FINAL PROGRAM - American Society of Gene & Cell Therapy

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