Keystone Symposia | Scientific Conferences on Biomedical and Life Science Topics

Therapeutic Modulation of RNA Using Oligonucleotides (B7)

Organizer(s): C. Frank Bennett and Thomas Tuschl
February 8 - 13, 2009
Fairmont Chateau Lake Louise · Lake Louise, AB
Abstract Deadline: October 9, 2008
Late Abstract Deadline: November 6, 2008
Scholarship Deadline: October 9, 2008
Early Registration Deadline: December 8, 2008

Sponsored by Alnylam Pharmaceuticals, Inc., Isis Pharmaceuticals, Inc. and Santaris Pharma A/S

This meeting took place in the 2009 season.

Listed below are current meetings that are similar to this meeting in nature/content:

MicroRNAs and Human Disease (J6)
February 11 - 16, 2011

For a complete list of the meetings for the upcoming/current season,
see our meeting list, or search for a meeting.

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.

Sunday, February 8
3:00 - 7:30 PM	Registration	Alpine Gallery
6:30 - 7:30 PM	Refreshments	Heritage Hall
7:30 - 7:45 PM	Opening Remarks	Mount Temple A-B
	* C. Frank Bennett, ISIS Pharmaceuticals, Inc.
	* Thomas Tuschl, Rockefeller University
7:45 - 9:45 PM	Keynote Session	Mount Temple A-B
	Thomas R. Gingeras, Cold Spring Harbor Laboratory The RNA World
	Stanley T. Crooke, ISIS Pharmaceuticals Antisense Technology: Past, Present, and Future
Monday, February 9
7:00 - 8:00 AM	Breakfast	Tom Wilson Restaurant
8:00 - 11:00 AM	Natural RNA Regulatory Pathways	Mount Temple A-B

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

	* Thomas Tuschl, Rockefeller University Small RNA Sequencing Resolves Posttranscriptional Regulatory Networks
	Dinshaw J. Patel, Memorial Sloan-Kettering Cancer Center Structural Biology of Argonaute-Mediated RNA Silencing
	Laura Ranum, University of Minnesota Double the Trouble: Bidirectional Expression of the SCA8 CAG/CTG Expansion Mutation - Evidence for RNA and Protein Gain of Function Effects
9:20 - 9:40 AM	Coffee Break	Heritage Hall
11:00 AM - 1:00 PM	Poster Setup	Mount Temple C
11:00 AM -	On Own for Lunch
1:00 - 10:00 PM	Poster Viewing	Mount Temple C
4:30 - 5:00 PM	Coffee Available	Heritage Hall
5:00 - 7:15 PM	Medicinal Chemistry of Oligonucleotides	Mount Temple A-B
	Steven A. Benner, Foundation for Applied Molecular Evolution Synthetic Biology for Human Diagnostics Applications
	Jesper Wengel, University of Southern Denmark LNA (Locked Nucleic Acid) and UNA (Unlocked Nucleic Acid)
	* Muthiah Manoharan, Alnylam Pharmaceuticals, Inc. Medicinal Chemistry of siRNAs
	Eric E. Swayze, ISIS Pharmaceuticals Short Talk: Conformational Constraint of Oligonucleotides with Non-Bicyclic Nucleosides: Hexitol Nucleoside Containing Antisense Oligonucleotides Improve Potency in Animals.
7:15 - 8:15 PM	Social Hour w/ Lite Bites	Mount Temple C
7:30 - 10:00 PM	Poster Session 1	Mount Temple C
Tuesday, February 10
7:00 - 8:00 AM	Breakfast	Tom Wilson Restaurant
8:00 - 11:00 AM	Mechanisms of Biodistribution of Oligonucleotides	Mount Temple A-B

	Steven F. Dowdy, University of California, San Diego Peptide Transduction Domain Delivery of siRNA Macromolecules
	* C. Frank Bennett, ISIS Pharmaceuticals, Inc. Mechanisms of Oligonucleotide Distribution To Cells
	Markus Stoffel, Institut f. Molekulare Systembiologie Mechanisms of Lipophile Oligonucleotide Biodistribution
	HaiFang Yin, Oxford University 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
9:20 - 9:40 AM	Coffee Break	Heritage Hall
11:00 AM - 12:00 PM	Lunch	Tom Wilson Restaurant
12:00 - 2:00 PM	Workshop: Issues with Target Validation Using Oligonucleotides	Mount Temple A-B
	* Victor Kotelianski, Alnylam Pharmaceuticals Inc.

