, CA
²University of Washington, Seattle, WA
³University of Washington, Washington Center for Bleeding Disorders, Seattle, WA
⁴UAMS at Arkansas Children's Hospital, Little Rock, AR
⁵University of South Florida, Tampa, FL
⁶University of Miami Miller School of Medicine, Miami, FL
⁷University of California Davis, Sacramento, CA
⁸Pfizer Inc., Cambridge, MA
⁹Pfizer Inc., New York, NY
¹⁰Pfizer Inc., Collegeville, PA
¹¹Sangamo Therapeutics, Brisbane, CA

11:45 AM

672

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Annette von Drygalski, MD, Pharm D¹, Adam Giermasz, MD, PhD², Giancarlo Castaman, MD³^*, Nigel S. Key, MD^4,5, Susan U. Lattimore, R.N.⁶^*, Frank W.G. Leebeek, MD, PhD⁷, Wolfgang A. Miesbach, MD⁸^*, Michael Recht, MD, PhD⁹, Esteban Gomez, MD¹⁰, Robert Gut, MD, PhD¹¹^* and Steven W. Pipe, MD¹²

¹Department of Medicine, Division of Hematology/Oncology, UC SAN DIEGO HEALTH, SAN DIEGO, CA
²University of California Davis, Sacramento, CA
³Azienda Ospedaliera Universitaria Careggi, Florence, Italy
⁴University of North Carolina at Chapel Hill, Chapel Hill, NC
⁵Division of Hematology, University of North Carolina at Chapel Hill, Chapel Hill, NC
⁶Oregon Health & Science University, Portland
⁷Erasmus University Medical Center, Rotterdam, Netherlands
⁸Hemophilia Centre, University Hospital Frankfurt, Frankfurt, Germany
⁹Oregon Health and Science University, Portland, OR
¹⁰Phoenix Children's Hospital, Phoenix, AZ
¹¹uniQure Inc, Lexington, MA
¹²University of Michigan, Ann Arbor, MI

12:00 PM

673

Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells

Jonathan S Yen, PhD¹, Gregory A. Newby, PhD²^*, Thiyagaraj Mayuranathan¹^*, Shaina N. Porter, PhD³^*, Yu Yao, MD¹^*, Kaitly J. Woodard, BS¹, Kalin Mayberry, BS¹^*, Kelcee Everette²^*, Jingjing Zhang, MS¹^*, Jordana M Henderson, MS⁴^*, Anton P Mccaffrey⁴^*, Shondra M. preutt-Miller, PhD³^*, John F. Tisdale, MD⁵, Shengdar Q. Tsai, PhD¹, David R. Liu, PhD^6,7,8^* and Mitchell J. Weiss, MD, PhD¹

¹Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
²Broad Institute of MIT and Harvard, Cambridge, MA
³Center for Advanced Genome Engineering (CAGE), St. Jude Children's Research Hospital, Memphis, TN
⁴Trilink Biotech, San Diego, CA
⁵Cellular and Molecular Therapeutics Branch, NHLBI, National Heart, Lung, and Blood Institute, Bethesda, MD
⁶Howard Hughes Medical Institute, Harvard University, Cambridge, MA
⁷Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA
⁸Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA

12:15 PM

674

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MD, PhD^1,2,3, Rajni Agarwal, MD^1,2,3, Julián Sevilla, MD, PhD⁴^*, Paula Río, PhD^5,6,7^*, Susana Navarro, PhD^5,6,7^*, Brian C Beard, PhD⁸^*, Kenneth M Law, PhD⁸^*, Grace Choi, BS⁸^*, Miriam Zeini, PhD⁸^*, Eileen Nicoletti, MD⁸^*, John E Wagner, MD⁹, Claire Booth, MBBS, PhD, MSc¹⁰^*, Jonathan D Schwartz, MD⁸, Juan A Bueren, PhD^5,6,7^* and Maria Grazia Roncarolo, MD^1,2,3

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

12:30 PM

675

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD¹, Gayatri R Rao, MD, JD²^*, Elena Almarza, PhD^2,3,4^*, Dayna Terrazas, RN⁵^*, Eileen Nicoletti, MD²^*, Augustine Fernandes, PhD⁵^*, Caroline Kuo, MD⁶^*, Satiro N. De Oliveira, MD⁷, Theodore B. Moore, MD⁸, Kenneth M Law, PhD²^*, Brian C Beard, PhD²^*, Julian Sevilla, MD, PhD⁹^*, Cristina Mesa-Núñez, PhD¹⁰^*, Juan A Bueren, PhD¹¹^* and Jonathan D Schwartz, MD²

¹Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics l Molecular & Medical Pharmacology, University of California – Los Angeles, Los Angeles, CA
²Rocket Pharmaceuticals, Inc., New York, NY
³Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, NY
⁴Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), Madrid, NY
⁵University of California, Los Angeles, Los Angeles, CA
⁶Dept. of Allergy and Immunology, UCLA Medical Center, Los Angeles, CA
⁷Dept. of Pediatrics, UCLA, Los Angeles, CA
⁸UCLA Division of Pediatric Hematology, University of California, Los Angeles, Los Angeles, CA
⁹The Departments of Pediatric Hematology–Oncology and Hematology and Oncology, Fundación para la Investigación Biomédica, Hospital Infantil Universitario Niño Jesús, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain
¹⁰Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain
¹¹Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Valencia, Spain

12:45 PM

676

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress - NCT04201405

Jane L Kinsella, MBChB¹^*, Robert F Wynn, MRCP, MD, FRCPath², Brian Bigger, PhD³^*, Adrian J. Thrasher, MBBS, PhD, FMedSci⁴^*, Claire Booth, MBBS, PhD, MSc⁵^*, Karen Buckland, PhD⁵^*, Natalia Izotova⁴^*, Stewart Rust, B.Sc. (Hons), D.Clin.Psych, Pg.Dip., C.Psychol., AfBPS.²^*, Daniel Weisberg, BSc, PhD, PGCert, ClinPsyD²^*, Heather J Church, PhD⁶^*, Kathryn L Brammeier⁷^*, Karen L Tylee⁷^*, Ceri Jones, BSc⁷^*, Helena Lee⁸^*, Laura Ford⁸^*, Atusa Sadegholnejat⁸^*, Stuart Ellison⁹^* and Simon A Jones, MBChB, BSc, MRCPCH⁷^*

¹Paediatric Bone Marrow Transplant, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom
²Royal Manchester Children's Hospital, Manchester, United Kingdom
³Stem Cell and Neurotherapies, University of Manchester, Manchester, United Kingdom
⁴UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁵University College London/Great Ormond Street Hospital, London, United Kingdom
⁶Willink Biochemical Genetics Unit, Manchester Foundation Trust, Manchester, United Kingdom
⁷Willink Biochemical Genetics Unit, Manchester NHS Foundation Trust, Manchester, United Kingdom
⁸Transplantation Laboratory, Manchester NHS Foundation Trust, Manchester, United Kingdom
⁹Stem Cell and Neurotherapies Laboratory, University of Manchester, Manchester, United Kingdom

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^*signifies non-member of ASH

801.O1.6 801. Gene Editing, Therapy and Transfer II