FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 45Program ScheduleSteven C. Ghivizzani, PhDGene Therapy for AchondroplasiaAchondroplasia is the most common form of short-limbed dwarfism and is caused by a specific mutation in the transmembrane portion of fibroblast growth factor receptor3 (FGFR3), a negative regulator of growth and maturation in growth plate chondrocytes. This “gain of function” mutation causes enhanced signaling from FGFR3, leadingto reduced growth in bones that grow by endochondral ossification. Using gene delivery, we have been exploring the utility of systemic delivery of a secreted FGFR3variant as a means to titrate circulating FGFR3 ligands and, thereby, reduce aberrant FGFR3 signaling to rescue bone growth. Our results thus far in explant culture and ina mouse model of achondroplasia have been highly encouraging, and indicate that a related post-natal treatment approach may be viable in humans.Joel R. Chamberlain, PhDSystemic Delivery of RNAi Therapeutics for Treatment of Myotonic DystrophyDominant genetic diseases pose a particular challenge for therapy since the disease-causing cell product must be eliminated. We have chosen an RNA interference-basedapproach to target toxic mRNA that causes myotonic dystrophy. We show that disease pathology in the transgenic HSALR mouse model of DM1 is mitigated by systemicapplication of AAV6 carrying an HSA-targeted RNAi expression cassette. Aspects of systemic delivery and endogenous RNAi pathway effects will be presented in the contextof therapeutic development. These studies highlight the potential of RNAi as a therapy for dominant muscle disease and for other diseases where body-wide therapeuticRNAi distribution is required.Carmen Bertoni, PhDEnhanced Cell-Mediated Regenerative Approaches to DMD Using RNA InterferenceMany of the diseases affecting muscle are distinguished by the impaired ability of muscle progenitor cells to fuse and form new fibers and the inability of the existingfibers to maintain normal homeostasis. Among those, Duchenne muscular dystrophy (DMD) is the most severe and is caused by lack of dystrophin expression. The loss ofmuscle mass and strength typically seen in DMD patients is thought to be attributed to the progressive atrophy and loss of individual muscle fibers, which is concomitantto the accumulation of fibrotic and fat tissues in skeletal muscles. The mdx mouse has been instrumental in determining the potential beneficial effects of some of thetherapeutic approaches currently under development for DMD. We have used High Throughput Screen (HTS) to identify modulators of muscle progenitor cells fusionin vitro and screened several siRNA libraries in order to identify genes that play active role during terminal differentiation. Secondary and tertiary screens were used toconfirm the effects of RNAi on wild type or mdx muscle cultures and to select for candidate hits that significantly enhance myotube size.Transplantation of wild type muscleprogenitor cells undergoing silencing of the genes identified by HTS enhanced the ability of those cells to repair skeletal muscles of mdx/nude mice and resulted in asignificant increase in dystrophin expression. Direct intramuscular injection of a lentiviral vector expressing shRNAs of those genes was also shown to dramatically improveregeneration of injected muscles. These studies advance cell-mediated regenerative approaches to DMD and may enable us to identify treatments capable of slowing downprogressive muscle wasting characteristic of many neuromuscular disorders.Thursday, May 20 thScientific Symposium 21510:30 am - 12:30 pmRoom: Wilson ABCNovel Cell and Gene Therapy Interventions for the LungChairBruce A. Bunnell, PhDSpeakersDaniel Weiss, MD, PhDRecent Advances in Gene and Cell Therapies for Lung DiseasesGene and cell therapy approaches for lung diseases continue to rapidly develop. Progress has been made in vector development and several notable advancements havebeen made over the past year with use of gene transfer approaches for lung cancer. In parallel, cell therapy approaches for lung diseases have broadened and includestudies of ex vivo lung regeneration using state of the art bioengineering approaches as well as increasing appreciation of immunomodulatory effects of adult mesenchymalstem cells in the lung. Most notably, progress has been made in clinical trials of cell therapies for both pulmonary hypertension and for chronic obstructive pulmonarydisease. The introductory comments and subsequent presentations in this session will highlight these advances.
