FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 67Program ScheduleScientific Symposium 4018:30 am - 10:30 amRoom: Virginia SuiteLocal Delivery of Chemical Gene Therapy – Mechanisms, Quantitative Understanding and Applications of Biodegradable CarriersChairErnst Wagner, PhDSpeakersLonnie D. Shea, PhDLocalized Gene Delivery by Biomaterial ImmobilizationBiomaterials are combined with gene delivery to provide a structure that guides cell growth, while also inducing the expression of inductive factors that drives the cellularprocesses leading to functional tissues. We have investigated immobilization of gene therapy vectors to biomaterials as a means to maintain the vectors locally, co-localizethe vectors to the surface for cell adhesion, and stabilize the vectors against degradation. The design principles we have developed for hydrogels and microporous scaffoldswill be presented, as well as their use with in vitro tissue models and in vivo regeneration. These biomaterial scaffolds provide opportunities to enhance or control genetransfer relative to more traditional delivery methods (e.g., injection), and provide an enabling technology to molecularly dissect tissue formation or to promote functionaltissue regeneration.Aliasger K. Salem, PhDPulsatile Release of Biomolecules from Polydimethylsiloxane ChipsCytosine-phosphorothioate-guanine oligodeoxynucleotides (CpG ODN) is a recent class of immunostimulatory adjuvants that includes unmethylated CpG dinucleotidesequences similar to those commonly found in bacterial DNA. CpG ODN specifically triggers toll like receptor 9 (TLR9), which is found within phagoendosomes of antigenpresenting cells (APCs) such as dendritic cells (DCs). CpG ODN triggers activation and maturation of DCs and helps to increase expression of antigens. CpG ODN can beused to induce polarized Th1 type immune responses. We have shown that antigens and CpG ODN must be co-delivered to generate the most potent therapeutic antigenspecificimmune responses. We have developed a series of delivery vehicles to ensure co-delivery of antigens and CpG ODN to the same APCs and to significantly increaseuptake by APCs. These strategies can result in antigen-specific immune responses that are 5 to 500-fold greater than administration of antigen alone. The approaches wehave developed to achieve this include the utilization of conjugate molecules, multicomponent nanorods, biodegradable microparticles, cell-microparticle hybrids and finallypolydimethylsiloxane based pulsatile release chips.David M. Lynn, PhDA ‘Multilayered’ Approach to the Delivery of DNA: Exploiting the Structure of Polyelectrolyte Multilayers to Promote Surface-MediatedTransfection and Tunable Multi-Agent DeliveryMethods for the layer-by-layer deposition of oppositely charged polymers on surfaces can be used to fabricate ultrathin multilayered films (or ‘polyelectrolyte multilayers’)using a broad range of natural and synthetic materials, including DNA. If the individual components of these assemblies are designed appropriately, these methods alsoprovide opportunities to design thin films that provide control over the release of DNA and other agents from surfaces. This talk will describe recent work from our laboratoryon the design of new polymers and the fabrication of thin films and coatings that provide spatial and temporal control over the release of DNA from surfaces in vitroand in vivo. These methods provide tunable control over the surface-mediated release of DNA over periods ranging from several hours to several days, weeks, or months,and can also be used to fabricate nanostructured films with hierarchical structures that permit control over the release of two or more DNA constructs with separate,distinct, and predictable release profiles (e.g., rapid release of one DNA construct followed by the slower, sustained release of a second DNA construct). With furtherdevelopment, these approaches could contribute to the design of thin films and coatings capable of delivering precise and well-defined quantities of multiple different DNAconstructs (or combinations of DNA and other therapeutic agents) in a broad range of fundamental and applied contexts.Daniel G. Anderson, PhDCombinatorial Development of Biomaterials for Gene DeliveryHigh throughput, combinatorial approaches have revolutionized small molecule drug discovery. Here we describe our work on high throughput methods for developing andcharacterizing biomaterials for genetic therapy and cell therapy. We have developed automated methods allowing for rapid synthesis of 1000’s of biomaterials, as wellas the testing of their chemical, material, and biological properties. These methods have also been applied towards the development of new methods to control stem cellbehavior, as well as vehicles for drug delivery. In particular, these combinatorial libraries of different biomaterials have enabled new methods for non-viral gene therapyand siRNA delivery.Saturday, May 22 nd
68American Society of Gene & Cell TherapyProgram ScheduleScientific Symposium 4028:30 am - 10:30 amRoom: Delaware SuiteEngineered T Cells in the Clinic: Emerging Clinical and Regulatory IssuesChairJacqueline Corrigan-Curay, MD, JDSpeakersDaniel M. Takefman, PhDGene Modified Cell Based Therapies: FDA PerspectivesThe purpose of this presentation is to provide FDA perspectives on gene modified cell based therapies. This presentation will outline some key regulatory considerationsfor the clinical use of patient specific gene therapies, such as engineered T-cells. The emphasis with be on manufacturing considerations as unique issues arise whenmanufacturing these types of products for clinical use.Joy Cavagnaro, PhD, DABTLimitations of Preclinical Studies for Predicting Safety of Uniquely Human Specific TherapiesCarl H. June, MDProlonged Engraftment and Decade Long Safety Record of Engineered T CellsIncreasing data from pre-clinical experiments and pilot clinical trials illustrate the potential of engineered T cells for therapy of cancer, chronic infection and autoimmunity.A key issue facing the field has been limited persistence of engineered T cells in most trials. Our clinical data indicates that T cells engineered to express a chimeric antigenreceptors can exhibit long term survival, and that in HIV infected patients, engineered T cells have a considerable safety record.PanelHelen E. Heslop, MDMichel Sadelain, MD, PhDSteven A. Rosenberg, MD, PhDSaturday, May 22 ndScientific Symposium 4038:30 am - 10:30 amRoom: Marriott Ballroom Salon 1Mechanisms of Innate Immunity Influencing Gene TherapyChairDavid B. Weiner, PhDSpeakersValder Arruda, MD, PhDGene Therapy for Tolerance Induction in Large Animal ModelsThe formation of neutralizing antibodies (inhibitors) to clotting Factor VIII or FIX is a major safety concern on the development of novel therapies for hemophilia. Inhibitorformation renders the protein replacement ineffective resulting in both high morbidity and mortality. Therefore the prevention or eradication of inhibitors is of fundamentalimportance. To eliminate the presence of inhibitors in hemophilia, an immune tolerance induction strategy consisting of large amounts of FVIII/FIX injected on a dailybasis for long periods of time (months to years) results in variable success rates. We will review gene-based strategies to prevent inhibitor formation in inhibitor-pronedogs. Notably, recent evidence demonstrates the possibility of the use of gene-based therapy for the eradication of preexisting inhibitors to FVIII from studies in hemophiliaA dogs. Emerging evidence on the role of T regulatory cells in modulating immune responses to the therapeutic protein now endogenously expressed by gene therapyopens a novel potential application of gene therapy, not only to prevent, but also to eradicate inhibitors in hemophilia and other diseases.EXHIBITOR PROSPECTUSfinal program
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FINAL PROGRAM - American Society of Gene & Cell Therapy

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