-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

801.Oral1.6 801. Gene Therapy and Transfer: Immune Disorders, CAR-T Cells, and B-hemoglobinopathies Clinically Relevant Abstract

Gene Therapy and Transfer Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Biological, sickle cell disease, Diseases, Therapies, thalassemia, CAR-Ts, Hemoglobinopathies, immunodeficiency, Immune Disorders, Clinically relevant, gene therapy, stem cells
Sunday, December 10, 2017: 4:30 PM-6:00 PM
Bldg C, Lvl 1, C101 Aud (Georgia World Congress Center)
Moderators:
Shannon L Maude, MD, PhD, Children's Hospital of Philadelphia and Jennifer L Gori, PhD, Editas Medicine
Disclosures:
Maude: Novartis Pharmaceuticals: Consultancy, Other: Medical Advisory Boards. Gori: Editas Medicine: Employment.
4:30 PM

Ewelina Mamcarz, MD1, Sheng Zhou, PhD2, Timothy Lockey, Ph.D.3*, Hossam Abdelsamed, Ph.D.4*, Shane Cross, Pharm D5*, Zhijun Ma, M.D.6*, Jola Dowdy7*, Brandon Triplett, MD8*, Mitchell J. Weiss, MD, PhD9, Byoung Y. Ryu, PhD7,10, Elif Dokmeci, MD11*, Juan Carlos Aldave, MD12*, Suk See De Ravin, MD, PhD13*, Benjamin Youngblood, Ph.D.4*, Janel R Long-Boyle, PharmD, PhD14*, Michael M Meagher, Ph.D.15*, Harry L Malech, M.D.16, Jennifer Puck, MD17*, Morton J Cowan, MD14* and Brian Sorrentino, MD18

1Department of Bone Marrow Transplantation and Cellular Therapy, Children's National Medical Center, Memphis, TN
2Department of Hematology, St. Jude Children's Rsch. Hosp. D2039, Memphis, TN
3Department of Therapeutics Production & Quality, St. Jude Children's Research Hospital, Memphis, TN
4Department of Immunology, St. Jude Children's Research Hospital, Memphis, TN
5Department of Pharmaceutical Services, St. Jude Children's Research Hospital, Memphis, TN
6Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
7St. Jude Children's Research Hospital, Memphis, TN
8Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN
9Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
10Department of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN
11Department of Pediatrics, Section of Pediatric Allergy and Immunology, Pediatric Primary Immunodeficiency Center, University of New Mexico, Albuquerque, NM
12Division of Allergy and Clinical Immunology, Hospital Nacional Edgardo Rebagliati Martins, Lima, Peru
13Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
14Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco, Benioff Children’s Hospital, San Francisco, CA
15Department of Therapeutics Production & Quaility, St. Jude Children's Research Hospital, Memphis, TN
16National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
17UCSF Department of Pediatrics, UCSF School of Medicine, San Francisco, CA
18Department of Hematology, St. Jude Children's Res. Hosp. Experimental Hematology, Memphis, TN

4:45 PM

Jennifer Brudno, MD1, Norris Lam, BS2*, Michael Wang, MD3, David Stroncek, MD4, Irina Maric5*, Maryalice Stetler-Stevenson6*, Constance Yuan6*, Steven Hartman7*, Jennifer A. Kanakry, MD2*, Steven Z. Pavletic, MD8, Steven A. Feldman9*, Judith Lawrence10*, Rashmika Patel7*, Brenna Hansen7*, Ronald E. Gress, MD8 and James N. Kochenderfer, MD11

1Experimental Transplantation and Immunology Branch, NIH, NCI, Bethesda, MD
2Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD
3U.T. M.D. Anderson Cancer Center, Houston, TX
4Cell Processing Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MD
5Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD
6National Cancer Institute/ Laboratory of Pathology, National Institutes of Health, Bethesda, MD
7NIH, NCI, Bethesda
8Experimental Transplantation and Immunology Branch, Center for Cancer Research (CCR), National Cancer Institute (NCI), National Institutes of Health (NIH), Bethesda, MD
9National Cancer Institute, National Institutes of Health, Bethesda, MD
10Experimental Transplantation and Immunology Branch, NIH, NCI, Bethesda
11Experimental Transplantation and Immunology Branch, NCI, Bethesda, MD

