Conference Documentation: RNAi, siRNA and miRNA (2010)
SMI Publishing, Ltd
June 28, 2010 SKU: SMI2773646
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This is SMi's fifth conference on the subject of RNAi, siRNA and miRNA. It will build on the success of the past four years, and brings together the leading experts in industry and academia to explore the cutting edge of new therapeutics being researched and brought to the marketplace.
Taking place in London, UK, this conference will look at a wide range of issues within the RNAi arena. It will combine a study of the latest successes in the research and development phase with presentations covering the key themes of delivery, target identification and IP issues.
Questions examined during the conference will include: What are the best delivery methods for siRNA and RNA-based therapeutics? How do you carry out gene silencing? What is the best method to use an siRNA library for target identification? How is cell stress related to decreased RNAi? What are the patent issues for oligonucleotides? How do miRNAs regulate gene expression?
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- DAY 1
- 8.30 Registration and coffee
- 9.00 Chairman's Opening Remarks
- Michael Keller, Senior Fellow, siRNA Project Leader, Technical Research and Development (TRD), Novartis.
- 9.10 OPENING KEYNOTE: Nanomedicinal siRNA delivery technologies and pulmonary siRNA delivery systems
- Development of nano- and micro-particle technology for siRNA delivery
- Nanotechnology based siRNA formulation in the clinics
- Active and passive targeting strategies for siRNA delivery
- Michael Keller, Senior Fellow, siRNA Project Leader, Technical Research and Development (TRD), Novartis.
- 9.40 KEYNOTE PRESENTATION: The promise and challenges of therapeutic siRNA for the treatment of respiratory disease
- Respiratory disease is an attractive opportunity for siRNA therapeutics, as molecules can be delivered locally by the inhaled or intranasal route - achieving high concentrations in the target tissue and avoiding the challenges associated with systemic delivery
- Nevertheless, the airways are adept at responding to and preventing the entry of oligonucleotides - many groups are addressing the challenge of turning the promise of therapeutic siRNA in respiratory disease into a clinical reality
- This presentation will review progress with respiratory siRNA therapeutics, discuss the challenges of development and consider how GSK is addressing them
- Ken Clark, Director, Respiratory siRNA DPU, GlaxoSmithKline.
- 10.10 Unique challenges associated with large-scale manufacturing of difference RNA sequences
- Review of the differences between DNA and RNA synthesis and of the impact of modifications into RNA sequences
- Specifics of annealing on manufacturing and QC of siRNA
- Keys to a successful manufacturing of Large-Scale siRNA
- Marc Lemaitre, Chef Executive Officer, Girindus America.
- 10.40 Morning Coffee
- 11.10 Targeting of junction oncogenes by siRNA
- Oncogenes present in cancers with chromosomal translocations
- Examples: Ewing sarcoma and papillary thyroid carcinoma
- Animal and human models
- Claude Paul Malvy, Director, IFSBM, Université Paris-Sud.
- 11.40 Gene silencing with reduced off-target effects and high potency using UNA-modified siRNA
- UsiRNAs are siRNAs which contain one or more UNA (Unlocked Nucleic Acid; Unlocked Nucleobase Analog) monomers
- Learn how to design highly potent UNA-modified siRNAs (UsiRNAs) which display significantly reduced off-target effects
- UsiRNAs typically contain strategic UNA monomers to induce strand selectivity and/or to prevent miRNA-type effects
- UsiRNAs can be modified in the 3'-ends for increased biostability
- Results from cell culture and in vivo experiments will be shown
- Jesper Wengel, Professor, Nucleic Acide Centre, University of Southern Denmark.
- Suzy Lena Wengel, Chef Executive Officer, RiboTask.
- 12.10 Preclinical development of SYL040012, a siRNA designed for glaucoma treatment
- Introduction to SYL040012
- R&D in vitro/in vivo data
- Preclinical development, IMPD submission, clinical update
- Victoria González, Preclinical Manager, Sylentis.
- 12.40 Networking Lunch
- 2.00 Design and manufacturing of siRNA nanomedicines
- Formulation strategies - lipid and polymer based delivery systems
- From concept to market: scaling-up and manufacturing
- Marie-Andrée Yessine, Scientist, OctoPlus Development.
- 2.30 KEYNOTE PRESENTATION: Transfection takes the leap: A 384-well HT Nucleofector® system for genome-wide siRNA screenings
- Efficient mRNA knock-down in hard-to-transfect cell types
- 384-well format and increased speed for genome-wide screenings
- Advanced protocols for large experiments using a single cell batch
- Herbert Müller-Hartmann, Director R&D, Lonza.
- 3.00 Afternoon Tea
- 3.30 Combination of oligo configuration and chemical modification provides drug-like properties to RNAi molecules
- RNAi compounds with improved stability, specificity, potency and distribution
- Self-delivering RNAi molecules (sd-rxRNA)
- Novel configurations for RNAi compounds: short duplex or single-oligo
- Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals.
