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RNAi, siRNA & miRNA
10 June - 11 June 2009
RNAi, siRNA & miRNA
RNA interference (RNAi) has the potential to become big business for all those involved.  Less than 10 years after its discovery by Andrew Fire, Craig Mello and co-workers, since rewarded for their research with a Nobel Prize in 2006, this discovery is being touted as the new innovation craved by the pharmaceutical industry. 
 

RNAi therapeutics research is still in the early stages of development, and therefore currently essentially not validated, making learning from the mistakes and successes of others working in this field is invaluable. With the market estimated to be worth more than $3 billion in 2007, and growing, this is one area of research Pharma can’t afford not to be involved in.

 
SAE Media Group’s 4th RNAi, siRNA & miRNA conference aims to bring together industry experts from both large Pharma and smaller biotechs to discuss the successes and pitfalls of their research into this area.  Covering developments in therapeutics, and providing a critical overview of current RNAi therapeutics this conference could prove to be the insight needed to boost your companies profile in the RNAi and related oligonucleotides field.
  • Jörg Vollmer, Managing Director, Coley Pharmaceuticals (A Pfizer Company)
  • Troels Koch, VP, Research Division, Santaris Pharma
  • Michael Keller, Senior Fellow, Global siRNA Expert Technical Research&Development, Novartis

Conference agenda

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8:30

Registration & Coffee

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9:00

Introduction to Exiqon Pharmaceutical Sciences

  • Merged technology platform (LNA enabling products and services for microRNA analysis combined with a tumor bank of 150,000 tumors as a resource for discovery)
  • Extreme Drug Resistance (EDR) Assay
  • microRNA vs mRNA, why Exiqon believe microRNA is better for drug development purposes
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    9:30

    MicroRNA Research using LNA Technology

  • Benefits of Locked Nucleic Acid (LNA)
  • Background of LNA array platform
  • Robust profiling of microRNAs isolated from archival FFPE material
  • microRNA knockdown, and microRNA PCR detection using LNA technologies
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    10:00

    MicroRNA Opportunities in pre-clinical and clinical research

  • Profiling of stable microRNAs in live tumors (from the Exiqon tumor bank) with validated models of clinical efficacy prediction (EDR assay) to correlate profile to drug response, and give information about drug mechanism
  • Identify microRNA signature for classification of resistant / responsive tumors
  • Identify microRNA biomarkers that may predict drug response
  • Use microRNA biomarkers to select/deselect patients for inclusion in trials resulting in smaller, more cost-effective trials with increased response rate
  • microRNAs as prognostic biomarkers
  • Clinical trial monitoring: blood/tumor samples sent to Exiqon from clinical trials can be accessioned and processed for microRNA arrays or other molecular assays
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    10:30

    Morning Coffee

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    11:00

    Blood-based screening of microRNA biomarkers

  • microRNA detection in blood, plasma and serum
  • What this means for clinical trials and diagnostic development
  • Monitor the activity of the drug through blood-based screening for microRNA
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    11:30

    Tissue opportunities

  • Tumor bank details; size, scope, availability
  • EDR Assay: determine which tumor types will respond best to a drug
  • Immunohistochemistry, in situ hybridization, custom assay validations available through Exiqon’s CLIA- and CAP- certified laboratory
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    12:00

    Review and Questions

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    12:30

    Close of Workshop

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    13:30

    Registration & Coffee

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    14:00

    Welcome and Introductions

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    14:10

    KEYNOTE ADDRESS(ES): THE CURRENT STATE OF OLIGONUCLEOTIDE DELIVERY IN VITRO AND IN VIVO

  • Technology that failed so far
  • Technology currently being tested
  • New concepts and future strategies
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    14:50

    OLIGONUCLEOTIDE DELIVERY

  • Cell- and ligand-targeted modes
  • HTS-based search for delivery options
  • Examples teaching promising future directions
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    15:40

    Afternoon Tea

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    16:00

    NEW LESSONS FROM BASIC RESEARCH FOR THE APPLICATION

  • Cellular uptake of naked nucleic acids
  • Intracellular trafficking and biological effectiveness
  • Improved delivery by novel chemistry
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    16:50

    DISCUSSION – ROUND-TABLE

  • The major technical problems to be solved
  • The role of novel chemistry
  • Does novel biology help to solve the delivery problem?
  • Is successful delivery related to the classes of oligonucleotide-based drugs?
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    17:00