	Devin Leake, Thermo Fisher Scientific, Dharmacon Products Design and Optimization of siRNA Oligonucleotides
	Steffen Panzner, Novosom AG Approaches in the Systemic Delivery Oligonucleotides: Chemistries, Mechanisms and in vivo Applications
	Xiao-Dong Yang, Intradigm Corporation RNAi Nanoplex Technology for Systematic Delivery of siRNA
	Susan M. Freier, ISIS Pharmaceuticals Identification of Antisense Oligonucleotides for Target Validation Using In Silico and In Vitro Methods
	Xavier J. de Mollerat du Jeu, Invitrogen Invivofectamine™-New Delivery Reagent for in vivo Delivery of Stealth™RNAi
2:00 - 2:30 PM	Coffee Available	Heritage Hall
2:30 - 4:30 PM	Intersection of RNAi/miRNA Silencing with other Cellular Pathways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses)	Mount Temple A-B
	Gunther Hartmann, Universitätsklinikum Bonn Oligonucleotide Interaction with Innate Immune Receptors
	Aimee Jackson, Regulus Therapeutics, Inc. microRNA Therapeutics: Opportunities and Challenges
	* Mark A. Kay, Stanford University Pharmacological Effects of siRNA/shRNA Expression on RISC Pathway
	Ivone Guzman Bruno, Baylor College of Medicine Short Talk: A Brain-Enriched miRNA Regulates mRNA Surveillance Pathways

Wednesday, February 11
7:00 - 8:00 AM	Breakfast	Tom Wilson Restaurant
8:00 - 11:00 AM	Target Validation	Mount Temple A-B

	* Stephen W. Fesik, Vanderbilt University School of Medicine Identification and Validation of Oncology Targets using RNAi
	Siew Peng Ho, Bristol-Myers Squibb Company Oligonucleotide based Approaches for in Vivo Validation of Drug Targets
	John Cryan, University College Cork Validating Neuronal Targets
	Varman T. Samuel, Yale University Short Talk: Validating Novel Metabolic Targets with Antisense Oligonucleotides: Surprises and Insights
9:20 - 9:40 AM	Coffee Break	Heritage Hall
11:00 AM -	On Own for Lunch
11:00 AM - 1:00 PM	Poster Setup	Mount Temple C
1:00 - 10:00 PM	Poster Viewing	Mount Temple C
4:30 - 5:00 PM	Coffee Available	Heritage Hall
5:00 - 7:00 PM	Nucleic Acids Formulations	Mount Temple A-B

	Alan Sachs, Merck Research Laboratories/ Sima Therapeutics Small Interfering RNA as a Therapeutic Modality
	* Daniel G. Anderson, Massachusetts Institute of Technology Combinatorial Development of Synthetic siRNA Delivery Systems


	Masahiko Kuroda, Tokyo Medical University Short Talk: Therapeutic Silencing of an Endogenous Gene by the siRNA Cream in an Arthritis Model Mouse
7:00 - 8:00 PM	Social Hour w/ Lite Bites	Mount Temple C
7:30 - 10:00 PM	Poster Session 2	Mount Temple C
Thursday, February 12
7:00 - 8:00 AM	Breakfast	Tom Wilson Restaurant
8:00 - 11:00 AM	Non-Traditional RNA Targets as Therapeutic Opportunities	Mount Temple A-B

	* Ryszard Kole, Avi Biopharma Splice Switching Oligonucleotides as Potential Therapeutics
	Christine Esau, Regulus Therapeutics Therapeutic Targeting of MicroRNAs

	Chang-Zheng Chen, Stanford University Micromanaging the Immune System by microRNA Genes
	Noga Yerushalmi, Rosetta Genomics The Use of Antisense Oligonucleotides Against miRNA as a Potential Therapy for Hepatocellular Carcinoma
	Eva van Rooij, Miragen Therapeutics Short Talk: The Myriad Roles of MicroRNAs in Heart Disease
9:20 - 9:40 AM	Coffee Break	Heritage Hall
11:00 AM -	On Own for Lunch
4:30 - 5:00 PM	Coffee Available	Heritage Hall
5:00 - 7:15 PM	Clinical Trials	Mount Temple A-B

	* Jack A. Schmidt, Alnylam Pharmaceuticals, Inc. Clinical Development Introduction
	Akshay Vaishnaw, Alnylam Pharmaceuticals Inc. A Phase II Trial of ALN-RSV01, an RNAi Therapeutic for Respiratory Syncytial Virus



	Nuket Desem, AntiSense Therapeutics ATL1102, an Antisense Drug Targeting VLA-4, Significantly Reduces the Development of Active Lesions in Relapsing-Remitting Multiple Sclerosis
	Sancy Ann Leachman, University of Utah Short Talk: First-in-Skin siRNA Clinical Trial

	Annemieke Aartsma-Rus, Leiden University Medical Center Short Talk: Clinical Trials for Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy
7:15 - 8:15 PM	Social Hour w/ Lite Bites	Victoria Room
8:00 - 11:00 PM	Entertainment	Victoria Room
Friday, February 13
	Departure

*Session Chair †Speaker invited, not yet responded.