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In 2012, SMi's RNAi, miRNA and siRNA conference will focus on the utility of nanotechnology to aid delivery and the development of novel therapeutics in the field. This event will provide a unique opportunity for debate and problem solving for key challenges facing RNAi development, as well as allowing leading experts from RNAi and Nanotechnolgy to work together to discuss current issues.

With senior industry executives all providing case-study led presentations on their current work in a variety of different therapeutic areas; from oncology to respiratory and novel therapeutic strategies in Epigenetics and Computational Support this year’s conference is not to be missed!

The event will provide a complete picture of developments in the RNAi field, focusing on siRNA Development, Nano-enabled Nucleic Acid Delivery as well asas technological, regulatory and intellectual property updates and will be the ideal opportunity for problem-solving discussion and idea-sharing debate.

 Feedback on last year's event included:

"A thoroughly enjoyable and informative event with a diverse group of attendees that helped shed light on various analysis of this complex field"

"Well represented by high collar attendees”

"A very positive experience.  Thank you organisers!"

     

 

          

Key Topics to be Covered: 

RNAi & Antisense Technologies
 
RNAi as a tool for target validation
 
Nanotech formulation and process engineering
 
Liquid and polymeric nanoparticle siRNA lead optimization
 
Delivering oligonucleotides across the BBB
 
Antisense RNA delivery by nanoconstructs
 
Epigenetic target modulation
 
Regulatory and IP issues

 

Hear Keynote Addresses from:

Mostafa Nakach, Head of Pharmaceutical Engineering Group, Sanofi-Aventis
Michael Keller,
Senior Fellow, Novartis
Dmitry Samarsky, Executive Vice President, RiboBio
Paul Wilson,
Senior Investigator, GlaxoSmithKline
Troels Koch,
VP, Research, Santaris Pharma
Elena Feinstein,
CSO, Quark Pharmaceuticals
Klaus Giese,
Chief Scientific Officer, Silence Therapeutics
Sterghios Moschos,
Reader of Industrial Biotechnology, University of Westminster
Nagy Habib,
GI Lead Clinician, Imperial College London
Samir El-Andaloussi,
Senior Postdoctoral Fellow, University of Oxford
Stephen Hart,
Reader in Molecular Genetics, University College London
Alessandra Ferlini,
Associate Professor, University of Ferrara
Raymond Schiffelers, Associate Professor, UMC Utrecht
Jeffrey Rosedale,
Partner, Woodcock Washburn
Brian Kelley,
Associate, Covington and Burling

Who should attend this event?

 Chief Executives, Vice Presidents, Heads, Directors, Chief Scientists and Project Leaders of

 

 

  • RNAi
  • miRNA/microRNA
  • siRNA
  • RNA silencing
  • Genomics technologies
  • Bioinformatics
  • Gene Modulation
  • Discovery Chemistry
  • Molecular Medicine
  • Molecular Discovery Research
                          

 

  • Molecular Biology  and Chemistry
  • Bioinformatics/Neuroinformatics
  • Regulatory Affairs
  • Chemical Engineering
  • Drug Delivery
  • Pharmaceutical Nanoscience
  • Formulation
  • Medicinal Chemistry
  • Preclinical Development
  • Process R&D

  

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Sterghios Moschos

Sterghios Moschos, Reader in Industrial Biotechnology, University of Westminster

9:10 miRNA therapeutic strategies: from identification to delivery

Raymond Schiffelers

Raymond Schiffelers, Associate Professor Clinical Chemistry, UMC Utrecht

  • miRNA identification
  • miRNA activity screening in vitro
  • Delivery systems for miRNA
  • 9:50 siRNA in regenerative medicine

    Nagy Habib

    Nagy Habib, GI Lead Clinician, Imperial College London

  • Using oligonucleotides in adult CD34+ stem cells differentiation
  • Developing insulin secreting cells responsive to glucose media.
  • Administering oligonucleotides as drugs in the liver, diabetes and neuro-degenerative conditions
  • 10:30 Morning Coffee