    Close of Workshop

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Jörg Vollmer

    Jörg Vollmer, Vice President, Coley Pharmaceutical

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    9:10

    SILENCING RNA EXPRESSION

    Troels Koch

    Troels Koch, VP, Research Division, Santaris Pharma

  • Silencing the expression of mRNA and microRNA has great therapeutic potential
  • An important component for executing the promise of RNA silencing is a molecular platform with high affinity combined with extensive design freedom
  • Short single stranded LNA represents such a platform - offering both mRNA and microRNA silencing
  • Data showing novel design concepts will be presented and the gene silencing mechanism will be discussed
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    9:50

    COMPREHENSIVE RNA CHARACTERIZATION USING THE GENOME SEQUENCER FLX NEXT GENERATION SEQUENCING SYSTEM

    Bruce Taillon

    Bruce Taillon, Director of Healthcare Business Development, Roche

  • Review of the Genome Sequencer FLX
  • Discussion of 454 Sequencing RNA characterization applications
  • Discussion of specific smRNA and miRNA studies
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    10:30

    Morning Coffee

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    11:00

    CHALLENGES & PROGRESS IN THE RNAi THERAPEUTIC ARENA

    Amina Benahmed

    Amina Benahmed, Associate Director Nucleic Acid Delivery, Coley Pharmaceutical

  • Challenges in design
  • Issues and challenges in the delivery of RNAi drugs
  • Overcoming drug-induced immune activation
  • Manufacturing and formulation challenges
  • Regulatory issues and path forward
     
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    11:40

    LINKING miRNA AND TOXICOLOGY

    Tejdip Singh

    Tejdip Singh, Medical Director, Pharmacovigilance, Genzyme

  • Emerging role of miRNA in toxicogenomics
  • Using miRNA signatures to help define patient risk:benefit
  • Examples of risk of therapy identified using miRNA technology
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    12:20

    Networking Lunch

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    13:50

    HIGH TROUGHPUT SCREENING OF miRNA ANTAGONISTS

    Chris Kitson

    Chris Kitson, Investigator, Immunoinflammation CEDD, GlaxoSmithKline

  • Running miRNA antagonist and mimic screens
  • Identifying miRNA as possible therapeutic targets
  • Understanding the role of miRNA in inflammatory disease
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    14:30

    A TOOLBOX FOR INHIBITING THE miRNA PATHWAY

    Petr Svoboda

    Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics

  • miRNA pathway introduction
  • Inhibition by eliminating protein components of the pathway
  • Protein inhibitors
  • Inhibition by blocking miRNAs with modified oligonucleotides
  • Small-compound inhibitors
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    15:10

    ROLE OF miRNA IN DIGNOSTICS

    Sterghios Moschos

    Sterghios Moschos, Senior Scientist, Pfizer Ltd

  • Identifying miRNA signatures for disease
  • Illustrating patterns of disease using miRNA
  • Use as basis for further clinical development
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    15:50

    Afternoon Tea

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    16:10

    STUDYING THE EXPRESSION AND FUNCTION OF THE MIR-214/199 CLUSTER

    Younbok Lee

    Younbok Lee, Research Associate, University of Bristol

  • Characterise the promoters controlling miR-214/199 cluster expression
  • In particular we have studied the control of expression by the transcription factor Twist1
  • Detail the differential expression of miR-214/199, Twist1 and upstream transcription factors during embryonic development
  • The function of MiR-214 targeted in stressed neuronal cells
     
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    16:50

    miRNA IN DIAGNOSTICS

    Søren Møller

    Søren Møller, Vice President, Research & Development, Exiqon

  • Abnormal expression of microRNAs is seen in cancer
  • This implies they play a role in oncogenesis
  • miRNAs may therefore comprise a novel class of diagnostic and prognostic signatures
  • Examples of using miRNA for cancer classification, prognosis and treatment selection
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    17:30

    microRNA IN CANCER AND OTHER DISEASES

    Irena Ivanovska

    Irena Ivanovska, Senior Research Biologist, Merck

  • microRNA function in cell cycle progression
  • microRNA target genes regulate molecular networks
  • Using miRs to identify new drug targets
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    18:10

