Web Desc
Therapeutic Modulation of RNA Using Oligonucleotides
Organizer(s): C. Frank Bennett and Thomas Tuschl
Date: February 08 - 13, 2009
Location: Fairmont Chateau Lake Louise, Lake Louise, AB, Canada
Sponsored by Alnylam Pharmaceuticals, Inc., Isis Pharmaceuticals, Inc. and Santaris Pharma A/S
For important information on the coronavirus, please click here
Summary of Meeting:
The recent discoveries of the RNAi pathway in mammalian cells and the finding that oligonucleotides can activate the innate immune system through Toll-like receptors have resulted in increased interest in oligonucleotide-based therapeutics. There are two basic therapeutic approaches in which oligonucleotides are being utilized: (1) binding to RNA resulting in selective inhibition of gene expression; and (2) modulating protein function by direct binding to proteins. Although both approaches share common manufacturing, toxicology and ADME issues, oligonucleotides that are designed to bind RNA inside the cell have a fundamental different set of challenges than oligonucleotides designed to bind to proteins outside the cell. This meeting will focus on the former application. A goal of this meeting is to bring together scientists from both the basic science disciplines and applied disciplines to gain a common understanding of the attributes and challenges the technology faces. This meeting will consider basic mechanisms by which oligonucleotides can be exploited to modulate gene expression, mechanisms for distribution of oligonucleotides to tissues and within cells and how chemistry and/or formulations can improve the pharmaceutical properties of oligonucleotides. Finally recent advances in both preclinical applications and clinical results will be presented.
Scholarship Deadline: October 9 2008
Discounted Abstract Deadline: October 9 2008
Abstract Deadline: November 6 2008
Discounted Registration Deadline: December 8 2008
We gratefully acknowledge additional support for this conference from:
Alnylam Pharmaceuticals, Inc.ChemGenes CorporationIntegrated DNA TechnologiesIsis Pharmaceuticals, Inc.Santaris Pharma A/S
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

Alnylam Pharmaceuticals, Inc.

Bristol-Myers Squibb Company

Isis Pharmaceuticals, Inc.

Merck & Co, Inc. /Sirna Therapeutics

novosom AG

Regulus Therapeutics Inc.
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:
Cell PressRoyal Society of Chemistry
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations
Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations

Program

Sunday, February 08 | 3:00PM - 7:30PM
Registration
Room: Alpine Gallery


Sunday, February 08 | 6:30PM - 7:30PM
Refreshments
Room: Heritage Hall


Sunday, February 08 | 7:30PM - 7:45PM
Opening Remarks
Room: Mount Temple A-B

Speaker 1 of 2
* C. Frank Bennett, Ionis Pharmaceuticals, Inc., USA

Sunday, February 08 | 7:30PM - 7:45PM
Opening Remarks
Room: Mount Temple A-B

Speaker 2 of 2
* Thomas Tuschl, HHMI/Rockefeller University, USA

Sunday, February 08 | 7:45PM - 9:45PM
Keynote Session
Room: Mount Temple A-B

Speaker 1 of 2
Thomas R. Gingeras, Cold Spring Harbor Laboratory, USA
The RNA World

Sunday, February 08 | 7:45PM - 9:45PM
Keynote Session
Room: Mount Temple A-B

Speaker 2 of 2
Stanley T. Crooke, Ionis Pharmaceuticals, Inc., USA
Antisense Technology: Past, Present, and Future

Monday, February 09 | 7:00AM - 8:00AM
Breakfast
Room: Tom Wilson Restaurant


Monday, February 09 | 8:00AM - 11:00AM
Natural RNA Regulatory Pathways
Room: Mount Temple A-B

Speaker 1 of 4
Jack D. Keene, Duke University Medical Center, USA
Post-Transcriptional Coordination of Gene Expression

Monday, February 09 | 8:00AM - 11:00AM
Natural RNA Regulatory Pathways
Room: Mount Temple A-B

Speaker 2 of 4
* Thomas Tuschl, HHMI/Rockefeller University, USA
Small RNA Sequencing Resolves Posttranscriptional Regulatory Networks

Monday, February 09 | 8:00AM - 11:00AM
Natural RNA Regulatory Pathways
Room: Mount Temple A-B

Speaker 3 of 4
Dinshaw J. Patel, Memorial Sloan Kettering Cancer Center, USA
Structural Biology of Argonaute-Mediated RNA Silencing

