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Washington Court Hotel

Sep 25, 2013 8:00 AM - Sep 27, 2013 5:00 PM

525 New Jersey Avenue NW, Washington, DC 20001

DIA/FDA Oligonucleotide-based Therapeutics Conference

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Keynote Address

Session Chair(s)

Arthur A. Levin, PHD

Arthur A. Levin, PHD

Distinguished Scientist

Avidity Biosciences, United States

Over the past 3 decades since the first in vitro antisense experiments were performed there has been remarkable progress in the field of oligonucleotide therapeutics. Significant advancements have been made in everything from sourcing raw materials through design of drug delivery systems. The initial concepts of antisense working through steric interference that was conceived in the early days has been supplanted by a multiple approaches to employ oligonucleotides with mechanisms that range from aptameric interactions with protein targets, enzymatic degradation of RNA targets, to oligonucleotide mediated alterations in RNA processing. A greater understanding of the multiple roles of RNA that extend beyond the transfer of information from the genome to RNA’s role in regulating gene expression has broadened the horizon for oligonucleotide therapeutics and at the same time added new levels of complexity. The field is poised to now exploit many of these mechanisms and to sort through the levels of complexity.

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