Sponsors
SEQUUS Pharmaceuticals
Heron Bioventures LLC
Rhône-Poulenc Rorer Gencell
International Union of Biochemistry and Molecular Biology (IUBMB)
Biovector Therapeutics, S.A.
Cell Therapy Research Foundation
GeneMedicine
ISIS Pharmaceuticals
æææææææ
Gene Therapy International Conference

Day 0: Sunday, August 16. Registration as you arrive. Reception 19:00-20:30

Day 1: August 17. Liposomal and Nonviral Gene Delivery
Liposomal delivery Monday Aug. 17
(Chair of session: Papahadjopoupos Demetrios & Henderson, Craig)
* Low, Philip Professor of Chemistry, Purdue University, West Lafayette, IN: "Folate-mediated targeting of liposomal and viral vectors to cancer cells" (25 min)
* Papahadjopoupos, Demetrios, California Pacific Medical Center Research Institute, San Francisco, CA: "Delivery of drugs and genes to tumor cells in vivo using sterically-stabilized immunoliposomes" (25 min)
* Henderson, Craig, CEO, SEQUUS Pharmaceuticals, Inc., Menlo Park, California: "Encapsulation of Nucleic Acids in Stealth Liposomes: Prospects for Enhanced Delivery to Occult Tumors After Intravenous Administration" (25 min)
* Woodle, Martin, Genetic Therapy Inc., A Novartis Company, Gaithersburg, MD: Extension of liposomal drug delivery to gene therapy: preparation and pulmonary gene expression activity of synthetic (non-viral) vectors (25 min)
* Gabizon, Alberto, Hadassah Hebrew University Medical Center, Jerusalem, Israel: "Intracellular drug delivery via targeted liposomes" (15 min)
* Thierry, Alain, Director, Department of Gene Therapy & Delivery, Biovector Therapeutics, S.A., Labège, France: "Optimization of Lipoplex for in vivo gene transfer: gallenic and efficiency" (25 min)
* Kostarelos, Kostas, R&D Section, Farmeco Co., Athens, Greece and Department of Chemical Engineering and Chemical Technology, Imperial College, UK: "Design of novel liposome structures" (15 min)

Nonviral Gene Delivery Monday Aug. 17
(Chair of session: Low, Philip)
* Midoux, Patrick, Research Director, INSERM, Centre de Biophysique Moléculaire, CNRS, Orléans, France: "Gene delivery based on the use of DNA/glycosylated polylysine complexes" (20 min)
* Storm, Gert, Associate Professor, Utrecht University, The Netherlands: "Methacrylate-based polymers and copolymers as transfection systems" (15 min)
* Nilsen, Thor, President, Polyprobe Inc., Bala Cynwyd, PA: "Applications for DNA dendrimers" (15 min)
* Höck, Katrin, Marketing Manager, Miltenyi Biotec GmbH, Bergisch Gladbach, Germany: "Selection of transfected cells with MACSelect" (15 min)
ææææææææææææææææææææææææææ
Day 2: August 18. Viral Gene Delivery
Retroviral delivery Tuesday Aug. 18
(Chair of session: Dornburg, Ralph)
* Dornburg, Ralph, Associate Professor of Medicine, Thomas Jefferson University, Philadelphia, PA: "Towards in vivo gene therapy: cell type-specific gene delivery with retroviral vectors containing engineered envelope proteins" (20 min)
* Raizada, Mohan (1998, 1999) Professor and Associate Dean, University of Florida College of Medicine, Gainesville, FL: "Potential for Gene Therapy in Long term Control of Angiotensin dependent Hypertension with a Retrovirally-mediated Delivery of AT1 Receptor Antisense (20 min)

