Final Program - American Society of Gene & Cell Therapy

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Program Schedule, Thursday, May 16, 2013 Carl H. June, MD T Cell Mediated Gene Transfer for Cancer Therapy The adoptive transfer of anti-tumor T cells can mediate regression of established metastatic cancers in patients. We have begun a series of clinical trials using the transduction of genes encoding chimeric antigen receptors (CARs) or anti-tumor T cell receptors (TCR) into normal peripheral lymphocytes for use in adoptive cell transfer. Anti-tumor TCRs have been identiied that recognize cancer testis antigens. Autologous cells transduced with genes encoding the anti-NY-ESO-1 TCR cancer-testes antigen mediated objective clinical responses in patients with myeloma. An afinity enhanced MAGE A3 TCR caused severe adverse events with fatal off-target cardiac toxicity, pointing to serious concerns with the ability of pre-clinical testing to identify off target effects of TCRs. Patients with acute pre-B cell leukemia or chronic lymphocytic leukemia exhibited a high objective regression rate using T cells transduced with a CD19 CAR. The cellular and molecular mechanisms of T cell destruction of cancers are under active investigation. Ongoing studies are evaluating chimeric antibody receptors targeting mesothelin, and EGFRvIII. Together these studies demonstrate the power and potential of adoptive T cell immunotherapy to mediate the regression of established advanced cancer in humans. Lieping Chen, MD, PhD New Perspectives in the Development of Cancer Vaccines There is ample evidence that human malignancies express tumor-associated or tumor-speciic antigens. Immunization with cancer antigen-based vaccine in animal models or patients with advanced cancer induce tumor-reactive T cells in lymphoid organs and these T cells can be detected in blood. However, increased circulating tumor-reactive T cells often do not correlate with regression of advanced cancer. Recent studies reveal that speciic resistant mechanisms are developed in the tumor microenvironment to suppress activity of tumor-iniltrating T cells. It is thus important to understand and identify molecules underlying these resistance mechanisms. I will focus my discussion on analysis of mechanisms in the tumor microenvironment in the context of improvement of the eficacy of cancer vaccines. Thursday, May 16, 2013 Final Program SALT LAKE CITY, UTAH May 15–18, 2013 55
Program Schedule, Thursday, May 16, 2013 Foundation Symposium 241 6:00 pm - 8:00 pm ROOM: BALLROOM A Association Francaise Contre les Myopathies/Muscular Dystrophy Association Symposium Jointly planned by the Association Francaise contre les Myopathies and the Muscular Dystrophy Association. CO-CHAIRS: Charles A. Gersbach, PhD and Kathryn R. Wagner, MD, PhD SPEAKERS Thursday, May 16, 2013 Jeffrey Chamberlain, PhD Lentiviral Modiication of Myogenic Stem Cells Over the past decade adeno-associated viral (AAV) vectors have merged as a potential therapeutic for muscle disorders. While AAV vectors can eficiently transduce post-mitotic myoibers, they display low tropism for myogenic stem cells and are rapidly lost during cell proliferation due to minimal genomic integration. In contrast, lentiviral vectors can stably transduce myogenic stem cells both in vitro and in vivo, enabling expansion and transplantation of genetically modiied cells for therapeutic interventions. This lecture will review approaches for modifying myogenic stem cells for in vivo and ex vivo gene therapy of myogenic disorders such as the muscular dystrophies. Xiao Xiao, PhD Regional Limb Vein Perfusion in Muscular Dystrophy Dogs and Human Patients Fulvio Mavilio, PhD Gene-based Therapies for Neuromuscular Disorders Most inherited neuromuscular diseases have no therapy, and collectively represent a signiicant unmet medical need. Genethon and its collaborating clinical centers have launched an ambitious program aimed at developing gene therapy approaches for at least three of these diseases (Duchenne muscular dystrophy, myotubular myopathy and spinal muscular atrophy) based on systemic or loco-regional delivery of AAV vectors. We will provide an overview of the program and of the scientiic, regulatory and clinical challenges associated with its development. Anna Buj Bello, MD, PhD Myotubularin Deicie ncy Myotubular myopathy (XLMTM) is a very severe congenital muscle disorder due to mutations in the MTM1 gene. An overview about the disease, animal models and current experimental therapies, in particular our results on intravenous AAV-mediated gene replacement therapy, will be presented. Outstanding Achievement Award Lecture 300 56 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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ASGCT Officers and Board of Directo
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Annual Meeting Exhibitors - as of A
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Abstract Directory, Saturday, May 1
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Abstract Author Index Aartsma-Rus,
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Abstract Author Index Bartel, Ronnd
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Abstract Author Index Bridges, Char
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Abstract Author Index Chicoine, Lou
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Abstract Author Index Yanovich, Sau
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Final Program - American Society of Gene & Cell Therapy

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