FINAL PROGRAM - American Society of Gene & Cell Therapy

More documents

Recommendations

Info

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 29Program ScheduleChae-Ok Yun, PhDA Phase I Study of Relaxin-Expressing Oncolytic AdenovirusOncolytic virotherapy is the use of genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue inreplicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aimof improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried outa phase I study of DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration of DWP418 is feasible, well tolerated,and associated with biological activity.Wednesday, May 19 thSeppo Ylä-Herttuala, MD, PhD, FESCAdVEGF-D Local Vascular Gene Transfer Program - To Reduce Graft Stenosis in Dialysis PatientsOncolytic virotherapy is the use of genetically engineered viruses that specifically target and destroy tumor cells via their cytolytic replication cycle. An important issue inreplicating virus-based cancer gene therapy that has received little attention is the limited distribution such viruses have been able to achieve in solid tumors. With the aimof improving viral distribution and tumor penetration for cancer gene therapy, we have developed a novel relaxin-expressing oncolytic adenovirus, DWP418. We carried outa phase I study of DWP418 in patients with recurrent solid tumors. The preliminary results show that intratumoral administration of DWP418 is feasible, well tolerated,and associated with biological activity.Alessandro Aiuti, MD, PhDGene Therapy for Primary ImmunodeficienciesGene therapy with hematopoietic stem cells is an attractive therapeutic strategy for several forms of primary immunodeficiencies. Results of the ADA-SCID and SCID-X1trials have shown long-term restoration of immune competence and clinical benefit. The use of a reduced-dose conditioning was crucial to allow substantial engraftment ofmultipotent gene-corrected hematopoietic stem cells. Self-inactivating lentiviral vectors have recently entered into the clinic and should provide significant advantages overgammaretroviral vectors.Lunch Break11:30 am – 1:00 pmScientific Symposium 1101:00 pm - 3:00 pmRoom: Thurgood Marshall WGene and Cell Therapy Translational StudiesChairJ. Kevin Donahue, MDSpeakersJ. Kevin Donahue, MDGene Therapy for Atrial FibrillationAtrial fibrillation affects 2-5 million people in the US and millions more around the world. Mechanisms known to sustain the arrhythmia include structural and electrical remodeling.We developed a novel method for atrial gene transfer that paints the vector on the atrial epicardium. Using this method with adenovirus vectors and a transgeneencoding a dominant negative potassium channel mutation, we show elimination of atrial fibrillation.Douglas W. Losordo, MDAutologous CD34 + Cell Therapy for Ischemic Tissue RepairPreclinical studies documented the potential therapeutic potency of endothelial progenitor cells, both as cultured and freshly isolated CD34+ cells. Phase I and II clinical trialsusing autologous, GCSF mobilized autologous CD34+ cells have been completed in patients with refractory angina and critical limb ischemia. These studies provide data supportingfeasibility, safety and bioactivity. Importantly, the early evidence suggests the efficacy of CD34+ cells and provides important data regarding endpoints and powercalculations for later phase studies. Accordingly, the goal of ischemic tissue repair appears feasible and is being approached in human clinical trials.
30American Society of Gene & Cell TherapyProgram ScheduleWednesday, May 19 thBarry J. Byrne, MD, PhDTherapeutic Strategies for Pompe Disease: Fact or FictionEarly presentation of cardiac dysfunction and the diagnosis of primary cardiomyopathy in infancy and childhood is most often associated with a genetic etiology. Oneexample of early cardiomyopathy is Pompe disease due to striated muscle glycogen storage. In order to develop a specific treatment based gene transfer, the presentationwill focus on systemic and cardiac gene transfer as an approach to disease management. Additional insight into therapeutic strategies is gained from the ongoing study ofa cohort of patients who have received enzyme replacement therapy. The rational for the first clinical gene transfer trial in Pompe