Adenoviral & AAV delivery Tuesday Aug. 18
(Chair of session: Unger, Roger)
Plenary lecture
* Unger, Roger Touchstone/West Distinguished Chair in Diabetes Research, Director, Center for Diabetes Research, University of Texas Southwestern Medical Center, Dallas, TX: "Leptin gene transfer: impact on obesity and diabetes" (45 min)
* Srivastava, Arun, Professor, Indiana University School of Medicine, Indianapolis, IN: "Parvovirus vectors for human gene therapy" (20 min)
* Nemerow, Glen Department of Immunology, The Scripps Research Institute, La Jolla, CA: "Recombinant Adenovirus Vectors for Treating Latent Virus Infections" (20 min)
* Byrne, Barry, Assistant Professor of Pediatrics and Molecular Genetics & Microbiology, University of Florida Gene Therapy Center, Gainesville FL: "Parvovirus-mediated Gene Replacement for Inherited Cardiomyopathy" (20 min)
* Lawrence, John Assistant Professor of Medicine and Pediatrics, The Johns Hopkins University School of Medicine, Baltimore, MD: "Optimized adenovirus-mediated gene transfer to intact hearts" (15 min)
* Savino, Rocco, Senior Scientist, Istituto di Richerche di Biologia Molecolare (IRBM), P. Angeletti, Pomezia, Rome, Italy: "Delivery of therapeutic cytokine genes with an Adenoviral vector lacking all viral genes." (20 min)
* Kajiwara, Koji Department of Neurosurgery,
Yamaguchi University School of Medicine, Japan: "Immune responses to adenoviral vectors during gene transfer in the brain" (15 min)

Viral Gene Delivery Tuesday Aug. 18
* Fuchs, Renate, Professor, University of Vienna, Vienna, Austria: "Establishment of an assay to determine the efficiency of endosome rupture required for receptor-mediated gene delivery" (15 min)

Baculovirus, EBV, HSV, Alphavirus, HIV/Lentiviral vectors Aug. 18
* Lundstrom, Kenneth, Section Head, F. Hoffmann-La Roche, Basel, Switzerland: "Alphaviruses, novel vectors for gene therapy" (20 min)
* Rosenblatt, Joseph, Chief, Hematology Oncology, University of Rochester Cancer Center, Rochester, NY: "Novel approaches to HIV-1 gene therapy" (15 min)

Day 3: August 19. Clinical Trials
Clinical Trials on Cancer Wednesday Aug. 19
(Chair: Lopez-Berestein, Gabriel, Rubin, Joseph, Law, Peter & Nemunaitis, John)
* Lopez-Berestein, Gabriel, Professor of Medicine, Section Chief, The University of Texas, MD Anderson Cancer Center, Houston, TX: "Signal transduction pathways as a target for novel cancer therapeutics" (30 min)
* Lanuti, Michael, Postdoctoral Research Fellow, Thoracic Oncology Research Laboratory, Hospital of the University of Pennsylvania, Philadelphia, PA: "Adenoviral gene therapy for mesothelioma; host cellular and humoral immune response to vector" (25 min)
* Cole, David, Associate Professor of Surgery, Medical University of South Carolina, Charleston, SC: " Therapeutic strategies utilizing tumor-associated antigens for cancer gene/vaccine therapy: from the laboratory to phase I rV-CEA vaccine trial and back" (25 min)
* Rubin, Joseph, Consultant, Mayo Clinic, Rochester, MN: "Immunotherapy of metastatic disease by direct gene transfer of the HLA-B7/B2 microglobulin genes" (15 min)
* Galanis, Evanthia, Assistant Professor of Oncology, Mayo Clinic, Rochester, MN: Phase I/II trial of immunotherapy by direct gene transfer of IL-2 DNA /DMRIE/DOPE lipid complex in patients with advanced melanoma, renal cell carcinoma, and sarcoma" (15 min)
* Steiner, Mitchell, Associate Professor, University of Tennessee, Memphis, TN: "BRCA1 retroviral gene therapy Phase I clinical trial for prostate cancer" (25 min) for Vol 3: "Gene therapy of urologic malignancies"
* Sanda, Martin, Clinical Director of Urologic Oncology, University of Michigan Medical Center, Ann Arbor, MI: "Clinical development of recombinant prostate cancer vaccines" (25 min)
* Aguilar-Cordova, Estuardo, Director, Cell and Molecular Therapy Laboratories, Texas Children's Hospital, Baylor College of Medicine, Houston, TX: "Characterization of viral vector potency" (25 min)
* Nemunaitis, John, Regional Director, PRN Research, Inc., Director, TOPA Research, Dallas, TX: p53- A target for gene therapy: review on clinical trials" (25 min)
* Adachi, Naoto, University Hospital Zürich, Switzerland: The antitumor effect and biosafety of the HSV-tk/GCV gene therapy for recurrent glioblastoma multiforme"
* Puumalainen, Anu-Maaria, Resident of Neurosurgery, Research Scientist, University of Kuopio, Finland: "Gene therapy for malignant glioma" (15 min)
* Dehlinger, Peter, Partner, Dehlinger & Associates, Intellectual Property Law, and HERON Bioventures LLC, Palo Alto, CA: "Using clinical trial data to support and refine patent claims in gene therapy" (15 min)