- 4.00 Cell stress is related to decreased RNAi in mammalian cells
- Sub-cellular localisation of Argonaute-2
- miRNA- and siRNA-induced RNA interference
- Transfection of oligoneucleotides
- Stress granules and P-bodies
- Georg Sczakiel, Professor, Institute for Molecular Medicine, Lübeck University.
- 4.30 GeneICE - Gene Inactivation through Chromatin Engineering
- Induced epigenetic regulation of gene expression
- Genome-wide mapping of nuclease accessible sites for target discovery
- Intracellular delivery - the final hurdle?
- Mark Eccleston, Director of Business Development, ValiRx.
- 5.00 Chairman’s Closing Remarks and Close of Day One
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- DAY 2
- 8.30 Re-registration and Coffee
- 9.00 Morning Chairman's opening remarks
- Marion Jurk, Director, Discovery & Development, Pfizer Oligonucleotide Therapeutics Unit.
- 9.10 KEYNOTE PRESENTATION: Pharmaceutical development of a novel systemic oligonucleotide delivery formulation
- Buccal delivery (non-invasive)
- Delivery to difficult tissues (e.g. muscle)
- Non-toxic formulation (full GLP tox)
- Stable and manufactured to GMP
- Peter Jackson, Chief Operating Officer, Medesis Pharma.
- 9.40 Dicer substrate oligonucleotides: technology and applications
- Potency and specificity
- In vivo results in normal and tumour-bearing animals
- Delivery options
- Therapeutic applications
- Roberto Guerciolini, Senior Vice President, Pharmaceutical Development, Dicerna Pharmaceuticals.
- 10.10 Patents and Oligonucleotides: the law and the reality
- What is patentable?
- The problems in RNA patenting - exclusions, sufficiency of disclosure and industrial application
- What infringes?
- How to negotiate a workable deal
- Lorna Brazell, Partner, Bird & Bird.
- 10.40 Morning Coffee
- 11.10 KEYNOTE PRESENTATION: Immunology of nucleic acids
- Immune modulatory effects of siRNAs - and avoiding them
- Combining modulatory effects with gene knockdown
- Additional immune modulatory activities
- Marion Jurk, Director, Discovery & Development, Pfizer Oligonucleotide Therapeutics Unit.
- 11.40 New approaches for development of nucleic acid based therapeutics: challenges and opportunities in RNA targeting
- RNA as a potential pharmaceutical target
- Gene-specific approaches for silencing of disease-relevant mRNA sequences
- Peptidyl-oligonucleotide biomimetics as a new approach for gene expression knockdown
- Elena Bichenkova, Senior Lecturer in Medicinal Chemistry, University of Manchester.
- 12.10 Developing a novel antisense technology-based drug
- Targeting cancer via TGF-β2
- Novel approach - directly tackles pathogenic cause
- Clinical and regulatory experience
- Risk mitigation in clinical development
- Karl-Hermann Schlingensiepen, Chef Executive Officer, Antisense Pharma.
- 12.40 Networking Lunch
- 1.55 Afternoon Chairman's welcome
- Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals.
- 2.00 Progress in the utility of microRNA as biomarkers
- Sterghios Moschos, Principal Scientist, Pfizer.
- 2.30 Anti-miR inhibition of microRNA function in immune cells
- Delivery of anti-miRs to immune cells in vivo
- Measurement of anti-miR target engagement by expression profiling
- Measurement of anti-miR function by phenotypic assays
- Aimee Jackson, Director of Drug Discovery, Regulus Therapeutics.
- 3.00 Afternoon Tea
- 3.30 The role of miRNAs in the regulation of gene expression
- Mechanism of action
- Involvement in human diseases
- Latest findings from the Cancer Research UK Viral Oncology Group
- Dimitris Lagos, Co-Principal Investigator, UCL Cancer Institute.
- 4.00 miR-155 regulates the immune system
- miR-155 was first described as miRNA with oncogenic potential - a role in the immune system has been established
- Data showing how miR-155 regulates lymphocyte function
- miRNAs may have an impact on gene therapies designed to block tumour progression
- Synthetic oligonucleotides of different chemistries have proven successful for blocking miRNA expression
- Data showing use of Peptide Nucleic Acids (PNA) to block miR-155 function in lymphocytes in vivo
- Elena Vigorito, Group Leader, Lymphocyte Signalling & Development, Babraham Institute.
- 4.30 miR-155 keeps inflammation in check
- miRNA-profiling of human dendritic cells by microarrays
- TAB2 is a target of miR-155 in activated human dendritic cells
- Identification of a miR-155-mediated negative feedback loop that helps to modulate the inflammatory response
- Targeting miRNA-155 signaling may be useful for decreasing autoimmune-associated inflammation
- Maurizio Ceppi, Project Team Manager (Group Leader), R&D, Genomic Vision.
- 5.00 Chairman’s Closing Remarks and Close of Day Two
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