Select Conferences | RNAi World Congress 2008

Agenda

Day One - 30 April

	RNAi Applications/Screening Chaired by Dennis Watson, Medical University of South Carolina
8:30am	Data Analysis and Experimental Designs in Genome-Wide RNAi Research Xiaohua Douglas Zhang, Associate Director, Merck Research Laboratories Present data analysis (including new development of analytic methods) and plate designs in genome-wide RNAi research and demonstrate how to implement them in RNAi HTS experiments.
9:00am	Genome-Wide Screen for Kinases Involved in Phosphorylation of Histone H2AX, using siRNA Microarrays Xavier Gidrol, Director of Laboratory, CEA, IRCM High throughput RNA interference using siRNA microarrays allowed characterization of new signalization pathways in response to DNA damage in human.
9:30am	Applications of RNAi to Functional Target Validation in Cellular Disease Models of AD Giuseppe Pollio, Principal Scientist, Siena Biotech The presentation will describe a rapid, sensitive and simple RNAi-based assay for the validation of genes putatively associated with amyloid beta-mediated neurotoxicity, allowing the generation of robust and reproducible data in primary neuronal cultures, closely representing, in vitro, the real disease situation.
10:00am	Lentiviral shRNA Screen of Multidrug Resistant Associated Genes Identifies PRP-4 as a New Regulator of Chemoresistance in Human Ovarian Cancer Zhenfeng Duan, Instructor in Orthopedic Surgery, Massachusetts General Hospital Lentiviral shRNA screen of multidrug resistant associated genes in ovarian cancer.
10:30am	Coffee Break Compliments of febit
	RNAi Tools (Session 1) Chaired by Dennis Watson, Medical University of South Carolina
11:00am	Use of Dicer-Substrate siRNAs in Animal Research Mark Behlke, Vice President, Integrated DNA Technologies Dicer-substrate siRNAs (DsiRNAs) have successfully been employed for in vivo studies using IP and IT routes of administration. In addition to use of conventional cationic lipid methods, a new nanoparticle IV delivery system will be described.
11:30am	Transfection using Magnetic Nanostructures Sarah Fredriksson, CEO and Founder, Genovis Nanostructures with a superparamagnetic core are excellent imaging agents and readily traced in animal models using MRI. Is it possible to combine the imaging features with efficient transfection properties to obtain a dual transfection/ labeling nanostructure?
12:00pm	Innovative Technology that Enables RNAi in Difficult to Transfect Cells Kirk Brown, Field Application Scientist, Thermo Scientific Dharmacon Products Recent investigations at Thermo Fisher Scientific have led to the development of an innovative molecule for lipid-independent delivery in a wide variety of cell types. These modified siRNAs have been found to effectively silence target genes in cell types that are typically difficult to transfect using standard delivery methods.
12:30pm	Lunch
	RNAi Therapeutics (Session 1) Chaired by Judy Lieberman, Harvard Medical School
1:30pm	The Silent Treatment: Delivering RNA Interference Judy Lieberman, Senior Investigator, Harvard Medical School A method for cell-specific systemic delivery of siRNAs by mixing siRNAs with an antibody fragment fused to protamine was designed to silence gene expression in vivo with exquisite specificity in cells bearing the receptor recognized by the antibody.
2:00pm	UNA (Unlocked Nucleic Acid) for Improved RNAi Applications Jesper Wengel, Professor and Chairman, RiboTask UNA (Unlocked Nucleic Acid) is an acyclic form of RNA that is remarkably useful as a novel constituent of siRNA duplexes. Results from cellular and in vivo studies will be presented to document that siUNA constructs display improved potency, reduced off-target effects and increased biostability relative to unmodified siRNA duplexes.
2:30pm	FANA-Modified Nucleic Acids as Versatile RNA-Targeting Agents Nicolay Ferrari, Director Pharmacology, Topigen Pharmaceuticals The presentation will focus on Topigen’s RNA-targeting technology and the use of 2′-deoxy-2′-fluoroarabinonucleic (FANA)-platform. They will then present TPI 1100, a novel inhaled human AON-based product containing two FANA-modified phosphorothioate AONs for the treatment of chronic obstructive pulmonary disease (COPD).
3:00pm	The Role of the CMO in the Large Scale Manufacture of siRNA Duplexes Kevin Fettes, Process Development Group Leader, Avecia Biotechnology The practicalities of manufacturing RNA duplexes at a scale required to provide materials for clinical trials and commercial launch will be discussed using case studies.
3:30pm	Coffee Break Compliments of febit
	RNAi Tools (Session 2) Chaired by Enal Razvi, Biotechnology Analyst
4:00pm	Cellaxess®HT - Automated High Throughput System for Genome Wide RNAi Screening Johan Pihl, Project Manager, Cellectricon The presentation will describe Cellaxess®HT, a fully automated system capable of genome-wide transfection in the RNAi screening process, along with gene silencing experiments carried out on a number of cell types.
4:30pm	Multiplex RNAi Michael Schlabach, Graduate Student, Harvard Medical School Multiplex RNAi offers a sensitive, robust, cost effective method for implementing whole genome knockdown studies. A review of the current state of the field and potentials for the short term future will be presented.
	The Business of RNAi Chaired by Enal Razvi, Biotechnology Analyst
5:00pm	Forecasting Technology Trends in RNAi Gary Oosta, Chief Scientific Officer, Oval Ideas Guiding research in fast-paced areas such as RNAi requires new tools and processes to identify and track technology trends and risks. A combination of text-clustering and systems analysis enables technology forecasting and reveals research and development opportunities.
5:30pm	Market Analysis of the RNAi Therapeutics Space Enal Razvi, Biotechnology Analyst, Select Biosciences This presentation will discuss the market landscape for RNAi therapeutics and will present some recent industry analysis data and highlight some of the trends in this space.