Gene therapy of rheumatoid arthritis Wednesday Aug. 19
(Chair: Lopez-Berestein, Gabriel, Rubin, Joseph, Law, Peter & Nemunaitis, John)
* Robbins, Paul, Associate Professor, University of Pittsburgh, School of Medicine, Pittsburgh, PA: "Development of gene therapy for arthritis: preclinical and clinical studies"

Clinical Trials II Wednesday Aug. 19
(Chair: Lopez-Berestein, Gabriel, Rubin, Joseph, Law, Peter & Nemunaitis, John)
* Boyer, Jean (1998, 1999) Assistant Professor, University of Pennsylvania, Institute For Human Gene Therapy, Philadelphia PA: "DNA vaccines induce antigen-specific cellular immune responses" (20 min)
* Law, Peter Founder, President and Chairman of the Board, Cell Therapy Research Foundation, Memphis, TN: "Myoblast transfer delivers the human genome for therapy" (20 min)

Day 4: August 20. Immunotherapy, vaccines, cancer, prodrug
Cancer immunotherapy Thursday Aug. 20
(Session Chair: Georgiev, Georgii & Rosenblatt, Joseph)
* Jakobovits, Aya, Principal Scientist, Director, Discovery Research, Abgenix, Inc. Fremont, CA: "Therapeutic human monoclonal antibodies from mice engineered with human immunoglobulin loci" (20 min)

Strategies for gene therapy of cancer Thursday Aug. 20
(Session Chair: Georgiev, Georgii & Rosenblatt, Joseph)
* Akporiaye, Emmanuel, Associate Professor, The University of Arizona, Health Sciences Center, Tucson, AZ: "Elimination of tumor-mediated immunosuppression as a strategy of cancer gene therapy" (15 min)
* Gardner, Thomas, Assistant Professor of Molecular Urology and Therapeutics, University of Virginia, Charlottesville, VA: "Exploiting stromal-epithelial interaction for model development and new strategies of gene therapy for localized and metastatic prostate cancer" (25 min)
* Georgiev, Georgii, Director, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia: "New results on the use of tag7 and mts1 for gene therapy" (20 min)
* Rosenblatt, Joseph, Chief, Hematology Oncology, University of Rochester Cancer Center, Rochester, NY: Use of HSV amplicons in cancer (15 min)
* Noteborn, Mathieu , Leiden University, The Netherlands and Scientific Director, LEADD, The Netherlands: "Specific tumor-cell killing by means of adenovirus vectors expressing the Apoptin gene" (15 min)

Pulmonary transfection Thursday Aug. 20
(Session Chair: Georgiev, Georgii & Rosenblatt, Joseph)
* Malone, Robert, Gene Medicine Program, University of Maryland at Baltimore, School of Medicine, Baltimore, MD: "Aurintricarboxylic acid (ATA) strongly enhances direct plasmid transfection of respiratory tissues" (20 min)

Recombination in gene therapy Aug. 20
(Session Chair: Georgiev, Georgii & Rosenblatt, Joseph)
* Li, Liangping, Gene Therapy, Max-Delbrück-Center for Molecular Medicine, Berlin-Buch, Germany: "Establishment of new tumor cell lines by genetic methods.-Transient expression of SV40 large T antigen by Cre/LoxP-mediated site-specific deletion in primary human normal and tumor cells" (20 min) Chapter

Day 5: August 21: Angiogenesis, HIV, Regulatory elements, Novel horizons

Angiogenesis/VEGF/Restenosis Friday Aug. 21
(Session Chair: Meyer, François & Chao, Julie)
* Meyer, François, Vice President WW Discovery Research, Rhône-Poulenc Rorer/Gencell, Vitry-sur-Seine, France: "Gene therapy of restenosis: a novel approach" (25 min)
* Mahfoudi, Abder, RPR, Gencell Vector Development, CRVA, Monod, Vitry sur Seine, France:
Nonviral delivery of the FGF gene (20 min)
* Athanasopoulos, Takis, School of Biological Sciences, Royal Holloway University of London, Surrey, UK: "Gene therapy for atherosclerosis: insights from muscle-based expression of high density lipoprotein (HDL) p components" (15 min)
* Chao, Lee, Professor, Department of Biochemistry & Molecular Biology, Medical University of South Carolina, Charleston, SC: "Kallikrein gene therapy in cardiovascular and renal diseases" (20 min)
* Laitinen, Marja, Research Assistant, Resident of Internal Medicine, A. I. Virtanen Institute, University of Kuopio, Finland: "Gene transfer into rabbit artery" (15 min)