46American Society of Gene & Cell TherapyProgram ScheduleThursday, May 20 thPaul B. McCray, Jr., MDLentiviral Vector Development for Prevention and Treatment of Cystic Fibrosis Lung DiseaseThis talk will provide an overview of recent developments in lentiviral vector technology and their applications for lung disease treatment. Additional data will be presentedregarding animal models of CF lung disease and their potential utility in pre-clinical studies of efficiency and efficacy of gene transfer.Blair Roszell, MSTwo Step Differentiation of Mouse Embryonic Stem Cells to Definitive Endoderm and Lung Epithelial CellsCell-based therapy is a novel and promising treatment option for lung disease, but deriving large quantities of lung epithelial cells from embryonic stem (ES) cells isstill inefficient. In my talk, I will compare growth factor-based and small molecule-based methods for deriving definitive endoderm. After an additional lung inductionstep, activin/wnt and IDE2 treated cells express a similar profile of lung-specific markers. Furthermore, in fetal pulmonary cell co-cultures, activin/wnt treated cells canintegrate into alveolar forming units; whereas IDE2 treated cells express SP-C, but do not integrate into lung-like structures. Finally, ES-derived lung cells were endotracheallyinjected into preterm mice with evidence of AEII distribution within the lung parenchyma. These studies concludes that a recapitulation of development may enhancederivation of an enriched population of lung-like cells for use in cell based-therapy.Peter I. Lelkes, PhDBiomedical Engineering in the LungThe goal of this invited lecture is to present an overview of current tissue engineering and regenerative medicine -based approaches to repair dysfunctional lungs, withfocus on neonatal and adult pulmonary diseases. Specifically, we will present our developmental biology-based studies to create vascularized alveolar structures in vitroand in vivo. In addition, we will discuss attempts by us and others to optimize the directed differentiation of embryonic stem cells towards pulmonary epithelial and otherlung-specific cell types for distal lung tissue engineering and regeneration.John F. Engelhardt, PhDNew Developments in Animal Models of Cystic FibrosisCystic fibrosis (CF) is a recessive disease that affects multiple organs and is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.Animal modeling of this disease has been challenging, with species- and strain-specific differences in organ biology and CFTR function influencing the emergence of diseasepathology. CF mice have been an invaluable system for dissecting the biology of CFTR function in certain organs, however, they generally lack pancreatic and spontaneouslung disease seen in humans with CF. Recently, CF pig and ferret models have been generated. Comparative analysis of these CF models may help to understand how thepleiotropic functions of CFTR in multiple organs influence disease progression.Special NIH Program Session 21610:30 am to 12:30 pmRoom: Thurgood Marshall WestNIH Resources for Cell and Gene Therapy InvestigatorsModeratorCheryl L. McDonald, MDSpeakersDaniel Rosenblum, MDPThe National Gene Vector Biorepository and Coordinating Center (NGVBCC)The NGVBCC provides an array of support services for NIH-supported investigators engaged in research on Gene Therapy. The services include archiving of biologicalmaterials, insertional site analysis, pharmacology and toxicology resources, and a reagent repository. It has an open database and educational resource. The archiving andanalytical services are limited to investigators at academic and other not for profit institutions. We will review the scope of the services, methods for accessing them, waysof meeting eligibility for their use, website link information, specimen submission and electronic access, and other relevant information pertinent to use of the facility.How the Clinical and Translational Science Awards (CTSA) Program can Support Cell and Gene Therapy InvestigatorsThe CTSA program replaced the GCRC (General Clinical Research Center Program) with a much broader support for novel product and team science within and betweenacademic institutions and other related research support such as the NIH and industrial sponsors. The aim is to streamline project development, review, and implementationof translational and clinical research by providing support from a variety of services such as biostatistics, research design, ethics, clinical research management,informatics, and regulatory knowledge (including FDA). It also facilitates the creation of networks between academic centers and the recruitment of trial participants fromthe community. The presentation will review pertinent summary information about the CTSA program including web access to program information, access to services,potential support from CTSA-related key functions for development of clinical trials, and a brief description of concierge service (a team of experts who can provide supportin the creation and approval of a clinical trial).EXHIBITOR PROSPECTUSfinal program
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FINAL PROGRAM - American Society of Gene & Cell Therapy

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