Monday, February 09 | 8:00AM - 11:00AM
Natural RNA Regulatory Pathways
Room: Mount Temple A-B

Speaker 4 of 4
Laura Ranum, University of Florida, USA
Double the Trouble: Bidirectional Expression of the SCA8 CAG/CTG Expansion Mutation - Evidence for RNA and Protein Gain of Function Effects

Monday, February 09 | 9:20AM - 9:40AM
Coffee Break
Room: Heritage Hall


Monday, February 09 | 11:00AM - 11:00AM
On Own for Lunch


Monday, February 09 | 1:00PM - 10:00PM
Poster Viewing
Room: Mount Temple C


Monday, February 09 | 4:30PM - 5:00PM
Coffee Available
Room: Heritage Hall


Monday, February 09 | 5:00PM - 7:15PM
Medicinal Chemistry of Oligonucleotides
Room: Mount Temple A-B

Speaker 1 of 4
Steven A. Benner, Foundation for Applied Molecular Evolution, USA
Synthetic Biology for Human Diagnostics Applications

Monday, February 09 | 5:00PM - 7:15PM
Medicinal Chemistry of Oligonucleotides
Room: Mount Temple A-B

Speaker 2 of 4
Jesper Wengel, University of Southern Denmark, Denmark
LNA (Locked Nucleic Acid) and UNA (Unlocked Nucleic Acid)

Monday, February 09 | 5:00PM - 7:15PM
Medicinal Chemistry of Oligonucleotides
Room: Mount Temple A-B

Speaker 3 of 4
* Muthiah Manoharan, Alnylam Pharmaceuticals, Inc., USA
Medicinal Chemistry of siRNAs

Monday, February 09 | 5:00PM - 7:15PM
Medicinal Chemistry of Oligonucleotides
Room: Mount Temple A-B

Speaker 4 of 4
Eric E. Swayze, Ionis Pharmaceuticals, Inc., USA
Short Talk: Conformational Constraint of Oligonucleotides with Non-Bicyclic Nucleosides: Hexitol Nucleoside Containing Antisense Oligonucleotides Improve Potency in Animals.

Monday, February 09 | 7:15PM - 8:15PM
Social Hour with Lite Bites
Room: Mount Temple C


Monday, February 09 | 7:30PM - 10:00PM
Poster Session 1
Room: Mount Temple C


Tuesday, February 10 | 7:00AM - 8:00AM
Breakfast
Room: Tom Wilson Restaurant


Tuesday, February 10 | 8:00AM - 11:00AM
Mechanisms of Biodistribution of Oligonucleotides
Room: Mount Temple A-B

Speaker 1 of 4
Steven F. Dowdy, University of California, San Diego, USA
Peptide Transduction Domain Delivery of siRNA Macromolecules

Tuesday, February 10 | 8:00AM - 11:00AM
Mechanisms of Biodistribution of Oligonucleotides
Room: Mount Temple A-B

Speaker 2 of 4
* C. Frank Bennett, Ionis Pharmaceuticals, Inc., USA
Mechanisms of Oligonucleotide Distribution To Cells

Tuesday, February 10 | 8:00AM - 11:00AM
Mechanisms of Biodistribution of Oligonucleotides
Room: Mount Temple A-B

Speaker 3 of 4
Markus Stoffel, ETH Zürich, Switzerland
Mechanisms of Lipophile Oligonucleotide Biodistribution

Tuesday, February 10 | 8:00AM - 11:00AM
Mechanisms of Biodistribution of Oligonucleotides
Room: Mount Temple A-B

Speaker 4 of 4
HaiFang Yin, Oxford University, UK
Short Talk: A Tissue-Specific Targeting Peptide Domain Directs Enhanced Systemic Antisense Oligonucleotide-Induced Splice Correction of the DMD Gene and Functional Restoration in Dystrophin-Deficient mdx Mice

Tuesday, February 10 | 9:20AM - 9:40AM
Coffee Break
Room: Heritage Hall


Tuesday, February 10 | 11:00AM - 12:00PM
Lunch
Room: Tom Wilson Restaurant


Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 1 of 6
* Victor Kotelianski, Promedior, USA

Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 2 of 6
Devin Leake, Catalog Technologies Inc, USA
Design and Optimization of siRNA Oligonucleotides

Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 3 of 6
Steffen Panzner, Novosom AG, Germany
Approaches in the Systemic Delivery Oligonucleotides: Chemistries, Mechanisms and in vivo Applications

Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 4 of 6
Xiao-Dong Yang, Intradigm Corporation, UK
RNAi Nanoplex Technology for Systematic Delivery of siRNA

Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 5 of 6
Susan M. Freier, Ionis Pharmaceuticals, Inc., USA
Identification of Antisense Oligonucleotides for Target Validation Using In Silico and In Vitro Methods

Tuesday, February 10 | 12:00PM - 2:00PM
Workshop: Issues with Target Validation Using Oligonucleotid
es
Room: Mount Temple A-B

Speaker 6 of 6
Xavier J. de Mollerat du Jeu, Life Techonologies, USA
Invivofectamine™-New Delivery Reagent for in vivo Delivery of Stealth™RNAi

Tuesday, February 10 | 2:00PM - 2:30PM
Coffee Available
Room: Heritage Hall


Tuesday, February 10 | 2:30PM - 4:30PM
Intersection of RNAi/miRNA Silencing with other Cellular Pat
hways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses)

Room: Mount Temple A-B

Speaker 1 of 4
Gunther Hartmann, Universitätsklinikum Bonn, Germany
Oligonucleotide Interaction with Innate Immune Receptors

Tuesday, February 10 | 2:30PM - 4:30PM
Intersection of RNAi/miRNA Silencing with other Cellular Pat
hways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses)

Room: Mount Temple A-B

Speaker 2 of 4
Aimee Jackson, miRagen Therapeutics, USA
microRNA Therapeutics: Opportunities and Challenges

Tuesday, February 10 | 2:30PM - 4:30PM
Intersection of RNAi/miRNA Silencing with other Cellular Pat
hways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses)

Room: Mount Temple A-B

Speaker 3 of 4
* Mark A. Kay, Stanford University, USA
Pharmacological Effects of siRNA/shRNA Expression on RISC Pathway

Tuesday, February 10 | 2:30PM - 4:30PM
Intersection of RNAi/miRNA Silencing with other Cellular Pat
hways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses)

Room: Mount Temple A-B

Speaker 4 of 4
Ivone Guzman Bruno, Baylor College of Medicine, USA
Short Talk: A Brain-Enriched miRNA Regulates mRNA Surveillance Pathways

Wednesday, February 11 | 7:00AM - 8:00AM
Breakfast
Room: Tom Wilson Restaurant


Wednesday, February 11 | 8:00AM - 11:00AM
Target Validation
Room: Mount Temple A-B

Speaker 1 of 4
* Stephen W. Fesik, Vanderbilt University School of Medicine, USA
Identification and Validation of Oncology Targets using RNAi

Wednesday, February 11 | 8:00AM - 11:00AM
Target Validation
Room: Mount Temple A-B

Speaker 2 of 4
Siew Peng Ho, Bristol-Myers Squibb Company, USA
Oligonucleotide based Approaches for in Vivo Validation of Drug Targets

Wednesday, February 11 | 8:00AM - 11:00AM
Target Validation
Room: Mount Temple A-B

Speaker 3 of 4
John Cryan, University College Cork, Ireland
Validating Neuronal Targets

Wednesday, February 11 | 8:00AM - 11:00AM
Target Validation
Room: Mount Temple A-B

Speaker 4 of 4
Varman T. Samuel, Yale University, USA
Short Talk: Validating Novel Metabolic Targets with Antisense Oligonucleotides: Surprises and Insights

Wednesday, February 11 | 9:20AM - 9:40AM
Coffee Break
Room: Heritage Hall


Wednesday, February 11 | 11:00AM - 11:00AM
On Own for Lunch


Wednesday, February 11 | 11:00AM - 1:00PM
Poster Setup
Room: Mount Temple C


Wednesday, February 11 | 1:00PM - 10:00PM
Poster Viewing
Room: Mount Temple C


Wednesday, February 11 | 4:30PM - 5:00PM
Coffee Available
Room: Heritage Hall


Wednesday, February 11 | 5:00PM - 7:00PM
Nucleic Acids Formulations
Room: Mount Temple A-B

Speaker 1 of 3
Alan Sachs, Thermo Fisher Scientific, USA
Small Interfering RNA as a Therapeutic Modality

Wednesday, February 11 | 5:00PM - 7:00PM
Nucleic Acids Formulations
Room: Mount Temple A-B

Speaker 2 of 3
* Daniel G. Anderson, Massachusetts Institute of Technology, USA
Combinatorial Development of Synthetic siRNA Delivery Systems