Day Two - 1 May

	microRNA Biology Chaired by Sam Gunderson, Rutgers University
8:30am	MicroRNA-Mediated Loss of PDEF Protein Expression Results in Progression to more Invasive Breast Cancer Dennis Watson, Professor, Medical University of South Carolina The group have identified two miRNAs that function as oncogenes by inhibiting the translation of mRNA of PDEF, a member of the Ets gene family that regulates cell proliferation, migration and invasion.
9:00am	Molecular Correlates of Prostate Cancer Subtypes Sunita Setlur, Post Doctoral Fellow, Brigham and Women’s Hospital, Harvard Medical School Prostate cancer is characterized by molecular subtypes that harbour gene fusions involving ETS family of transcription factors. This study focuses on identifying micro RNA profile that distinguishes fusion prostate cancer from prostate cancers that are negative for gene fusion events.
9:30am	MicroRNA Regulation in 5-FU Treated Pancreatic Cancer Cells Boa-Ling Adam, Assistant Professor, Center for Biotechnology and Genomic Medicine, Medical College of Georgia Using a mass spectrometry-based proteomic approach, the group explored a potential role of miRNA regulation in cancer therapeutic response, and concentrate on miRNA targets, which can be used to manipulate the sensitivity of pancreatic cancer cells to 5-Fluorouracil treatment.
10:00am	Regulation of Mir-1 and Mir-133 by Insulin is Altered in the Skeletal Muscle of Type 2 Diabetic Patients Sophie Rome, Research Scientist, INRA This study demonstrated that insulin regulated in vivo the expression of microRNAs in the human skeletal muscle. This regulation was not observed in the muscle of type 2 diabetic patients, revealing their potential roles in molecular defects of insulin resistance.
10:30am	Coffee Break and Networking in the Exhibition Hall Compliments of febit
11:15am	Conserved Roles for Small RNAs in Genome Defense Gregory Hannon, Professor, Cold Spring Harbor Laboratory Through studies of a clade of Argonaute proteins, which shows largely gonad-restricted expression patterns, the group identified a class of small RNAs with conserved roles in transposon suppression. These piRNAs form the basis of an adaptive immune system that silences transposons.
11:45am	Intra-Axonal RNA Interference: Regulation of Intra-Axonal mRNA Translation in Regenerating Axons Alexander Murashov, Associate Professor, East Carolina University A concept of local regulation of intra-axonal translation via RNAi.
12:15pm	Downregulation of Cardiomyocyte-Enriched microRNAs Contributes to Altered Gene Expression in Heart Failure Sadakatsu Ikeda, Research Fellow, Children's Hospital Boston/Harvard Medical School Results suggest that altered miRNA expression contributes to abnormal gene expression in heart failure, and add to the growing evidence that miRNAs may be broadly involved in the pathogenesis of human disease.
12:45pm	Lunch and Networking in the Exhibition Hall
1:45pm	Poster Viewing
2:15pm	Gene Silencing with U1 Adaptor Oligonucleotides Sam Gunderson, Associate Professor, Rutgers University The group developed a novel gene silencing method that uses oligonucleotides to inhibit polyadenylation in the nucleus in a gene specific fashion. The method can be used alone or synergistically with other gene silencing methods.
2:45pm	Pressing the Genetic Off Switch! RNA-Mediated Long-Term Epigenetic Silencing in Human Cells Kevin Morris, Assistant Professor, The Scripps Research Institute Small RNA targeting of the UbC promoter in human cells results in long-term gene silencing the correlates with targeted histone and DNA methylation at the targeted loci and requires Ago-1 and the epigenetic related proteins DNMT3a and HDAC-1.
3:15pm	miRNAs: Cancer Biomarkers and Functional Regulators of Cancer Development Irena Ivanovska, Senior Research Biologist, Rosetta Inpharmatics The group found that the miR-106b family promotes cell-cycle progression, consistent with their over-expression in tumors and correlation with the expression of cell-cycle genes. The cyclin-dependent kinase inhibitor p21/CDKN1A is a key target of the miR-106b family for the cell-cycle function.
3:45pm	Coffee Break and Networking in the Exhibition Hall Compliments of febit
4:30pm	Alteration of Cell Phenotype through the Stable Ectopic Expression of microRNAs Graham Brock, Director, Target and Drug Discovery, Ordway Research Institute and miRNAcor The group have identified cancer relevant microRNAs by stably expressing them in cell lines and examining phenotypic change. In one example, mesenchymal cells become more epithelial in both appearance and growth characteristics. Potential changes in drug response in these cells will be discussed.
5:00pm	Dual Role for Argonaute Proteins in microRNA Processing and Post-Transcriptional Regulation of microRNA Expression Sven Diederichs, Research Fellow, Harvard Medical School Argonaute proteins, known effectors of RNA interference, are newly characterized as post-transcriptional regulators of mature miRNA expression. Ago2, the RNase in RNAi, also cleaves the pre-miRNA into a novel precursor and thus actively participates in microRNA processing.
5:30pm	Drinks Reception Compliments of Hokkaido System Science

