FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 25 SCIENTIFIC PROGRAM WEDNESDAY, JUNE 1, 2005 EDUCATION SESSION I – 3:30 PM - 5:00 PM Education Session 100 Retrovirus and Lentivirus Vectors This session will introduce three of the most popular versions of retroviral delivery systems, for which an increasing repertoire of strains and genera becomes available: gammaretroviral (based on murine leukemia virus), lentiviral (based on human immunodeficiency virus type 1) and spumaviral (based on “human” foamyvirus). They share the default aspects of the retroviral life cycle: receptor-mediated uptake of an enveloped particle, reverse transcription of a plus-stranded mRNA, and more or less random integration of double-stranded DNA following nuclear uptake of the preintegration complex. However, we will also discuss important differences in efficiency and biosafety related to viral origin and vector architecture. Room: 263/267 Chair: Christopher Baum, MD Speakers: Christopher Baum, MD - Retrovirus Vectors: We Are Family! Luigi Naldini, MD, PhD - Lentiviral Vectors: Challenges and Expectations David W. Russell, MD, PhD - Foamy Virus Vectors Education Session 101 Nonviral Vectors Nonviral gene therapy approaches may be simpler and safer than those using viral vectors. The challenges of non-viral gene therapy involve 1) development of vectors that provide long-term expression of therapeutic genes, and 2) development of suitable methods to deliver vector DNA to target cells. This session will examine the latest integrating and non-integrating vectors for achieving long-term gene expression and will describe current physical and chemical methods for DNA delivery. Room: 260/264 Chair: Michele P. Calos, PhD Speakers: Michele P. Calos, PhD - Integrating and Non-integrating Plasmids for Non-viral Gene Therapy Daniel Scherman, PhD - In Vivo Electrotransfer of Plasmids or Synthetic Nucleotides: Basic Concepts and Potential Therapeutic Applications Dexi Liu, PhD - What Constitutes a Better Synthetic Vector? Education Session 102 Immune Responses In vivo delivery of genes for therapeutic purposes and vaccines induces immune responses directed both against the vector as well as the gene-product. The goal of this educational session is to provide an overview of the role of immune responses in strategies for development of vector systems for gene therapy and vaccines. The overview of the immune response will include innate immunity, antigen processing and presentation, lymphocyte activation, induction of effecter responses and regulation of the immune responses. There will be a presentation and discussion on the use of gene transfer to induce tolerance to therapeutic or transplant antigens. Several exciting new approaches involving mechanisms of deletion, energy, or suppression will be discussed. In addition experiences of immunologic factors influencing efficacy, and safety in clinical trials with viral vectors will be reviewed. Room: 120/124 Chair: Maria Grazia Roncarolo, MD Speakers: Maria Grazia Roncarolo, MD - The Role of Regulatory T Cells in Suppressing Immune Responses to Transgenes and Inducing Immune Tolerance Jacques Banchereau, PhD - Dendritic Cells: Controllers of Immune Responses Education Session 103 Regulatory Hurdles to Phase III Clinical Trials This session will discuss FDA’s perspectives on what investigator/ sponsors can do to help move the field of gene therapy forward and get products to licensure. FDA will discuss what is the most efficient way to establish safety and efficacy, as well as what type of preclinical studies should be performed prior to initiation of clinical trials. Questions regarding the conduct of exploratory and confirmatory clinical trials will be discussed. FDA will also discuss why product characterization is important during early product development. These issues will be discussed as a way of explaining how the academic investigator can play a role in partnering with industry to help bring gene therapy products to licensure. Room: 265/266 Chair: Stephanie Simek, PhD Speakers: Andrew Byrnes, PhD - Common Challenges in the Development of Gene Therapy Products Mercedes Serabian, MS, DABT - Preclinical Considerations Beyond Phase 1 Daniel Rosenblum, MD - How to Make Optimal Use of the Approval Process
