Program Schedule

(This program is subject to updates/changes.)

• Wednesday, May 31
• Thursday, June 1
• Friday, June 2
• Saturday, June 3
• Sunday, June 4

Thursday, June 1

George Stamatoyannopoulos Lecture 200 8:00 am – 9:15 am George Stamatoyannopoulos Lecture Room: Ballroom - Level 400
Chair: Theodore Friedmann, MD Speakers:
C. David Allis, PhD Beyond the Double Helix: Reading and Writing the "Historic Code"	Celia Witten, MD, PhD Gene Therapy Past and Future: FDA Collaborations
Presentation of Excellence in Research Awards

Workshop 210
9:30 am - 11:00 am
Cancer: Animal Models for Cancer Gene Therapy
Room: 314/315
Chair: James S. Norris, PhD

Speakers
Robert M. Hoffman, PhD
Metastatic Disease in Cancer

E. Antonio Chiocca, MD, PhD
Brain Tumor Models

David M. Spencer, PhD
Imaging in Murine Models: EZC Optical versus Molecular Imaging

Carolina Berger, MD
Genetic Modification of T Cells for Immunotherapy

Workshop 211
9:30 am - 11:00 am
Cardiovascular: Tools and Resources for Cardiovascular Gene Therapy
Room: 324/326
Co-Chairs:
Sonia I. Skarlatos, PhD
David A. Dichek, MD

Speakers:
Sonia I. Skarlatos, PhD
NHLBI Resources for Gene and Cell-Based Therapies

David A. Dichek, MD
Vascular Gene Transfer with Helper-Dependent Adenoviral Vectors

David Root, PhD
Lentiviral RNAi Libraries

Andrew H. Baker, PhD
Novel Vectors for In Vivo Gene Delivery for Vascular Disorders: Potential and Limitations

J. Kevin Donahue, MD
Large Animal Models in Gene Therapy Preclinical Studies

Workshop 212
9:30 am - 11:00 am
Hemopoietic: Hemopoietic Cell Trafficking and In Vivo Imaging
Room: 318/320
Chair: Jan Nolta, PhD

Speakers:
Jan Nolta, PhD
Tracking Human Stem Cells to Sites of Tissue Damage and Hypoxia

Alice F. Tarantal, PhD
MicroPET and Bioluminescence Imaging - Studies with Nonhuman Primate Models

Gay Crooks, MD
Imaging on Luciferase with Hemopoietic Cells in Mice

Joseph Frank, MS MD
Tracking the Fate of Magnetically Labeled Hemopoietic Stem Cells by MRI

Workshop 213
9:30 am - 11:00 am
Immunology of Gene Therapy: Innate Immunity to Viral Vectors, siRNA, and Transplanted Cells
Room: 339/342
Chair: Daniel R. Salomon, MD

Speakers:
Terence R. Flotte, MD
Innate Immune Responses to AAV

Hildegund CJ Ertl, MD
Innate Immunity to Adenoviral Vectors

Ian MacLachlan, PhD
The Innate Immune Response to siRNA

Daniel R. Salomon, MD
Innate Immunity to Transplantation

Selda Samakoglu, PhD
Position Dependence of Intron-Encoded ShRNA Processing and Interferon Response

Workshop 214
9:30 am - 11:00 am
Industrial Liaison: Clinical Developments in Gene Vaccines
Room: 345/346
Chair: Alain Rolland, PharmD PhD

Speakers
Alain Rolland, PharmD PhD
Overview of Gene Vaccines

Timothy Tobery
Strategies for HIV Vaccination

Michael Fons, PhD
Clinical Electroporation

Julie E. Martin, DO
Safety and Immunogenicity of a DNA Vaccine for West Nile Virus: Report of a Phase I Clinical

Suzanne Epstein, PhD
Control of Influenza via Conserved Features

Workshop 215
9:30 am - 11:00 am
Nonviral Gene Therapy: siRNA Based Therapeutics and Delivery
Room: 327/329
Chair: Seng H. Cheng, PhD

Speakers:
Andrew D. Miller, PhD
Synthetic Non-Viral siRNA Delivery: The Role of Formulation in Enabling siRNA Based Therapeutics