    11:00 Targeting RNA with locked nucleic acid: enabling translational research

    Troels Koch

    Troels Koch, VP, Research Division, Santaris Pharma A/S

  • LNA oligonucleotides are potent inhibitors of coding and non-coding RNA
  • SPC3649 is the first micro-RNA inhibitor in man and has shown a safe and significant reduction in HCV viral load in patients
  • The clinical and pre-clinical data closely interrelate and this has formed the basis for the fast and coherent development path for the drug
  • Case study that suggests LNA oligonucleotides targeting disease related microRNAs may enable translational research from bench to clinic
  • 11:40 Non-viral delivery of self-amplifying RNA vaccines

    Andrew Geall

    Andrew Geall, Platform Technology Leader, Novartis Vaccines And Diagnostics Inc

  • Novartis has developed a self-amplifying mRNA (SAMT) vaccine platform
  • The Platform takes advantage of cell-free RNA production from a transcription reaction and delivery with a synthetic delivery system
  • The broad utility of this novel vaccine technology has been demonstrated with genes encoding antigens from several pathogens and found to elicit broad and potent protective immune responses
  • Responses are comparable to a viral delivery technology, but without the inherent limitations of viral vectors
  • 12:20 Networking Lunch

    13:30 Structure-activity relationships of chitosan/siRNA nanoparticles in vitro and vivo

    Michael Keller

    Michael Keller, Senior Fellow, siRNA Project Leader, Technical Research and Development (TRD), Novartis Pharma

  • Selection of most appropriate chitosan source and N:P ratio is pivotal for a succesful siRNA delivery system
  • Structural feature of the siRNA polyplexes (SAXs, TEM) and cellular trafficking
  • Preliminary in vivo experiments
  • 14:10 DPC technology for siRNA delivery: from rodents to primates

    David Lewis

    David Lewis, Vice President and Site Head, Arrowhead Research

    Maturation of a targeted, polymer-based siRNA delivery platform
    Safety and efficacy studies for delivery to liver and tumors
    Pre-clinical studies of a DPC-based therapeutic for HBV

    14:50 Insights in shRNA processing and siRNA mode of action using Next Generation Sequencing

    Hubert Denise

    Hubert Denise, Researcher, Sanger Institute

  • The precision of key enzymes involved in the maturation and mechanism of action of RNAi therapeutics
  • A focus on an active viral based therapeutic program soon to enter clinical trials
  • Questions on the specificity of RNAi
  • 15:30 Afternoon Tea

    15:50 Lipid-peptide nanocomplexes for the targeted delivery of siRNA

    Stephen Hart

    Stephen Hart, Reader in Molecular Genetics, University College London

  • Developing receptor-targeted nanocomplexes
  • Comparative structural and functional studies
  • Tumor-specific delivery
  • 16:30 Nanoparticles for the delivery of oligonucleotides targeting EWS/Fli-1

    Claude Paul Malvy

    Claude Paul Malvy, Director IFSBM Dean of the Medicine Facility, Universite Paris Sud

  • The prevalence of EWS/Fli-1 oncogene in Ewing sarcoma patients
  • The need to protect antisense oligonucleotides and siRNA until they reach their EWS/Fli-1 mRNA target
  • Biodegradable and biocompatible polyalkylcyanoacrylate nanoparticles coated with chitozan
  • Developing cationic  nanodiamond vectors and evaluating  their  efficiency  for anionic siRNA delivery
  • 17:10 Chairman’s Closing Remarks and Close of Day One