26 8th Annual Meeting • June 1 - 5, 2005 • St. Louis SCIENTIFIC PROGRAM WEDNESDAY, JUNE 1, 2005 Education Session 104 Expression Cassette Design Gene Therapy projects require expression of an exogenously introduced therapeutic gene. In The 1995 Orkin and Motulsky report identified inadequate knowledge of vector components necessary to maintain adequate expression of the therapeutic gene as a major fault in the field. Over the last 10 years, a significant amount of effort has been invested toward the development of optimized expression cassettes that provide sustained, high – level expression of therapeutic proteins in relevant disease models with viral and non – viral vectors. This session will provide guidance for the development of expression cassettes yielding high level, sustained expression of a desired therapeutic protein. The three presentations will provide (1) an overview to expression cassette design for viral and non – viral vectors, with a focus on using viral and house – keeping promoters for highest level of gene expression initially following gene transfer, (2) strategies that have been successfully used to provide sustained, high level tissue – specific expression, and (3) vector strategies that optimize protein expression through the use of optimal intron configuration and mRNA splicing, mRNA stability elements and translation initiation site configuration. A question and answer session will follow. Room: 123/127 Chair: Mitchell Finer, PhD Speakers: Mitchell Finer, PhD - Expression Cassette Design - 20 Years in 20 Minutes Carol H. Miao, PhD - Establishment of High-Level and Tissue-Specific Gene Expression Cassettes Thomas J. Hope, PhD - Post Transcriptional Regulation in Gene Expression Vectors Education Session 105 Cancer Gene Therapy Cancer has been a target application for numerous gene therapy trials. Recent advances in the use of oncolytic viruses, the type of anticancer transgenes employed for biologic effects and the effects of the immune system will be discussed. The understanding of these topics will facilitate improved applications in human trials. Room: 240 Complex Chair: E. Antonio Chiocca, MD, PhD Speakers: E. Antonio Chiocca, MD, PhD - HSV Oncolytic Viruses Glen Barber, PhD - VSV as an Oncolytic Vector: Mechanisms of Action Noriyuki Kasahara, MD, PhD - Retrovirus Vectors for Cancer Gene Therapy: Oncolytic, Anti-angiogenic, and Immunotherapeutic Strategies BREAK – 5:00 PM - 5:15 PM EDUCATION SESSION II – 5:15 PM - 6:45 PM Education Session 110 Adenovirus Vectors The goal of the Adenovirus Education Session is to survey the current state-of-the-art in our development and use of adenovirus gene transfer vectors. The session will begin with a discussion of the molecular biology of adenovirus including the production of replication –deficient vectors and packaging cell lines, the benefits and deficits of deletions of subsets or all the viral genes in the vector, the logic behind conditionally replicating adenoviruses, and variations relating to the Transgene expression cassette. The second topic of discussion will address the efficiency with which adenovirus interacts with target cells to deliver its genome to the nucleus. This discussion will include the basic infection pathway, altered vector tropism, variables relating to target cell type and physiology, and in vivo vector trafficking. The final topic of the session will be the interaction between adenovirus and the immune system including innate immunity, acquired immunity, and strategies for circumventing immune system barriers. Room: 263/267 Chair: Philip L. Leopold, PhD Speakers: Neil R. Hackett, PhD - Molecular Biology of Adenovirus Vectors Philip L. Leopold, PhD - Cell Biology of Adenovirus Infection: Subcellular Mimicry James M. Wilson, MD PhD - Immunology of Adenoviral Vectors Education Session 111 HSV Vectors This session will describe design features and production features, plus experimental and therapeutic uses of recombinant oncolytic, replication defective and amplicon vectors derived from herpes simplex virus type 1. Oncolytic vectors are designed for treatment of cancer based on targeted delivery and selective virus replication in tumor cells. Clinical trials using these vectors for brain tumors will be described. Replication defective viruses deleted for essential viral functions have been engineered for peripheral nervous system applications since they naturally persist in sensory nerves following local delivery to the skin. Amplicon vectors incorporate no viral genes and can include up to 150 kb of genetic information, including complete genes, self-contained regulatory elements and AAV genome integrating components. These vectors provide versatile tools for systematic genetics and therapeutics. Room: 260/264 Chair: Xandra Breakefield, PhD Speakers: Xandra Breakefield, PhD - Mega-multi Capacity HSV Amplicon Vectors Joseph C. Glorioso, PhD - HSV Gene Vectors for Treatment of Pain and Neuropathy E. Antonio Chiocca, MD, PhD - HSV Vectors for Chemotherapy Delivery
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156 NOTES 8th Annual Meeting • Ju
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