57th ASH Annual Meeting and Exposition (December 5-8, 2015): 801. Gene Therapy and Transfer: Poster II

Gene Therapy and Transfer Oral and Poster Abstracts
Poster

Hall A, Level 2 (Orange County Convention Center)

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Julie Kanter, MD¹, Mark C. Walters, MD², Matthew Hsieh, MD³, Alexis A. Thompson, MD⁴, Lakshmanan Krishnamurti, MD⁵, Janet Kwiatkowski, MD⁶, Rammurti T Kamble, MD⁷, Christof von Kalle, MD⁸^*, Frans A. Kuypers, Ph.D.⁹, Marina Cavazzana, MD, PhD¹⁰, Philippe Leboulch, MD^11,12,13^*, Laura Sandler, MPH¹⁴^*, Sandeep Soni, MD¹⁴ and John F. Tisdale, MD³

¹Medical University of South Carolina, Charleston, SC
²UCSF Benioff Children's Hospital, Oakland, CA
³National Institutes of Health, Bethesda, MD
⁴Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Chicago, IL
⁵Emory University, Atlanta, GA
⁶Children's Hospital of Philadelphia, Philadelphia, PA
⁷Center for Cell and Gene Therapy, Baylor College of Medicine and Houston Methodist Hospital, Houston, TX
⁸National Center for Tumor Diseases (NCT), German Cancer Research Center (DKFZ), Heidelberg, Germany
⁹Children's Hospital Oakland Research Institute, Oakland, CA
¹⁰Biotherapy Department, Hôpital Universitaire Necker -Enfants Malades, Paris, France
¹¹Brigham & Women’s Hospital and Harvard Medical School, Boston, MA
¹²Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
¹³CEA (iMETI) and University of Paris-Sud, Fontenay-aux-Roses, France
¹⁴bluebird bio, Inc., Cambridge, MA

3234

Clonal Analysis of Human Bone Marrow CD34+ Cells Edited By BCL11A-Targeting Zinc Finger Nucleases Reveals Clinically Relevant Levels of Fetal Globin Expression in Edited Erythroid Progeny

Kai-Hsin Chang, PhD¹^*, Timothy Sullivan, MS¹^*, Mei Liu, BS¹^*, Xiao Yang, PhD¹^*, Chao Sun, PhD¹^*, Benjamin Vieira, BS¹^*, Ming Zhang, PhD²^*, Vu P. Hong, PhD²^*, Kai Chen, MS¹^*, Sarah Smith, BS¹^*, Siyuan Tan, PhD¹^*, Andreas Reik, PhD³^*, Fyodor D Urnov, PhD³^*, Edward J. Rebar, PhD³^*, Olivier Danos, PhD¹^* and Haiyan Jiang, PhD¹

¹Biogen, Cambridge, MA
²Hematology Research, Biogen, Cambridge, MA
³Sangamo BioSciences, Richmond, CA

3235

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

Alessandra Magnani, MD, PhD^1,2^*, Cécile Roudaut, MSc¹^*, Aurelie Gabrion¹^*, Laure Caccavelli, PhD¹^*, Fabien Touzot, MD, PhD^1,2^*, Benedicte Neven, MD, PhD³^*, Despina Moshous, MD³^*, Nizar Mahlaoui⁴^*, Emmanuel Clave⁵^*, Antoine Toubert⁵^*, Alain Fischer, MD, PhD⁶^* and Marina Cavazzana, MD, PhD^1,2,7

¹Biotherapy Department, Hôpital Universitaire Necker -Enfants Malades, Paris, France
²IMAGINE Institute, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
³Pediatric Hematology-Immunology and Rhumatology Department, University Hospital Necker-Enfants Malades, Paris, France
⁴Hôpital Universitaire Necker -Enfants Malades, Paris Cedex 15, France
⁵INSERM UMR 1160, Hopital Saint-Louis - APHP, Paris, France
⁶Service d'Immunologie et Hematologie Pediatrique, CEREDIH, Université Paris Descartes, AP-HP Hopital Necker Enfants-Malades, Paris, France
⁷INSERM-APHP, CIC Biothérapie GHU Ouest, Paris, France

3236

Maintenance of Leukocyte Telomere Length after Transplant and Chemoselection in Macaques with Polyclonal Gene Modified Cell Engraftment

Jennifer E Adair, PhD^1,2^*, Christopher R Burtner, PhD¹^* and Hans-Peter Kiem, MD^1,2,3

¹Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
²University of Washington, Seattle, WA
³Department of Pathology, University of Washington School of Medicine, Seattle

3237

Human Induced Pluripotent Stem Cells Incorporating Safe Harbor Loci Integrated Inducible Suicide Systems for Use in the Application of Cellular Therapeutics