    Chairman’s Closing Remarks and Close of Day One

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    8:30

    Registration & Coffee

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    9:00

    Chairman's Opening Remarks

    Mark Edbrooke

    Mark Edbrooke, Head of Gene Modulation, GlaxoSmithKline

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    9:10

    DEALING WITH OFF-TARGET EFFECTS OF siRNA THERAPEUTICS

    Jörg Vollmer

    Jörg Vollmer, Vice President, Coley Pharmaceutical

  • siRNAs and their non-target-related biological effects
  • Role of nucleic acid receptors present in endosomes and cytoplasm
  • Ways to avoid immune activation by chemical modifications or use of selected delivery systems
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    9:50

    DEVELOPMENT OF THE AtuPLEX FOR RNAi BASED THERAPIES

    Ansgar  Santel

    Ansgar Santel, Senior Scientist, Silence Therapeutics

  • Requirement of siRNA delivery vehicles; liposomal formulation of siRNA
  • Endothelial targeting by the AtuPLEX (siRNA-lipoplex)~: functional RNAi in vivo
  • RNAi therapeutics in disease areas with dysfunctional endothelium
  • Update on ST's current development programme Atu027 in oncology
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    10:30

    Morning Coffee

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    11:00

    DICER SUBSTRATE RNAS AS NOVEL THERAPEUTICS

    Roberto Guerciolini

    Roberto Guerciolini, SVP Pharmaceutical Development , Dicerna Pharmaceuticals

  • Natural entry to the RNAi pathway
  • Chemical configuration for optimised RISC loading and picomolar potency
  • Chemical optimisation for prolonged effect and evasion of immunostimulatory
  • Therapeutic applications
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    11:40

    THERAPEUTIC DEVELOPMENT OF siRNA DRUGS

    Michael Keller

    Michael Keller, Senior Fellow, Global siRNA Expert Technical Research & Development, Novartis Pharma

  • Recent preclinical progress in systemic and topical siRNA delivery for PoP
  • Current clinical siRNA studies
  • Analytical developments for the analysis of lipids in formulations
  • Progress in formulation for animal and human studies
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    12:20

    Networking Lunch

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    13:50

    THE COMPLEXATION APPROACH FOR siRNA DELIVERY

    Patrick Erbacher

    Patrick Erbacher, Manager, Research, Polyplus-Transfection SAS

  • The complexation approach: advantages and limits
  • Cationic amphiphile or polymer
  • Sticky siRNA: improvement of complex stability and gene silencing efficiency
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    14:30

    FUNCTIONAL DELIVERY OF siRNA IN VIVO USING SOFT, TRIGGERABLE OR TARGETED NANOPARTICLES

    Andy Miller

    Andy Miller, Professor of Organic Chemistry & Chemical Biology, Imperial College London

  • The transformation of gene therapy approaches to disease by siRNA
  • Crafting delivery solutions from tool kits of chemical components
  • ABC and ABCD nanoparticles effect delivery to liver and tumour in vivo
  • What are the next steps for other organs?
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    15:10

    Afternoon Tea

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    15:40

    TUMOR TARGETED RNA POLYPLEXES AS PROGRAMMED NANOMEDICINES

    Ernst Wagner

    Ernst Wagner, Chair, Pharmaceutical Biology - Biotechnology, University of Munich

  • Bioresponsive dynamic RNA formulations
  • Tumor-targeted and receptor-mediated delivery
  • Intra- and extra-cellular barriers
  • Therapeutic strategies using siRNA or dsRNA
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    16:20

    CATIONIC siRNAS FOR EFFECTIVE GENE SILENCING

    Joachim Engels

    Joachim Engels, Professor, University of Frankfurt

  • Easy to synthesise
  • Stable in sera
  • Very good silencing
  • Facilitated uptake
  • Easy to combine with other chemistries
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    17:00

    Chairman’s Closing Remarks and Close of Conference

    Workshops

    MicroRNAs and Oncology Drug Development

    MicroRNAs and Oncology Drug Development

    Crowne Plaza Hotel - St James
    9 June 2009
    London, United Kingdom

    Delivery of Oligonucleotide-Based Drugs

    Delivery of Oligonucleotide-Based Drugs

    Crowne Plaza Hotel - St James
    9 June 2009
    London, United Kingdom

    Crowne Plaza Hotel - St James

    Buckingham Gate 45/51
    London SW1E 6AF
    United Kingdom

    Crowne Plaza Hotel - St James

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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