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Delivery of Oligonucleotide-Based Drugs
9 June 2009
Delivery of Oligonucleotide-Based Drugs

The therapeutic use of chemically synthesized oligonucleotides was boosted when target gene suppression mediated by silencing RNA (siRNA)-induced RNA interference (RNAi) was first reported. This extended the repertoire of molecular mechanisms based on which oligonucleotides can be applied as molecular tools and drugs. Fundamental progress on the biological level, however, is still not reflected by solutions on the major technical hurdle, i.e. the efficient functional delivery of oligonucleotides to target cells and tissues in vivo.

With regard to the overall non-satisfactory methodology for oligonucleotide delivery it is not surprising that antisense oligonucleotides as a cargo moved back into the focus of current nucleic acid-based drug development and the search for suitable delivery technology.

This workshop aims to describe new mechanistic concepts for the cellular and intracellular delivery of oligonucleotides including siRNA and antisense oligonucleotides. We will attempt to derive new lessons and insights from examples of the successful delivery of oligonucleotides in vivo. Finally, a round-table-discussion is aimed to define the current state of knowledge and to draw conclusion for promising future directions and strategies of developing methodology for delivery.

About the Workshop Leader:

Georg Sczakiel is the director of the Institute of Molecular Medicine of the University of Lubeck and he has been working in the fields of antisense-RNA, ribozymes, antisense oligonucleotides and siRNA for twenty years. He received the diploma in chemistry at the University of Freiburg and his Ph.D. at the Max-Planck-Institute of Medical Research in Heidelberg. He continued his career in the field of HIV and HPV virology at the German Cancer Resarch Center where his mentor for thirteen years was the Novel laureate Prof. Harald zur Hausen.
Among his scientific activities Georg serves as an executive editor of Nucleic Acids Search and Chemie in unserer Zeit, he is guest executive editor of Current Pharmaceutical Design, and editorial board member of Oligonucleotides and the new journal Journal of Nucleic Acids. He was one of five co-founders of the Oligonucleotide Therapeutics Society (OTS).

Workshop agenda

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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

    Crowne Plaza Hotel - St James

    Buckingham Gate 45/51
    London SW1E 6AF
    United Kingdom

    Crowne Plaza Hotel - St James

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