Amy C. H. Lee
Systemic Delivery of siRNA Resulting in ApoB Silencing and Amelioration of Hypercholesterolaemia

Muthiah Manoharan, PhD
Chemical Conjugates of siRNA

Samuel J. Reich
C and S, an Anti-VeOF siRNA for the Treatment of Wet Age-Related Macular Degeneration

Workshop 216
9:30 am - 11:00 am
Respiratory Tract: Therapeutic Approaches to Gene Regulation - Pulmonary Applications
Room: 337/338
Co-Chairs:
Paul B. McCray, Jr., MD
David A. Dean, PhD

Speakers
Adolfo Garcia-Sastre, PhD
Genetic Engineering of Influenza Viruses

Eric W.F.W. Alton, MD, FRCP
Delivery of Oligo/RNAi to Respiratory Cells - Where do they go?

Sailen Barik, PhD
Uses of RNAi to Inhibit Respiratory Viral Infection

Jianzhu Chen, PhD
RNA Interference of Influenza Virus

John J. Rossi, PhD
Rational Design of RNAi Triggers for Targeted Down regulation of mRNAs

Workshop 217
9:30 am - 11:00 am
Viral: Pre-Clinical/Clinical Trials with Retroviral Vectors
Room: 321/323
Chair: Cynthia Dunbar, MD

Speakers
Cynthia Dunbar, MD
Insertion Site Analysis of Retroviral Vectors in a Non-Human Primate Model of Autologous Stem Cell Transplantation

Adrian J. Thrasher, MD, PhD
Clinical Therapy for Immunodeficiency

Alessandro Aiuti, MD PhD
The ADA-SCID Gene Therapy Trial: Long-Term Safety and Efficacy

Manuel Grez, PhD
Clinical results from a Phase I Gene Therapy Trial for Chronic Granulomotous Disease

Brian P. Sorrentino, MD
MGMT-Mediated Selection and Chemo Protection in a Rhesus Macaque Transplant Model

Break
11:00 am - 11:15 am

Workshop 220
11:15 am - 12:45 pm
Clinical & Regulatory Affairs: A Critical Review of How IRBs and IBCs Regulate and Influence Clinical Research in Gene Therapy
Room: 343/344
Co-Chairs:
Joy Cavagnaro, PhD, DABT, RAC
Daniel R. Salomon, MD

Speakers
Ernest Prentice, PhD
History, Organization, and Mission of the Modern IRB that Define Challenges Today

Gwenn Oki, MPH
Specific Considerations and Challenges for Gene Therapies in Review: the IRB/IBC Administrator’s View

John A. Zaia, MD
Specific Considerations and Challenges for Gene and Cell Therapies in Review: the Clinical Investigator’s View

Marcia C. Finucane, MS, CBSP
IBCs: The Mission and the Challenges

Workshop 221
11:15 am - 12:45 pm
Embryonic/Somatic Stem Cells & Tissue Engineering: Mechanisms of Cardiovascular Repair and Regeneration
Room: 324/326
Chair: Stanton L. Gerson, MD

Speakers
Loren Field, PhD
Cellular Mechanisms to Repair Infracted Hearts

Duncan J. Stewart, MD, FRCPC
Therapeutic Neovascularization: Have We Done the Right Trials?

Marc Penn, MD, PhD
Exploitation of Stem Cell Homing for Gene Delivery

Douglas Losordo, MD
The Microvasculature as a Therapeutic Target in Ischemic Tissue Repair

Workshop 222
11:15 am - 12:45 pm
Gene-Based Vaccines: Advances in Antigens and Adjuvants
Room: 345/346
Chair: David B. Weiner, PhD

Speakers:
David B. Weiner, PhD
Advances in Antigens and Adjuvants: Overview
DNA Based Adjuvants

Alex Franzusoff, PhD
Preclinical & Clinical Development of Yeast-Based Targeted Molecular Immunogens for Immunotherapy

Mary Lynne Hedley, PhD
Targeting CYP1B1 with a Therapeutic Cancer Vaccine: Results of Preclinical and Clinical

