Program Schedule
Scientific Symposium 100
8:30 am - 11:30 am
The Biologic License Application Validation Process
Co-Chairs
Boro Dropulic, PhD
Thomas Tillett
Speakers
Stephanie Simek, PhD
FDA Overview: The BLA Process and How You Get There?
Robert Shaw
Gene Based Therapies from Clinical Supply to Product Registration
Henry Hebel
Formulation and Stability of High Concentration, Multi-Component Plasmid Products
Carlos E. Ibanez, PhD
Potency Assays: How Do You Define Product Activity?
Daniel Giroux
Cell Lines, Growth Condition and Downstream Purification
Strategies for Optimizing Process Yields
Paul W. Shabram
End Point Titer Assays for Adenovirus Revisited
Scientific Symposium 101
8:30 am - 11:30 am
Clinical Trials from around the Globe
Chair
Olivier Danos, PhD
Speakers
Philippe Leboulch, PhD
Clinical Benefit with Clonal Dominance after Lentiviral Gene Therapy in Severe Human Thalassemia
Fulvio Mavilio, PhD
Gene Therapy for Skin Adhesion Disorders
Bobby Gaspar, MD, PhD
Gene Therapy for Adenosine Deaminase Deficiency: Twenty Years Later
Nathalie Cartier-Lacave, MD
Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in X-Adrenoleukodystrophy
Lunch Break
11:30 am – 1:00 pm
Scientific Symposium 110
1:00 pm - 3:00 pm
Cancer Clinical Trials
Chair
Kristen Hege, MD
Speakers
Gianpietro Dotti, MD
Gene Modified T lymphocytes for Cancer Immunotherapy
Neil Senzer, MD
From Lab to Clinic: an Intratumoral GM-CSF Encoding Gene
Enhanced, Immune Modulated Oncolytic Herpesvirus (OncoVEX
GM-CSF) in Patients with Unresectable Metastatic Melanoma
Michael Jensen, MD
Engineering T Cells For Cancer Immunotherapy: Which T Cells are Worthy of a Transgene?
Jack A. Roth, MD
Tumor Suppressor Gene Therapy
Scientific Symposium 111
1:00 pm - 3:00 pm
Overcoming Key Challenges in Advancing a Clinical
Trial through the Various Phases of FDA Approval
Co-Chairs
Joy Cavagnaro, PhD, DABT, RAC
Carl H. June, MD
Speakers
Jerry R. Mendell, MD
Taking Benchside Gene Therapy to the Bedside: More Than Proof of Concept
Changting Haudenschild, MD
FDA Perspectives on Clinical Trial Development of Gene Therapy
Panel
Malcolm K. Brenner, MD, PhD
Katherine A. High, MD
Donald B. Kohn, MD
Scientific Symposium 112
1:00 pm - 3:00 pm
Gene Therapies Going Global: Ethics and Policy Changes
Chair
Jonathan Kimmelman, PhD
Speakers
Alex John London, PhD
Gene Therapy Research in the Developing World: Some Considerations of Justice
Britta Wahren, MD, PhD
The Logistical and Ethical Challenges of Conducting Gene Transfer and Gene Vaccine Studies in Low-Income Countries
Panel
Valder Arruda, MD, PhD
John Dong, MD, PhD
Donna Przepiorka, MD, PhD
Scientific Symposium 113
1:00 pm - 3:00 pm
Late Stage Industry Clinical Trials
Co-Chair
Thomas Tillett
Douglas J. Jolly, PhD
Speakers
Raymond T. Bartus, PhD
CERE-120 (AAV2-NTN) for Parkinson’s Disease: Review of
Progress and Future Plans
Barbara Sosnowski, PhD
Phase 2b Study of GAM501 (Ad PDGF-B/Collagen) in the Treatment of Diabetic Ulcers
Estuardo Aguilar-Cordova, PhD
Synergizing Gene Transfer and Cancer Therapy in a Late Stage Trial
Alain Rolland, PharmD, PhD
Allovectin-7 Late Stage Clinical Development
Break
3:00 pm – 3:30 pm
Education Sessions
The goal of the ASGT Education Program is to provide participants
with a broad background and introduction to topics relevant to the
field of gene therapy. Education Sessions will be subdivided into two
headings. “Topical Review” sessions will provide a broad perspective
of an area of importance to the gene therapy community such as
major vector systems, specific target organs or diseases, and funding/
regulatory issues. “Emerging Field Review” sessions will provide a
broad perspective of a technological or scientific discipline that has
recently become of great significance to our members.
