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DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 2 Track B: Targeted Delivery

Session Chair(s)

Patrik  Andersson, PhD

Patrik Andersson, PhD

  • Principal Scientist, Discovery Safety Specialist
  • AstraZeneca R&D, Sweden
Donald N. Jensen, DVM, MS

Donald N. Jensen, DVM, MS

  • Pharmacologist
  • FDA, United States
Single and double stranded oligonucleotide therapeutics share the challenge of limited uptake into many cell types of therapeutic interest. This restricted cellular uptake is one of the major hurdles to solve before oligonucleotides can be utilized to their full potential. One way to increase productive uptake is to hitchhike on receptors that internalize ligands, best exemplified by GalNAc-conjugated oligonucleotides to increase uptake into hepatocytes via the asioalyglycoprotein receptor. This session will cover new DMPK understanding of GalNAc conjugated siRNA and antisense oligonucleotides like parameters driving potency, bioavailability, and intracellular trafficking as well as aspects of receptor saturation studied in preclinical models and the clinic. The session will also cover novel strategies of targeted delivery of miR-29 mimics to the lung as well as delivery to muscle using antibody-conjugates targeting the transferrin receptor.


Arthur A. Levin, PhD

Oligonucleotide Therapeutics: Now on Target

Arthur A. Levin, PhD

  • Executive Vice President, Research and Development
  • Avidity Biosciences , United States
Ben-Fillippo  Krippendorff, PhD

Preclinical and Clinical PK of GalNAc SSOs at Doses Below and Above ASGPR Saturation

Ben-Fillippo Krippendorff, PhD

  • DMPK/PD Project Leader, Pharmaceutical Sciences
  • Roche Pharma Research and Early Development, Roche Innovation Center, Switzerland
Charlotte  Bon, DVM, PhD

Preclinical and Clinical PK of GalNAc SSOs at Doses Below and Above ASGPR Saturation

Charlotte Bon, DVM, PhD

  • Translational Modeling and Simulation Scientist
  • F. Hoffman-La Roche AG, Switzerland
Jing-Tao  Wu, PhD

ADME Properties of GalNAc siRNAs

Jing-Tao Wu, PhD

  • Vice President, Early Development
  • Alnylam Pharmaceuticals, United States
Rusty  Montgomery, PhD

Targeted Delivery (Lung) of MicroRNA-Modulating Drugs

Rusty Montgomery, PhD

  • Director, Research
  • miRagen Therapeutics, Inc., United States