Richard G. Carroll, PhD
Development of Antigen-Presenting Tumor Cells

Workshop 223
11:15 am - 12:45 pm
Gene Therapy for Genetic Diseases: Gene Therapy of the Senses
Room: 318/320
Chair: Jean Bennett, MD, PhD

Speakers
Alfred Lewin, PhD
Gene Therapy for Retinal Degeneration

Jean Bennett, MD, PhD
Leber Congenital Amaurosis (LCA)

Peter A. Campochiaro, MD
Gene Therapy for Macular Degeneration

Alberto Auricchio, MD
AAV-Mediated Gene Transfer for Retinal Diseases

Yehoash Raphael, PhD
Atoh Restores Hearing

Workshop 224
11:15 am - 12:45 pm
Infectious Disease: Manipulating Regulatory/Suppressor T Cells
Room: 339/342
Chair: David Klatzmann, MD, PhD

Speakers:
David Klatzmann, MD, PhD
Regulatory/Suppressor T Cells Dynamics and Their Therapeutic Implications

Maria Grazia Roncarolo, MD PhD
In Vivo Modulation of Anti-Transgene Specific Immune Response

Crystal L. Mackall, MD
Homeostasis of CD4+CD25+Regulatory T Cells During Lymphopenia and Following Il2

Ethan Shevach
Therapeutic Potential of T Regulatory Cells

Workshop 225
11:15 am - 12:45 pm
Musculo-Skeletal: Gene Expression and Immunology of Musculoskeletal Gene Therapy
Room: 337/338
Chair: Paul D. Robbins, PhD

Speakers:
James Kang, MD
Gene Therapy for Disc Degeneration

Steven C. Ghivizzani, PhD
Gene Therapy for Arthritis: Maintenance of Transgene Expression

Pervin Anklesaria, PhD
Clinical Development of an AAV Vector Expressing the TNF Receptor: Fc Fusion Gene in Inflammatory Arthritis

Christian Kuhr, MD
Immunology of AAV Therapy in Canine Model of Muscular Dystrophy

Xiao Xiao, PhD
Gene Transfer and Persistence in Muscles of Large Animals

Workshop 226
11:15 am - 12:45 pm
Neural Disorders: Neuro-Imaging
Room: 321/323
Chair: John H. Wolfe, VMD, PhD

Speakers:
Michael A. Barry, PhD
Dynamic Imaging of Cell Targeting Ligands and Vectors

Khalid Shah, PhD
Imaging Fate and Therapeutic Effects of Neural Stem Cells in Brain Tumor Models

Yoshinaga Saeki, MD PhD
Imaging Delivery and Replication of Oncolytic HSV Vectors

Charles H. Vite, DVM
Nuclear Magnetic Resonance Measurements of Brain Histopathology

David Piwnica-Worms, MD PhD
Molecular Imaging In Vivo with Genetic Encoded Reporters

Workshop 227
11:15 am - 12:45 pm
Nonviral Gene Therapy
Room: 327/329
Chair: Jon A. Wolff, MD

Speakers:
Richard Heller, PhD
Electroporation Based Gene Therapy in Humans

Nagy A. Habib, ChM, FRCS
Clinical Study with Hydrodynamic Gene Delivery into Hepatocytes in Humans

Jon A. Wolff, MD
Intravascular Delivery of Naked DNA for Duchenne Muscular Dystrophy

Krys Bankiewicz, MD, PhD
Connection Enhanced Delivery of Liposomal Nanoparticles into Central Nervous System

Song Li, MD, PhD
Complex Roles of CpG Immune Response in Gene Therapy

Workshop 228
11:15 am - 12:45 pm
Oligonucleotide Based Therapies: Bringing Oligonucleotide to the Clinic
Room: 314/315 – 316/317
Chair: Cy A. Stein, MD, PhD

Speakers:
Alan Gewirtz, MD
The C-MYB Gene: From Nucleotides to Bedsides

Larry A. Couture, PhD
Producing Biologics for Gene Therapy Applications under GMP

