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Bethesda North Marriott Hotel and Conference Center

25 oct 2017 7:00 a.m. - 27 oct 2017 3:00 p.m.

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.

Session 9BC: Expanding Therapeutic Utility via Targeted Delivery

Session Chair(s)

Patrik  Andersson, PHD

Patrik Andersson, PHD

Principal Scientist, Discovery Safety Specialist, AstraZeneca R&D, Sweden

This joint preclinical/clinical session will present novel translational insights of GalNAc-conjugated ASOs as well as novel targeting strategies for increasing productive uptake into cell types that normally are challenging to reach with oligonucleotide therapeutics. These include muscle and immune cells as well as pancreatic islets. The presentations will be followed by a 30 minute panel discussion. Discussion topics include:

  • Main utilities and obstacles of targeted delivery of oligos
  • Specific regulatory considerations to targeting approaches

  • Learning Objective : Recognize the opportunities and challenges of developing novel targeting approaches

    Speaker(s)

    Sree  Rayavarapu, DVM, PHD

    Session Co-Chair

    Toxicologist, FDA, United States

    Wouter  Driessen, PHD, MS

    Translational Development of a GalNAC-Conjugated LNA-Based Single Stranded Oligonucleotide

    DMPK Project Leader , F. Hoffmann-La Roche AG, Switzerland

    Arthur A. Levin, PHD

    Oligonucleotide Therapeutics Now on Target

    Distinguished Scientist, Avidity Biosciences, United States

    Patrik  Andersson, PHD

    Targeting Antisense Oligonucleotides to Pancreatic Islets

    Principal Scientist, Discovery Safety Specialist, AstraZeneca R&D, Sweden

      Panel Discussion

    Panel Discussion

    , All Session Speakers, United States

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