Keystone Symposia Conference | Therapeutic Modulation of RNA Using Oligonucleotides

Meeting Program

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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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B
	* C. Frank Bennett, ISIS Pharmaceuticals, Inc., USA
	* Thomas Tuschl, Rockefeller University, USA
7:45 - 9:45 PM	Keynote Session Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B
	Thomas R. Gingeras, Cold Spring Harbor Laboratory, USA The RNA World
	Stanley T. Crooke, ISIS Pharmaceuticals, USA 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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B

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

	* Thomas Tuschl, Rockefeller University, USA Biochemistry of RNAi and miRNPs
	Dinshaw J. Patel, Memorial Sloan-Kettering Cancer Center, USA Structural Biology of Argonaute-Mediated RNA Silencing
	Laura Ranum, University of Minnesota, USA 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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B
	Steven A. Benner, Foundation for Applied Molecular Evolution, USA Synthetic Biology for Human Diagnostics Applications
	Jesper Wengel, University of Southern Denmark, Denmark LNA (Locked Nucleic Acid) and UNA (Unlocked Nucleic Acid)
	* Muthiah Manoharan, Alnylam Pharmaceuticals Inc., USA Medicinal Chemistry of siRNAs
	Eric E. Swayze, ISIS Pharmaceuticals, USA Short Talk: Conformational Constraint of Oligonucleotides with Non-Bicyclic Nucleosides: Hexitol Nucleoside Containing Antisense Oligonucleotides Improve Potency in Animals.
	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
7:15 - 8:15 PM	Social Hour w/ Lite Bites	Mount Temple C
7:30 - 10:00 PM	Poster Session 1 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B
	Lucas Pelkmans, Institute of Molecular Systems Biology, ETH Zurich, Switzerland The Human Infectome: Using RNAi Screens to Dissect Virus Entry Pathways
	Steven F. Dowdy, University of California, San Diego, USA Peptide Transduction Domain Delivery of siRNA Macromolecules
	* C. Frank Bennett, ISIS Pharmaceuticals, Inc., USA Mechanisms of Oligonucleotide Distribution To Cells
	Markus Stoffel, Institut f. Molekulare Systembiologie, Switzerland Mechanisms of Lipophile Oligonucleotide Biodistribution
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., USA

	Devin Leake, Thermo Fisher Scientific, Dharmacon Products, USA Design and Optimization of siRNA Oligonucleotides
	Steffen Panzner, Novosom AG, Germany Approaches in the Systemic Delivery Oligonucleotides: Chemistries, Mechanisms and in vivo Applications
	Xiao-Dong Yang, Intradigm Corporation, USA RNAi Nanoplex Technology for Systematic Delivery of siRNA
	Susan M. Freier, ISIS Pharmaceuticals, USA Identification of Antisense Oligonucleotides for Target Validation Using In Silico and In Vitro Methods
	Xavier J. de Mollerat du Jeu, Invitrogen, USA Invivofectamine™-New Delivery Reagent for in vivo Delivery of Stealth™RNAi
2:00 - 2:30 PM	Coffee Available	Heritage Hall
2:30 - 4:45 PM	Intersection of RNAi/miRNA Silencing with other Cellular Pathways (Innate Immunity, Editing, Intercellular Traffic of RNA, Viruses) Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B
	Gunther Hartmann, Universitätsklinikum Bonn, Germany Oligonucleotide Interaction with Innate Immune Receptors
	Aimee Jackson, Regulus Therapeutics, USA microRNA Therapeutics: Opportunities and Challenges
	* Mark A. Kay, Stanford University, USA Pharmacological Effects of siRNA/shRNA Expression on RISC Pathway
	Ivone Guzman Bruno, University of Texas MD Anderson Cancer Center, USA Short Talk: A Brain-Enriched miRNA Regulates mRNA Surveillance Pathways
	Nicole-Claudia Meisner†, Novartis Institutes for Biomedical Research, Switzerland Short Talk: mRNA Openers –Small RNAs Acting as Posttranscriptional Activators
Wednesday, February 11
7:00 - 8:00 AM	Breakfast	Tom Wilson Restaurant
8:00 - 11:00 AM	Target Validation Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B

	* Stephen W. Fesik, Abbott Laboratories, USA Identification and Validation of Oncology Targets using RNAi
	Siew Peng Ho, Bristol-Myers Squibb Company, USA Oligonucleotide based Approaches for in Vivo Validation of Drug Targets
	John Cryan, University College Cork, Ireland Validating Neuronal Targets
	Varman T. Samuel, Yale University, USA 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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B

	Alan Sachs, Merck Research Laboratories/ Sima Therapeutics, USA Small Interfering RNA as a Therapeutic Modality
	* Daniel G. Anderson, Massachusetts Institute of Technology, USA Combinatorial development of synthetic siRNA delivery systems

	Anne-Laure D. Bolcato-Bellemin†, Bioparc-BP90018, France Short Talk: Non Viral Systems for Enhanced siRNA Delivery
	Masahiko Kuroda†, Tokyo Medical University, Japan 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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	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 Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B

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

	Chang-Zheng Chen, Stanford University, USA Micromanaging the Immune System by microRNA Genes
	Noga Yerushalmi, Rosetta Genomics, Israel The Use of Antisense Oligonucleotides Against miRNA as a Potential Therapy for Hepatocellular Carcinoma
	Eva van Rooij, University of Texas Southwestern Medical Center/miRagen Therapeutics, Inc., USA 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 - 7:00 PM	Clinical Trials Registered attendees for this meeting can view Abstracts for this session starting on 01/08/2009	Mount Temple A-B

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



	Nuket Desem, AntiSense Therapeutics, Australia 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, USA Short Talk: First-in-Skin siRNA Clinical Trial
	Henrik Orum, Santaris Pharma A/S, Denmark Short Talk: Progress in miRNA Therapeutics
7:00 - 8:00 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.

Keystone Symposia would like to thank the sponsors of this meeting for their generous support:

We gratefully acknowledge additional support for this conference from:

	Bristol-Myers Squibb Company
Merck & Co, Inc. /Sirna Therapeutics	novosom AG
Regulus Therapeutics LLC

We gratefully acknowledge the organizations that provide Keystone Symposia with additional support, such as marketing and advertising...

Click here to view these companies

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.

Therapeutic Modulation of RNA Using Oligonucleotides (B7)