Education Session 120
3:30 pm - 5:00 pm
Emerging Field Review: Omics in Science Part I
Recent advances in the study of “whole systems” by array technology has lead to the development of powerful research tools that are yielding opportunities to understand complex biological systems from both a cellular and molecular perspective. In two back-to-back sessions recent advances in these technologies and the application of several different types of “omics” will be discussed as they relate to both understanding disease biology and gene transfer vector development.
Topics to be covered include:
Session I: Overview of the topic of Glycomics and Glcan Arrays,
Genome Wide Analysis of SNPs and Deep Sequencing, and
miRNAs in Disease Biology (Education Session 120)
Session II: Systems Biology/Protein Networks and Disease,
HapMap and Disease Gene Identification, Microbiome (Education
Session 130)
Chair
John A. Chiorini, PhD
Speakers
Ajit Varki, MD
Glycans and Glycomics - An Overview
Kelly Frazer, PhD
Human Genome Variation and Population-based Sequencing
Louise Laurent, MD, PhD
The Role of microRNAs in Disease Biology
Education Session 121
3:30 pm - 5:00 pm
Topical Review: Gene Delivery to the CNS: Problems and Progress
Gene therapeutic applications to the central nervous system (CNS) have met with impediments to efficient gene delivery such as spread of the vector to regions containing the target cells, host immunity to the vector and the therapeutic gene and sufficient expression within the target cell. The three speakers in this session will address different aspects of these issues and discuss potential strategies to overcome such barriers. Talks will focus on the issue of overcoming the host innate and memory immune response to CNS gene delivery, the issue of using CNS stem cells to overcome impediments of efficient CNS gene delivery, and the use of novel delivery systems to enhance vector delivery and spread within target sites in the CNS.
Chair
William F. Goins, PhD
Speakers
Pedro Lowenstein, MD, PhD
Immune Challenges to Gene Therapy of Brain Diseases: Practical
Implications for the Lab and Clinic
Brian K. Kaspar, PhD
Gene Delivery to the CNS: Problems & Progress
Krys Bankiewicz, MD, PhD
Importance of Efficient and Safe Gene Transfer to the Brain and How to Get There
Education Session 122
3:30 pm - 5:00 pm
Emerging Field Review: Regulatory RNA’s
Regulatory RNAs are small RNAs that control RNA expression by targeting transcripts for cleavage or translational repression. Found across plants, animals, and viruses, these genetic rheostats play critical roles in a wide spectrum of activities ranging from normal development to disease states such as cancer and heart disease. In this session, we will define and discuss the various classes of regulatory RNAs, outline their linkage to various human disease, and conclude by discussing their potential application as therapeutics for various human maladies.
Chair
Terry Hermiston, PhD
Speakers
John J. Rossi, PhD
Small RNA Biology and Therapeutic Applications
Jurgen Soutschek, PhD
Opportunities for micro RNA Therapeutics
Anton P. McCaffrey, PhD
MicroRNAs: New Tools and Targets for Gene Therapists
Education Session 123
3:30 pm - 5:00 pm
Emerging Field Review: Differentiation of Embryonic
Stem Cells and Reprogramming of Somatic Stem Cells
The emerging field review session, “Differentiation of Embryonic Stem Cells and Reprogramming of Somatic Stem Cells,” will cover three topics:
- Methodologies of generating induced pluripotent stem cells
- Genetic modifications of neural stem cells
- Hepatic engraftment of embryonic stem cells
Chair
Tal Kafri, PhD
Speakers
Juan Carlos Izpisua Belmonte, PhD
Generation of Disease-Free Hematopoietic Progenitors from
Fanconi Anemia-Specific Induced Pluripotent Stem Cells
Ilyas Singec, MD, PhD
Neural Differentiation of Embryonic Stem Cells and Reprogrammed Somatic Cells
Jeffrey H. Fair, MD
Strategies for In Vitro Differentiation of Embryonic Stem Cells to
Derivates of Endodermal Lineage
Education Session 124
3:30 pm - 5:00 pm
Topical Review: Innate Immunity
Cells of the immune system have receptors and cytoplasmic sensors for molecular structures and nucleic acids of invading pathogens such as bacteria or viruses. The resulting signaling process can initiate a rapid innate immune response characterized by release of proinflammatory cytokines, type I interferon, and inflammation. Following gene transfer, the consequence of innate immunity can be local or systemic immunotoxicity, tissue damage, and induction of an antigenspecific adaptive immune response against the transgene product and/or the vector. Incredible progress has been made in recent year in uncovering the mechanisms that lead to innate immunity. The session will discuss innate immune responses against bacterial pathogens, therapeutic RNA molecules, and adenoviral vectors.