HIV therapy Friday Aug. 21
(Session Chair: Lebkowski, Jane & White-Scharf, Mary)
* Veres, Gabor, Senior Scientist, SyStemix, Inc., Palo Alto, CA: "Inhibition of HIV replication by antisense RNA" (20 min)
* Weiner, David, Associate Professor of Pathology, University of Pennsylvania, Philadelphia, PA: Engineering DNA vaccines for HIV-1" (20 min)

Molecular Mechanisms of Human Disease/ Novel horizons for gene therapy of disease Friday Aug. 21
(Chair: Lebkowski, Jane & White-Scharf, Mary)
* Lebkowski, Jane, Senior Director of Cell and Gene Therapy, GERON Corporation, Menlo Park CA: "hTERT gene transfer increases the life-span of normal primary human cells" (25 min)
* Helm, Gregory, Assistant Professor of Neurosurgery, Director, Molecular Neurosurgery & Endovascular Therapy Lab, University of Virginia Health System, Charlottsville VA: "Bone morphogenetic protein gene therapy" (20 min)

Gene therapy in transplantation Aug. 21
(Session Chair: Lebkowski, Jane & White-Scharf, Mary)
* White-Scharf, Mary, Vice President of Research, Biotransplant, Inc., Charlestown, MA: "Use of Gene Therapy in Xenotransplantation to Establish Specific Immune Tolerance" (20 min)

Regulatory elements /Vector improvement Friday Aug. 21
(Chair: Müller, Rolf & Harris, David)
* Müller, Rolf (1998, 1999) Professor, Institut für Molekularbiologie und Tumorforschung, Phillips-Universität Marburg, Germany: "Cell cycle-regulated and dual-specificity promoters for gene therapy" (25 min)
* Tsang, Tom, Assistant Research Scientist, University of Arizona, Tucson, AZ: "New vectors for very high levels of gene expression, inducible gene expression, and expression of two genes simultaneously" (20 min)
* Vasanwala, Farha, Graduate Student, University of Arizona, Tucson, AZ: The human HSP70B promoter is induced by heat, g-radiation and an antileukemic drug, geldanamycin" (10 min)

Fibroblast Growth Factor Receptor Aug. 21
(Chair: Müller, Rolf & Harris, David)
* Madar-Shapiro, Liora, Scientist, ProChon Biotech, Kiryat Weizmann Science Park, Rehovot, Israel: "Retroviral targeting of Fibroblast Growth Factor Receptor 3 as a potential therapy for Achondroplasia" (15 min)

Gala Dinner, Friday Aug 21: 18:30-Midnight

Day 6: August 22 Genomics, Gene & Drug Discovery

Genomics Saturday Aug. 22
(Chair: Müller, Rolf & Lai, Mei)
* Dehlinger, Peter Partner, Dehlinger & Associates, Intellectual Property Law, and HERON Bioventures LLC, Palo Alto, CA: "Computer-assisted DNA sequence inventions" (15 min)
* Boulikas, Teni, Senior Investigator, Institute of Molecular Medical Sciences, and President & CEO, Regulon, Inc., Palo Alto, CA: "Exploitation of matrix-attached regions/origins of replication from the human genome in gene therapy" (25 min)
* Schaumberg, Jack, Director, Product Development, GeneMedicine Inc., The Woodlands, TX: "Clinical experience with hIL-2 Gene Medicine in head and neck cancer" (15 min)

Gene & Drug Discovery Saturday Aug. 22
(Session Chair: Ayoubi, Torik)
* Ayoubi, Torik, Senior Scientist, Center for Human Genetics, University of Leuven, Flanders Interuniversity Institute for Biotechnology, Leuven, Belgium: "Molecular dissection of the genetic basis of benign tumour development" (15 min)
* Lai, Mei, Head, BRCA1 Group, Cancer Research Division, Lilly Corporate Center, Indianapolis, IN: "Functional Studies of BRCA1" (25 min).
* Talbot, Dale, Senior Research Scientist, ARIAD Pharmaceuticals, Inc., Cambridge, MA: "Regulation of gene expression through small molecule drugs" (25 min)