4:59 PM

Partow Kebriaei, MD1, Helen Huls2*, Susan Lynn Neel, RN, OCN3*, Simon Olivares4*, Aaron F Orozco5*, Shihuang Su5*, Sourindra N Maiti5*, Amy Smith6*, Elenanor De Groot7*, Hagop M. Kantarjian, MD8, Maro Ohanian, DO9*, Katy Rezvani, MD, PhD10, Elizabeth J. Shpall, MD11, Richard E Champlin, MD11, Laurence J N Cooper, MD, PhD12 and Harjeet Singh, MSc, PhD5*

1Stem Cell Transplantation and Cellular Therapy, M.D. Anderson Cancer Center, Houston, TX
2Formerly Intrexon, Houston, TX
3Stem Cell Transplantation and Cellular Therapy, University of Texas MD Anderson Cancer Center, Houston, TX
4Pediatrics Research, University of Texas MD Anderson Cancer Center, HOUSTON, TX
5Pediatrics Research, University of Texas MD Anderson Cancer Center, Houston, TX
6Ziophram, Houston
7Ziopharm, Houston, TX
8Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX
9Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
10Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
11Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
12Ziopharm Oncology, Boston, MA

5:14 PM

Mark C. Walters, MD1, Suradej Hongeng, MD2, Janet L. Kwiatkowski, MD3, Franco Locatelli, MD, PhD4, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD5*, Martin G. Sauer6, Adrian J Thrasher, MD, PhD, FMedSci7*, Isabelle Thuret, MD8*, Evangelia Yannaki, MD9*, Heidi Elliot10*, Marisa Gayron10*, Mohammed Asmal, MD, PhD10* and Alexis A Thompson, MD, MPH11

1UCSF Benioff Children’s Hospital, Oakland, CA
2Ramathibodi Hospital Mahidol University, Bangkok, THA
3Children's Hospital of Philadelphia, Philadelphia, PA
4Department of Hematology and Oncology, IRCCS Bambino Gesù Children's Hospital, Rome, Italy
5Research Haematology Department, University College London, London, United Kingdom
6Pediatric Hematology and Oncology, Medizinische Hochschule Hannover, Hannover, Germany
7UCL Great Ormond Street Institute of Child Health, London, United Kingdom
8Hemato-pediatrics, La Timone Hospital, Marseille, France
9Gene and Cell Therapy Center, Hematology-HCT Unit, G. Papanikolaou General Hospital, Thessaloniki, GRC
10bluebird bio, Inc., Cambridge, MA
11Division of Hematology, Oncology & Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

5:29 PM

Julie Kanter, MD1, Mark C. Walters, MD2, Matthew Hsieh, MD3, Lakshmanan Krishnamurti, MD4, Janet L. Kwiatkowski, MD5, Rammurti Kamble, MD6, Christof von Kalle, MD7*, Marcelyne Joseney-Antoine8*, Francis J. Pierciey Jr.8*, Weiliang Shi, PhD8*, Mohammed Asmal, MD, PhD8*, Alexis A Thompson, MD, MPH9 and John F Tisdale, MD3

1Lifespan Comprehensive Sickle Cell Center, Medical University Of South Carolina, Charleston, SC
2UCSF Benioff Children’s Hospital, Oakland, CA
3MCHB, NHLBI/NIDDK, National Institutes of Health, Bethesda, MD
4Department of Pediatrics, Division of Hematology/Oncology/BMT, Emory University School of Medicine, Atlanta, GA
5Children's Hospital of Philadelphia, Philadelphia, PA
6Baylor College of Medicine Methodist Hospital, Houston, TX
7German Cancer Research Center (DKFZ), National Center for Tumor Diseases (NCT), Heidelberg, Germany
8bluebird bio, Inc., Cambridge, MA
9Division of Hematology, Oncology & Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL

5:44 PM

Katelyn E Masiuk, BS, Jennifer Laborada, BS*, Roger P Hollis, PhD* and Donald B Kohn, MD

Department of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, CA

*signifies non-member of ASH