Chair
Kirsten A. Weigel-Van Aken, MD
Speakers
Victor Nizet, MD
Probing Bacterial Evasion of Host Innate Defenses for New
Therapeutic Targets
Ian MacLachlan, PhD
Immunological Considerations for the Development of siRNA
Based Drugs
Dmitry M. Shayakhmetov, PhD
Innate Immunity to Adenovirus Vectors
Break
5:00 pm – 5:30 pm
Education Session 130
5:30 pm - 7:00 pm
Emerging Field Review: Omics in Science Part II
Recent advances in the study of “whole systems” by array technology has lead to the development of powerful research tools that are yielding opportunities to understand complex biological systems from both a cellular and molecular perspective. In two back-to-back sessions recent advances in these technologies and the application of several different types of “omics” will be discussed as they relate to both understanding disease biology and gene transfer vector development.
Topics to be covered include:
Session I: Overview of the topic of Glycomics and Glcan Arrays,
Genome Wide Analysis of SNPs and Deep Sequencing, and
miRNAs in Disease Biology (Education Session 120)
Session II: Systems Biology/Protein Networks and Disease,
HapMap and Disease Gene Identification, Microbiome (Education
Session 130)
Chair
Paul B. McCray, Jr., MD
Speakers
Trey G. Ideker, PhD
Protein Network Based Diagnostic Medicine
David Relman, MD
The Human Microbiome
Nicholas Schork, PhD
Gene Mapping, the HapMap, and Genomics-Based Personalized
Medicine
Education Session 131
5:30 pm - 7:00 pm
Topical Review: Cell and Gene Therapies for Liver
Safe and efficient delivery of cellular and molecular therapeutics is required for successful treatment of liver diseases. This session will review the most promising viral and non-viral vectors for delivery of DNA and regulatory RNAs to the cell. The mechanism of delivery and the pharmaceutical and immunological barriers to delivering these nucleic acids will be presented, as well as the therapeutic niches that each vector can fill. The session will also review the various types of stem cells that are being developed for therapeutic treatment of liver disease and the steps that are required to successfully translate these to the clinic.
Chair
Linda B. Couto, PhD
Speakers
Katherine P. Ponder, MD
Viral Vector Delivery to the Liver
Dexi Liu, PhD
Nonviral Gene Delivery to Liver
Markus Grompe, MD
Therapeutic Repopulation of the Liver
Education Session 132
5:30 pm - 7:00 pm
Topical Review: Targeted Chromosomal Integration
Uncontrolled insertion of foreign DNA into the human genome may have deleterious consequences, and is a serious safety concern for gene therapy applications. New molecular tools are being developed to target transgene integration to specific sites in the genome, and increase the frequency of homologous recombination with the purpose of achieving gene correction or replacement in the original genomic environment. These advances include viral and non-viral delivery of integrases, transposases and Zinc-finger nucleases, and will be discussed in the context of developing better and safer gene transfer technology.
Chair
Fulvio Mavilio, PhD
Speakers
Luigi M. Naldini, MD, PhD
Gene Targeting and Site-Specific Transgene Integration Mediated
by Zinc Finger Nucleases
Andre Lieber, MD, PhD
AAV-Rep 78 Based Systems for Site-Specific Integration
R. Scott McIvor, PhD
Transposon Mediated Gene Transfer
Education Session 133
5:30 pm - 7:00 pm
Emerging Field Review: Cancer Stem Cells
Detailed knowledge of the events that occur during stem cell differentiation could help answer fundamental questions about cancer stem cells. We discuss several standard stem cell model systems and will illuminate major parallels and differences between normal and malignant stem cells.
Chair
Thomas Zwaka, MD
Speakers
Timothy P. Cripe, MD, PhD
Identifying and Targeting Solid Tumor Stem Cells
David A. Williams, MD
Leukemia Stem Cells-Potential New Therapeutic Approaches
Thomas Zwaka, MD
Embryonic Stem and Carcinoma Cells
Education Session 134
5:30 pm - 7:00 pm
Topical Review: Subversion of Innate Immunity
Innate immunity can control many virus infections even in the absence of adaptive immunity. Several classes of pattern-recognition receptors, including Toll-like receptors and cytoplasmic receptors, recognize distinct microbial components and directly activate immune cells. However, viruses can still replicate and cause disease in vivo, because they have strategies for at least partially circumventing the innate immune response, including the interferon systems. This session introduces topical examples of these strategies, and discusses how innate immunity mechanisms influence the success of gene transfer protocols, and of oncolytic therapies based on replicating viral vectors.
Chair
Roberto Cattaneo, PhD
Speakers
Gregory E. Lemke, PhD
TAM Receptor Regulation of the Innate Immune Response
Yiping Yang, MD, PhD
Subversion of Innate Immunity by DNA Viruses
Adolfo Garcia-Sastre, PhD
Inhibition of Type I Interferon Responses by RNA Viruses
Exhibit Hall Social
7:00 pm – 9:00 pm