disease will be presented.Patrick Most, MDS100A1: A Novel Molecular Therapeutic Advance for Heart FailureAdvanced understanding of the functional networks controlling cardiomyocyte performance together with the evolution of safe and efficient gene transfer technologieshave provided the foundation for the development of an innovative and novel genetically-targeted HF therapy resulting from the unique molecular profile of the cardiomyocyteCa2+ sensor protein S100A1. Here we show profound long-term therapeutic effectiveness and a favorable safety profile for AAV9-S100A1 gene therapy in apreclinical HF model employing cardiac-targeted gene delivery techniques and a gene dosage in pigs that are directly applicable to humans.Scientific Symposium 1111:00 pm - 3:00 pmRoom: Wilson ABCEthical Issues with Embryonic Stem Cells and Induced Pluripotent Stem CellsChairJonathan Kimmelman, PhDSpeakersJonathan D. Moreno, PhDUpdate on Ethical Issues in Stem Cell ResearchIn this talk I review the history of the embryonic stem cell debate and describe the current policy situation in the US and abroad. I then characterize the significance ofwork on induced pluripotent stem cell (iPSC) technology for the ongoing debate. In brief, with iPSC’s the controversy is likely to shift from the origins of the cell lines inhuman embryos to their fate as human regenerative and even reproductive materials.Insoo Hyun, PhDMedical Innovation Outside of Clinical Trials: Proceed with CautionRecently the U.S. Food and Drug Administration inaugurated a new policy allowing expanded access to investigational interventions by clinicians attempting to treatpatients outside the research context. For those interested in creating reliable platforms for a whole new generation of medical tools through genomics, gene transfer, andstem cell research, the FDA’s new policy holds many hazards. New models for local oversight of evidence-based medical innovation will be needed in order to avoid theuncontrolled and premature clinical exploitation of these platform technologies and still translate them successfully to the clinic.Jeremy Sugarman, MD, MPH, MAEthical Issues in Translational Research Involving Human Pluripotent Stem CellsThe clinical translation of human pluripotent stem cells may take several different paths including clinical translational research, clinical innovation, and the use of untestedor unproven interventions. Each of these translational pathways raises a unique set of ethical issues. Furthermore, there are distinct standards of practice and proceduralmechanisms for addressing the ethical issues inherent to each pathway. As such, it is essential that these differences be recognized to best protect not only the well-beingof patients, but also of the scientific enterprise as it seeks to understand the true value of stem-cell based interventions.PanelRoger H. Bertolotti, PhDJames Ellis, PhDDeborah A. Hursh, PhDEXHIBITOR PROSPECTUSfinal program
Page 1 and 2: FINAL PROGRAMAmerican Society of Ge
Page 4: 13 th AnnUAL MEETing | Washington,
Page 7 and 8: 6American Society of Gene & Cell Th
Page 12 and 13: 13 th AnnUAL MEETing | Washington,
Page 80 and 81:
13 th AnnUAL MEETing | Washington,
Page 82 and 83:
Page 84 and 85:
Page 86 and 87:
Page 88 and 89:
Page 90 and 91:
Page 92 and 93:
Page 94 and 95:
Page 96 and 97:
Page 98 and 99:
Page 100 and 101:
Page 102 and 103:
Page 104 and 105:
Page 106 and 107:
Page 108 and 109:
Page 110 and 111:
Page 112 and 113:
Page 114 and 115:
Page 116 and 117:
Page 118 and 119:
Page 120 and 121:
Page 122 and 123:
Page 124 and 125:
Page 127 and 128:
126American Society of Gene & Cell
Page 129 and 130:
Page 131 and 132:
Page 133 and 134:
Page 135 and 136:
Page 137 and 138:
Page 139 and 140:
Page 141 and 142:
Page 143 and 144:
Page 145 and 146:
Page 147 and 148:
Page 149 and 150:
Page 151 and 152:
Page 153 and 154:
Page 155 and 156:
Page 157 and 158:
Page 159 and 160:
Page 161 and 162:
Page 163 and 164:
Page 165 and 166:
Page 167 and 168:
Page 169 and 170:
Page 171 and 172:
Page 173 and 174:
Page 175 and 176:
Page 177 and 178:
Page 179 and 180:
Page 181 and 182:
Page 183 and 184:
Page 185 and 186:
Page 187 and 188:
show all

FINAL PROGRAM - American Society of Gene & Cell Therapy

Create successful ePaper yourself

Delete template?

Save as template?