Free Afternoon. Visit to Knossos/Museum of Heraklion


Day 7: August 23 Ribozymes, Ligands, Antisense

Ribozymes/Aptamers Sunday Aug. 23
(Chair: Crooke, Stanley & Taira, Kazunari)
* Taira, Kazunari Professor, Institute of Applied Biochemistry, University of Tsukuba, Tsukuba Science City, Japan: "Novel dimeric minizymes with high potential as gene-inactivating agents" (25 min)
* Clawson, Gary, Professor, Penn State University, College of Medicine, University Hospital, Acting Director, Gittlen Cancer Research Institute, Hershey, Pennsylvania
* Tabler, Martin, Institute of Molecular Biology and Biotechnology, Heraklion, Greece: "Gene suppression by catalytic antisense RNA based on hammerhead ribozymes" (15 min)

Ligands/Peptide libraries Sunday Aug. 23
(Chair: Crooke, Stanley & Taira, Kazunari)
* Kuo, Tien Professor and Chief, Section of Eukaryotic Cell Research, The University of Texas M. D. Anderson Cancer Center, Houston, TX: "Hepatic gene targeting/therapy using a novel ligand from a malaria parasite protein"
* Bukau, Bernd Professor, Institut für Biochemie und Molekularbiologie, Albert-Ludwigs Universität Freiburg, Germany: "The use of peptide libraries for identification of substrate specificities of molecular chaperones" (25 min)

Antisense Sunday Aug. 23
(Chair: Crooke, Stanley & Taira, Kazunari)
Plenary lecture
* Crooke, Stanley Chairman of the Board, Chief Executive Officer, ISIS Pharmaceuticals, Carlsbad Research Center, Carlsbad, CA: Antisense oligonucleotides. (45 min)
* Graham, Roger, Director, Gene Therapy, Inex Pharmaceuticals Corp., Burnaby, B.C., Canada: (25 min)

Discussions & Concluding Remarks
End of Symposium

Posters
* Lanuti, Michael /Coukos, George /Sterman, Daniel, Division of Gynecologic Oncology, University of Pennsylvania Cancer Center: "Intraperitoneal application of recombinant herpes simplex virus-1 for the treatment of epithelial ovarian cancer"
* Kotsopoulou, Ekaterini, PhD student, Department of Biochemistry, Oxford University, UK: "RNA-based anti-HIV strategies. Exploiting the HIV codon bias"
* Yap, Melvyn, PhD student, Department of Biochemistry, Oxford University, UK: "Factors affecting retroviral production"
* Lu, Albert, Student, Medical School, Barcelona, Spain: "Caveolae in Signal Transduction: EGF triggers the recruitment of caveolin-1 into the sorting endocytic compartment of hepatocytes"
* Aguilar, Laura, Pediatric Hematology/Oncology Fellow, Baylor College of Medicine, Houston TX: "Efficient transduction of dendritic cells for the stimulation of a CTL response"

Participants

From Academic Institutions
* Chinopoulos, Christos, Budapest, Hungary
* Chao, Julie, Professor, Department of Biochemistry & Molecular Biology, Medical University of South Carolina, Charleston, SC
* Gabizon, Ruth, Senior Lecturer, Hadassah Hebrew University Medical Center, Hadassah Medical Center, Jerusalem, Israel
* Harris, David, Director, Cord Blood Bank, Professor of Immunology, Head, Gene Therapy Group, University of Arizona
* Krueger, Achim, Frauenklinik der Technischen Universität, Klinikum rechts der Isar, München, Germany
* Lehnart, Stephan, Molecular Cardiology, University Hospital of Freiburg, Germany
* Linardakis, Emma, ICRF Laboratory of Molecular Therapy, Imperial College School of Medicine, London, UK
* Suber, Michael, Baylor College of Medicine, Houston TX
* Uchida, Tetsuya, Postgraduate Student, Yamaguchi University School of Medicine, Kogushi, Ube, Yamaguchi, Japan

From Biotech Companies
* Arvanitidis Nick, Founder, SEQUUS Pharmaceuticals, & Partner, HERON Bioventures LLC, Menlo Park, CA
* Baron, Laurence, General Manager, Quantum Biotechnologies, Montreuil, France
* Gurevich, Vladimir, President, Fugent L.L.C., Madison, WI
* Mountain, Andrew, Director of Research, Cobra Therapeutics, The Science Park, University of Keele, Staffordshire, UK
* Okarma, Thomas, Vice President of Research & Development, GERON Corporation, Menlo Park CA
* Senoo, Makoto, Nippon Roche Research Center, Kamakura city, Japan
* Van Heuverswyn, Hugo, CEO, Innogenetics, Ghent, Belgium