Xiaosong Huang, PhD^*, Weijie Lan, PhD^*, Philippe Parone, PhD^*, Raedun Clarke, PhD^*, Ramzey Abujarour, PhD^*, Megan Robinson, BS^*, Miguel Meza, MS^*, Sarah Raynel, MS^*, Betsy Rezner, BS^*, David Robbins, BS^*, Tom Tong Lee, PhD^*, Daniel Shoemaker, PhD^* and Bahram Valamehr, PhD, MBA^*

Fate Therapeutics, San Diego, CA

3238

Clotting Factor VIII Overexpression Shows Signs of ER Stress but Does Not Cause Toxicity upon Gene Transfer

Irene Zolotukhin, MS^*, Brett Palaschak, BA^*, David M Markusic, PhD and Roland Herzog, PhD

Pediatrics/CMT, University of Florida, Gainesville, FL

3239

Human Factor VIII Expression and Normalization of Bleeding Following AAV Gene Therapy in a Double Knockout Mouse Model of Hemophilia

Stuart Bunting, PhD^*, Lening Zhang, PhD^*, Lin Xie, BS^*, Sherry Bullens, AB^*, Rajeev Mahimkar, PhD^*, Sylvia Fong, PhD^*, Krystal Sandza, BS^*, Peter Colosi, PhD^*, Shinong Long, PhD^*, Gordon Allen Vehar, PhD^* and Barrie Carter, PhD^*

Development Sciences, BioMarin Pharmaceutical, Novato, CA

3240

CRISPR/Cas9 Mediated Gene Repair in Inherited Bleeding Disorders

Satoshi Morishige¹^*, Hidetoshi Ozawa¹^*, Shinichi Mizuno^1,2^*, Satoko Koteda¹^*, Kuniki Kawaguchi¹^*, Takayuki Nakamura¹^*, Kei Nomura¹^*, Harumi Takeda¹^*, Yuka Takata¹^*, Ritsuko Seki¹, Fumihiko Mouri¹^*, Koichi Osaki¹^*, Michitoshi Hashiguchi^1,3^*, Koji Yoshimoto¹^*, Takashi Okamura^1,4 and Koji Nagafuji¹

¹Division of Hematology and Oncology, Department of Medicine, Kurume University School of Medicine, Kurume, Japan
²Center for Advanced Medical Innovation, Kyushu University, Fukuoka, Japan
³Department of Hematology, St. Mary's Hospital, Kurume, Japan
⁴Center for Hematology and Oncology, St. Mary's Hospital, Kurume, Japan

3241

Hepatic AAV Gene Transfer of Cytoplasmic Transgene Induces Transgene Product-Specific T Cell Activation Initially in the Liver and Celiac Lymph Node Preceding Treg Induction

Roland W. Herzog, PhD¹ and George Q. Perrin, PhD²^*

¹Pediatrics, University of Florida, Gainesville, FL
²Dept. of Pediatrics, University of Florida, Gainesville, FL

3242

Mitochondrial Biomass As a Measure of Fitness for T Cells Expressing Chimeric Antigen Receptors

Bipulendu Jena, PhD¹^*, David Rushworth, MD, PhD²^*, George T McNamara, PhD³^* and Laurence JN Cooper, MD, PhD^1,4

¹Pediatrics-Research, University of Texas MD Anderson Cancer Center, Houston, TX
²Department of Neurology, Baylor College of Medicine, Houston, TX
³Pediatrics Research, University of Texas MD Anderson Cancer Center, Houston, TX
⁴Ziopharm Oncology Inc., Boston, MA

3243

Characterization of Lentiviral Vector Derived Anti-Bcma CAR T Cells Reveals Key Parameters for Robust Manufacturing of Cell-Based Gene Therapies for Multiple Myeloma

Graham Lilley, M.S.¹^*, Alden Ladd¹^*, Daniel Cossette¹^*, Laura Viggiano¹^*, Gregory Hopkins¹^*, John W. Evans, PhD¹^*, Mingli Li, M.S.¹^*, Howard J. Latimer IV, BS¹^*, Sara M. Miller²^*, Michael Kuczewski, MS¹^*, Wendy Bakeman¹^*, Kayla MacLeod¹^*, Kevin M. Friedman, PhD¹^*, Dawn A Maier, M.S.¹^*, Michael Paglia, M.S.³^*, Richard A. Morgan, PhD¹^* and Mark D Angelino, Ph.D.¹^*

¹bluebird bio, Inc, Cambridge, MA
²bluebird bio, Cambridge, MA
³bluebird bio, Inc, Cambridge

3244

Expanding the Ligand Binding Repertoire of Chimeric Antigen Receptors Using Lamprey Variable Lymphocyte Receptors

Robert Moot, BS¹^*, Sunil Raikar, MD², Lauren C Fleischer, BS³^*, David McCarty²^*, Melissa Querrey²^*, Christopher B Doering, PhD⁴ and H. Trent Spencer, PhD²

¹Department of Pediatrics, Aflac Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA
²Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA
³Graduate Program in Molecular and Systems Pharmacology, Emory University, Atlanta, GA
⁴Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

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^*signifies non-member of ASH