FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 29 SCIENTIFIC PROGRAM WEDNESDAY, JUNE 1, 2005 Education Session 124 Bioavailability and Pharmacokinetics Pharmacokinetic analysis translates the disposition characteristics of macromolecules into quantitative parameters that can be used for direct comparison of medicinal agents as well as with physiological parameters such as blood flow and the rate of cellular endocytosis. Careful collection and experimental analysis of biological samples as well as theoretical development of appropriate mathematical models are necessary for accurate prediction of drug disposition and comparison of treatment regimens. This session, designed for the bench scientist and the clinician alike, will discuss basic pharmacokinetic concepts, methods for modeling, study design, data analysis, population kinetics and their application to gene delivery systems. Room: 265/266 Chair: Maria A. Croyle, PhD Speakers: Maria A. Croyle, PhD - Pharmacokinetics Made Easy Sally Choe, PhD - Bridging Preclinical and Clinical Pharmacokinetics in Gene Therapy Education Session 125 Gene-Based Vaccines This session will give the fundamentals as well as updates about Gene-based vaccines. The technology, immune mechanisms, and second generation advances describing new delivery systems and the use of adjuvants will be presented. An overview of clinical and research applications will be described. Room: 120/124 Chair: Margaret A. Liu, MD Speakers: Margaret A. Liu, MD - Gene-based Vaccines: An Overview Jeffrey Ulmer, PhD - Toward the Development of an Effective DNA Vaccine for Humans Hildegund CJ Ertl, MD - Adaptive Immune Responses to Viral Vectors THURSDAY, JUNE 2, 2005 GEORGE STAMATOYANNOPOULOS LECTURE 200 8:00 am - 9:00 am Room: America’s Ballroom Chair: Mark A. Kay, MD, PhD Speaker: Thomas Tuschl, PhD - Mechanisms of siRNA- and miRNA-guided Gene Regulation in Mammals Presentation of Excellence in Research Awards BREAK – 9:00 AM - 9:15 AM WORKSHOPS – 9:15 AM - 10:45 AM Workshop 210 Cancer: Optimization of T-Cell Gene Therapy Room: 123/127 Chair: Laurence JN Cooper, MD, PhD Speakers: John C. Morris, MD - Role of Interleutem-15 in T cell Development and Function Laurence JN Cooper, MD, PhD - Redirected Specifity of T cells - Improving Their Potential Steven M. Albelda, MD - Augmenting T-cell Trafficking to Tumors Bruce L. Levine, PhD - Cancer: Optimization of T-Cell Gene Therapy Francesco M. Marincola, MD - Gene Profiling of Immune Responses Against Tumors Carolyn Wilson, PhD - T-cell Gene Therapy: Regulatory Issues
30 8th Annual Meeting • June 1 - 5, 2005 • St. Louis SCIENTIFIC PROGRAM THURSDAY, JUNE 2, 2005 Workshop 211 Gene-Based Vaccines: New Developments for Genetic Vaccines Room: 120/124 Chair: David B. Weiner, PhD Speakers: Richard Heller, PhD - Electroporation Mediated Immunotherapy of Solid Tumors: Preclinical Results and Initiation of Clinical Study Leaf Huang, PhD - LPD Nanoparticles: A Gene Vector Turned into a Potent Vaccine Carrier David B. Weiner, PhD - Expanding the Potency and Breadth of the Immune Responses Induced by DNA Vaccines Britta Elisabeth Wahren, MD, PhD - Means to Induce Mucosal Immunity in HIV Genetic Immunization Ning-Sun Yang, PhD - Developing Multiple Viral Protein Genes and Virus-like Particles as Gene-based Vaccines Workshop 212 Hemopoietic: Hemopoietic Stem Cell Gene Therapy Room: Ferrara Theater Chair: Jan Nolta, PhD Speakers: Carolyn Wilson, PhD - Addressing Safety Concerns for Hematopoietic Stem Cell Gene Therapy - A FDA Perspective Manuel Grez, PhD - Correction of Chronic Granulomatous Disease by Gene Therapy Marina Cavazzana-Calvo, MD, PhD - Clinical and Biological Results from the SCID-XI Gene Therapy Trial Stanton L. Gerson, MD - In Vivo Stem Cell Selection without Myeloablation Alessandro Aiuti, MD, PhD - Gene Therapy of ADA-deficient SCID Workshop 213 Immunology of Gene Therapy: Organ Specific Immunity Room: 260/264 Chair: Roland W. Herzog, PhD Speakers: Pedro Lowenstein, MD, PhD - Immune Responses to Viral Vectors in the Central Nervous System John Iacomini, PhD - Reshaping the Immune System Using Gene Therapy Roland W. Herzog, PhD - In Vivo Gene Transfer to Liver and Muscle; Two Organs, Two Different Stories Hans-Peter Kiem, MD - Immune Responses to Gene-modified Hematopoietic Cells Workshop 214 Industrial Liaison: Pre-Clinical Issues in Gene Therapy Room: 263/267 Chair: Douglas J. Jolly, PhD Speakers: Rebecca Sheets, PhD - Pre-clinical Development of Adenovirus-vectored HIV Vaccine Candidate Joy Cavagnaro, PhD, DABT, RAC - Optimizing Design and Analysis of Preclinical Development Programs Boro Dropulic, PhD - Clinical Translation of a Lentiviral Vector for the Treatment of HIV/AIDS Peter K. Working, PhD, DABT - Preclinical Strategies: Taking an “Armed” Oncolytic Adenovirus to the Clinic Workshop 215 Programs of Excellence in Gene Therapy Room: 230/231 Chair: Sonia I. Skarlatos, PhD Speakers: Sonia I. Skarlatos, PhD - PEGT Overview Haig H. Kazazian, Jr., MD - Preclinical Gene Therapy of Hemophilia A with New AAV Serotypes Ronald G. Crystal, MD - Gene therapy for Alpha 1-antitrypsin Deficiency with Intrapleural Administration of Novel AAV Serotypes Barry London, MD, PhD - Gene Therapy During LVAD Support: Can We Fix the Broken Heart? Stanley Riddell, MD - Regulating T cells for Immunotherapy by Gene Insertion Richard A. Knazek, MD - The National Gene Vector Laboratories - Production, Toxicology, Repository, Clonality Testing and Long-Term Follow Up Workshop 216 Respiratory Tract: What’s New in Animal Models for Lung Gene Therapy Room: 265/266 Chair: Stephen Hyde, PhD Speakers: Bruce Bunnell, PhD - Mouse or Monkey: What is the FDA Looking for in Pre-clinical Data? Alice F. Tarantal, PhD - Monkey Model for Fetal Intrapulmonary Gene Transfer John F. Engelhardt, PhD - Are Men Mice or Pigs? A Lung Gene Therapy and Cell Biology Perspective Uta Griesenbach, PhD - FABp-CF Knockout Mice as Models for CFTR Gene Transfer Gerry McLachlan, PhD - Evaluation Non-viral Gene Transfer in the Ovine Lung
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156 NOTES 8th Annual Meeting • Ju
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FINAL PROGR AM - American Society of Gene & Cell Therapy

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