Wednesday, February 11 | 5:00PM - 7:00PM
Nucleic Acids Formulations
Room: Mount Temple A-B

Speaker 3 of 3
Masahiko Kuroda, Tokyo Medical University, Japan
Short Talk: Therapeutic Silencing of an Endogenous Gene by the siRNA Cream in an Arthritis Model Mouse

Wednesday, February 11 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Mount Temple C


Wednesday, February 11 | 7:30PM - 10:00PM
Poster Session 2
Room: Mount Temple C


Thursday, February 12 | 7:00AM - 8:00AM
Breakfast
Room: Tom Wilson Restaurant


Thursday, February 12 | 8:00AM - 11:00AM
Non-Traditional RNA Targets as Therapeutic Opportunities
Room: Mount Temple A-B

Speaker 1 of 5
* Ryszard Kole, Ercole Biotech Consulting, USA
Splice Switching Oligonucleotides as Potential Therapeutics

Thursday, February 12 | 8:00AM - 11:00AM
Non-Traditional RNA Targets as Therapeutic Opportunities
Room: Mount Temple A-B

Speaker 2 of 5
Christine Esau, Arcturus Therapeutics, USA
Therapeutic Targeting of MicroRNAs

Thursday, February 12 | 8:00AM - 11:00AM
Non-Traditional RNA Targets as Therapeutic Opportunities
Room: Mount Temple A-B

Speaker 3 of 5
Chang-Zheng Chen, Stanford University, USA
Micromanaging the Immune System by microRNA Genes

Thursday, February 12 | 8:00AM - 11:00AM
Non-Traditional RNA Targets as Therapeutic Opportunities
Room: Mount Temple A-B

Speaker 4 of 5
Noga Yerushalmi, Rosetta Genomics, Israel
The Use of Antisense Oligonucleotides Against miRNA as a Potential Therapy for Hepatocellular Carcinoma

Thursday, February 12 | 8:00AM - 11:00AM
Non-Traditional RNA Targets as Therapeutic Opportunities
Room: Mount Temple A-B

Speaker 5 of 5
Eva van Rooij, Hubrecht Institute, Netherlands
Short Talk: The Myriad Roles of MicroRNAs in Heart Disease

Thursday, February 12 | 9:20AM - 9:40AM
Coffee Break
Room: Heritage Hall


Thursday, February 12 | 11:00AM - 11:00AM
On Own for Lunch


Thursday, February 12 | 4:30PM - 5:00PM
Coffee Available
Room: Heritage Hall


Thursday, February 12 | 5:00PM - 7:15PM
Clinical Trials
Room: Mount Temple A-B

Speaker 1 of 5
* John A. Schmidt, National Institutes of Health, USA
Clinical Development Introduction

Thursday, February 12 | 5:00PM - 7:15PM
Clinical Trials
Room: Mount Temple A-B

Speaker 2 of 5
Akshay Vaishnaw, Alnylam Pharmaceuticals Inc., USA
A Phase II Trial of ALN-RSV01, an RNAi Therapeutic for Respiratory Syncytial Virus

Thursday, February 12 | 5:00PM - 7:15PM
Clinical Trials
Room: Mount Temple A-B

Speaker 3 of 5
Nuket Desem, Spinifex Pharmaceuticals Pty Ltd, Australia
ATL1102, an Antisense Drug Targeting VLA-4, Significantly Reduces the Development of Active Lesions in Relapsing-Remitting Multiple Sclerosis

Thursday, February 12 | 5:00PM - 7:15PM
Clinical Trials
Room: Mount Temple A-B

Speaker 4 of 5
Sancy Ann Leachman, Oregon Health & Science University, USA
Short Talk: First-in-Skin siRNA Clinical Trial

Thursday, February 12 | 5:00PM - 7:15PM
Clinical Trials
Room: Mount Temple A-B

Speaker 5 of 5
Annemieke Aartsma-Rus, Leiden University Medical Center, Netherlands
Short Talk: Clinical Trials for Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy

Thursday, February 12 | 7:15PM - 8:15PM
Social Hour with Lite Bites
Room: Victoria Room


Thursday, February 12 | 8:00PM - 11:00PM
Entertainment
Room: Victoria Room


Thursday, February 12 | 8:00PM - 11:00PM
Cash Bar
Room: Victoria Room


Friday, February 13 | 10:22AM - 10:22AM
Departure


*Session Chair
†Invited, not yet responded.