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Bethesda North Marriott Hotel and Conference Center

2017 年 10 月 25 日 7:00 上午 - 2017 年 10 月 27 日 3:00 下午

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

Senior Director, RNA Therapeutics Safety

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

    Sree Rayavarapu, DVM, PhD

    FDA, United States

    Toxicologist

    Wouter  Driessen, PhD, MS

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

    Wouter Driessen, PhD, MS

    F. Hoffmann-La Roche AG, Switzerland

    DMPK Project Leader

    Arthur A. Levin, PhD

    Oligonucleotide Therapeutics Now on Target

    Arthur A. Levin, PhD

    Avidity Biosciences, United States

    Distinguished Scientist

    Patrik  Andersson, PhD

    Targeting Antisense Oligonucleotides to Pancreatic Islets

    Patrik Andersson, PhD

    AstraZeneca R&D, Sweden

    Senior Director, RNA Therapeutics Safety

      Panel Discussion

    Panel Discussion

    All Session Speakers, United States

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