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Dmitry Samarsky

    Dmitry Samarsky, Executive Vice President, RiboBio

    9:10 Topical dosing siRNA and LNA antisense to the lung: the liver is the target

    Sterghios Moschos

    Sterghios Moschos, Reader in Industrial Biotechnology, University of Westminster

  • A novel method for assessing oligonucleotide loading and efficacy in specific cell types
  • Evidence of rapid elimination and emiction of both therapetics from the lung
  • Efficacious loading of LNA antisense in the liver following administration in the lung
  • 9:50 Analysis of a paired miRNA/mRNA skin trauma dataset

    Paul Wilson

    Paul Wilson, Senior Investigator, GlaxoSmithKline

  • Case study on humans during a tape stripping procedure
  • Integrated analysis identified correlations between miRNA and mRNA
  • Discussion of results that show indication of possible transcriptional regulatory mechanisms
  • 10:30 Morning Coffee

    11:00 The direct visualisation, sizing and counting of drug delivery nanoparticles by Nanoparticle Tracking Analysis

    Bob Carr

    Bob Carr, CTO, Nanosight Ltd

    • A novel laser based microscopical technique will be described which allows nanoscale particles to be directly visualised, sized and counted in real-time with minimal sample preparation
    • The technique is rapid and ideal for polydisperse sample types in complex backgrounds
    • The technique can be used in fluorescence mode allowing suitably labelled sub-populations to be detected
    • Similarly, the zeta-potential of such nanoparticles can be made at the same time as being sized and counted

    11:40 RNAi and antisense technologies

    Dmitry Samarsky

    Dmitry Samarsky, Executive Vice President, RiboBio

  • What can RNAi learn from antisense technologies and vice versa
  • Developments in blockers and cleavers
  • Nanotechnological development in RNAi therapeutics
  • 12:20 Networking Lunch

    13:40 Pre-clinical and clinical development of Atu027; a novel RNAi therapeutic currently being tested in Phase 1 clinical trial in oncology

    Volker Fehring

    Volker Fehring, Associate Director Formulation Development, Silence Therapeutics AG

  • The drug targets the expression of PKN3 in the vascular endothelium
  • Drug shows strong anti-tumor and anti-metastatic activity in various pre-clinical models
  • Latest developments in Atu027 will be discusses; RNA interference, delivery systems, oncology and clinical trials
  • 14:20 Small-molecule DNA-interactive agents as alternatives to RNAi or antisense technologies: example of a novel NFkB inhibitor

    David Thurston

    David Thurston, Professor of Drug Discovery, King's College London

  • Introduction to small-molecule gene regulation
  • Drug targets being evaluated with this technology
  • Advantages and disadvantages compared to RNAi and Antisense Technologies
  • Example of a novel NFkB inhibitor with significant in vitro cytotoxicity and in vivo antitumour activity
  • 15:00 Afternoon Tea

    15:30 What should you be doing in view of the new US Patent Laws

    Jeffrey Rosedale

    Jeffrey Rosedale, Partner, Woodcock Washburn LLP

  • Ths US patent law was greatly revised for the first time in 60 years; some provisions took effect immediately in 2011, others are taking effect in 2012 and 2013
  • A number of changes in the US patent laws give rise to new IP strategies for companies in RNAi and Nanotechnology
  • Key changes affecting IP strategies include: first inventor-to-file, post-grant cancellation/re-exam procedures, pre-issuance 3rd party submissions and prior-use defenses
  • 16:10 Commercialising nanotechnology

    Brian Kelly

    Brian Kelly, Associate, Covington & Burling Llp

  • Regulatory framework
  • Challenges: classification, testing and risk management
  • Routes to patient use
  • Reimbursement
  • Case studies and lessons learnt from other sectors
  • 16:50 Chairman’s Closing Remarks and Close of Day Two

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    Workshops

    Novel siRNA Delivery Techniques
    Workshop

    Novel siRNA Delivery Techniques

    Copthorne Tara Hotel
    13th June 2012
    London, United Kingdom

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    Copthorne Tara Hotel

    Scarsdale Place, Kensington, London, United Kingdom

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    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    London W8 5SR
    United Kingdom

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    WHAT IS CPD?

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