Cy A. Stein, MD, PhD
Molecular Therapeutics with G3139: Clinical and Basic Science Results with a Bcl-2-Targeting Antisense Oligodeoxyribonucleotide

Barry Polisky, PhD
Progress Toward Therapeutic Applications of siRNA

Alexander Kolykhalov, PhD
shRNA-Inhibitors of HCV Replication

Meet-the-Investigator Lunch 230
1:00 pm - 2:00 pm
Room: 334
Speaker:
Stephen J. Russell, MD, PhD
Oncolytic Viruses: Barriers to Success

Meet-the-Investigator Lunch 231
1:00 pm - 2:00 pm
Room: 335
Speakers:
Katherine A. High, MD and Alan E. Smith, PhD
Choosing a Career: Academics versus Biotech

Simultaneous Oral Abstract Sessions
2:30 pm – 5:00 pm

Oral Abstract Session 240
AAV Vectors Biology
Room: 327/329
Moderators: John F. Engelhardt, PhD, and Jay A. Chiorini, MD
(Abstracts #1 - #10)

Oral Abstract Session 241
Adenovirus Vectors: Hybrid Vectors/Novel Serotypes/Targeting
Room : 314/315
Moderators: Maria A. Croyle, PhD, and Andrew Byrnes, MD
(Abstracts #11 - #20)

Oral Abstract Session 242
Cardiovascular Gene Therapy
Room: 337/338
Moderators: Timothy O’Brien, MD, PhD, and J. Kevin Donahue, MD
(Abstracts #21 - #30)

Oral Abstract Session 243
Muscle and Connective Tissue: Muscle
Room: 321/323
Moderators: Gyula Acsadi, MD, PhD and Gadi Pelled, DMD
(Abstracts #31 - #40)

Oral Abstract Session 244
Cancer-Apoptosis and Suicide
Room: 345/346
Moderators: Svend O. Freytag, PhD, and Lily Wu, MD, PhD
(Abstracts #41 - #50)

Oral Abstract Session 245
Cancer-Targeted Gene Therapy: Clinical Trials and Preclinical Studies I
Room: 339/342
Moderators: Estuardo Aguilar-Cordova, PhD, and Albert B. Deisseroth, MD, PhD
(Abstracts #51 - #60)

Oral Abstract Session 246
Hematologic
Room: 343/344
Moderators: Patrick Kelly, MD, and Harry L. Malech, MD
(Abstracts #61 - #70)

Oral Abstract Session 247
Vector and Transgene Immunology
Room: 324/326
Moderators: Isabelle Riviere, PhD, and Paul D. Robbins, PhD
(Abstracts #71 - #80)

Oral Abstract Session 248
Oligonucleotide Therapies: Non-Viral Targets
Room: 318/320
Moderators: Bruce Sullenger, PhD, and Peter Glazer, MD, PhD
(Abstracts #81 - #90)

Oral Abstract Session 249
Stem Cell Biology
Room: 316/317
Moderators: Carolyn Lutzko, PhD, and Jan Nolta, PhD
(Abstracts #91 - #100)

Poster Session I
5:00 pm – 8:00 pm
Hall F and Swing Hall

AAV Vectors: Biology
(Abstracts #101 through #120)

Production/Optimization of Adenoviral Vectors
(Abstracts #121 through #150, and Abstract #837)

Other DNA Viruses
(Abstracts #151 through #166)

Non-Viral Gene Delivery
(Abstracts #167 through #204)

Metabolic Disorders
(Abstracts #205 through #227)

Gene Therapy of Cardiac Muscle
(Abstracts #228 through #239)

Neurologic: Therapeutic Applications
(Abstracts #240 through #261)

Cancer-Immunotherapy: Immuno-Gene Therapy Strategies I
(Abstracts #262 through #287)

Cancer-Targeted Gene Therapy: Clinical and Preclinical Studies
(Abstracts #288 through #311)

Cell Processing and Vector Production
(Abstracts #312 through #319)

Gene Expression I
(Abstracts #320 through #340)

Stem Cells
(Abstracts #341 through #366)

• Wednesday, May 31
• Thursday, June 1
• Friday, June 2
• Saturday, June 3
• Sunday, June 4