Day Three - 2 May

	microRNA Tools Chaired by Mark Behlke, Integrated DNA Technologies
9:00am	miRNAs in the Marsupial Monodelphis Domestica Mark Behlke, Vice President, Integrated DNA Technologies Marsupial miRNAs were cloned from Monodelphis domestica using the new miRCat miRNA Cloning Kit. 85 distinct miRNAs were identified, of which 72 are shared in common with other species and 13 appear to be unique to marsupials.
9:30am	MicroRNA Labeling Methods Influence the Accuracy and Sensitivity of their Detection Shannon Bruse, Senior Scientist, Mirus Bio Corporation MicroRNA microarray expression profiling is dependent on robust labeling of the RNA sample. The presentation will discuss the implications of recent findings that enzymatic labeling methods systematically miss specific microRNAs, while chemical labeling results in unbiased detection of all microRNAs.
10:00am	Coffee Break and Networking in the Exhibition Hall Compliments of febit
	RNAi Therapeutics (Session 2) Chaired by Mark Behlke, Integrated DNA Technologies
10:45am	Development of a Novel RNAi Therapeutics Platform Dmitry Samarsky, Vice President, Technology Development, RXi Pharmaceuticals The design and effective delivery of synthetic RNAi compounds are important factors for therapeutic applications. RXi will present data using their proprietary rxRNA™ compounds. rxRNAs can be up to 100 times more potent than conventional siRNAs, demonstrate nuclease resistance, and are potentially more specific for their intended targets.
11:15am	Development of Liposomal siRNA for RNAi Mediated Therapeutic Applications Ansgar Santel, Senior Scientist, Silence Therapeutics Lipsomal formulation represents one strategy for the development of an RNAi based therapeutics. The AtuPLEX (siRNA-lipoplex) technology is currently being developed as a therapeutic modality in oncology.
11:45am	Development of an siRNA Targeting p53 to Protect Cells from Acute Injury James Thompson, VP Pharmaceutical Development, Quark Pharmaceuticals This is the first siRNA ever administered to patients systemically (IV). Talk will include tissue distribution and residence time of the siRNA, results from safety and toxicology studies, and patient data from the Phase I/II studies.
12:15pm	Lunch and Networking in the Exhibition Hall
1:15pm	Poster Viewing
	RNAi Therapeutics (Session 3) Chaired by Dmitry Samarsky, RXi Pharmaceuticals
1:45pm	RNAi: From Mechanism to Medicine Craig Mello, Professor, University of Massachusetts RNAi offers astounding potential for understanding and manipulating the genetic basis of disease and yet there are still many mysteries regarding its underlying mechanism. This talk will describe the discovery of RNAi and explore those remaining mysteries.
2:45pm	Silencing of Tyrosinase in a Three Dimensional Organotypic Skin Model Christine Collin-Djangoné, Project Manager, L'Oréal Research Silencing of tyrosinase in a pigmented reconstructed skin model was successfully performed using human primary transfected melanocytes with potent Stealth RNA against tyrosinase. This result shows a phenotypically long term lasting effect of RNAi (> 2 weeks).
3:15pm	Poster Award
3:25pm	Coffee Break and Networking in the Exhibition Hall Compliments of febit
3:55pm	Liposome-Based Manufacturing and Delivery Technologies for Oligonucleotide Therapeutics Wendi Rodrigueza, Vice President, Product Development, ProNAi Therapeutics This paper will discuss the development of liposome-based manufacturing and delivery technologies for emerging oligonucleotide therapeutics for various forms of cancer.
4:25pm	Treatment of Gastrointestinal Targets using RNA Interference Johannes Fruehauf, VP, Research, Cequent Pharmaceuticals Transkingdom RNA interference (tkRNAi) is a novel technology to deliver therapeutic RNA interference into the gastrointestinal tract. It uses live attenuated bacteria to produce and deliver RNAi from the luminal side to the GI mucosa, which allows oral application of RNAi drugs.
4:55pm	Development of RNAi-Based Therapeutics for Hepatitis C Brian Johnston, Chief Executive Officer/President, SomaGenics SomaGenics have designed shRNAs that potently inhibit hepatitis C virus based on replicon and mouse reporter models. Parameters including target sequence, stem length, loop size, and end features have been optimized, providing IC50s that are substantially below 1 nanomolar.
5:25pm	Close of Conference