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Bethesda North Marriott Hotel and Conference Center

Oct 28, 2019 7:00 AM - Oct 30, 2019 12:30 PM

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

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 2 Track B: Targeted Delivery

Session Chair(s)

Patrik  Andersson, PhD

Patrik Andersson, PhD

Senior Director, RNA Therapeutics Safety

AstraZeneca R&D, Sweden

Donald N. Jensen, DVM, MS

Donald N. Jensen, DVM, MS


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

Avidity Biosciences, United States

Distinguished Scientist

Ben-Fillippo  Krippendorff, PhD

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

Ben-Fillippo Krippendorff, PhD

Roche Pharma Research and Early Development, Roche Innovation Center, Switzerland

DMPK/PD Project Leader, Pharmaceutical Sciences

Charlotte  Bon, DVM, PhD

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

Charlotte Bon, DVM, PhD

F. Hoffman-La Roche AG, Switzerland

Translational Modeling and Simulation Scientist

Jing-Tao  Wu, PhD

ADME Properties of GalNAc siRNAs

Jing-Tao Wu, PhD

Alnylam Pharmaceuticals, United States

Vice President, Early Development

Rusty  Montgomery, PhD

Targeted Delivery (Lung) of MicroRNA-Modulating Drugs

Rusty Montgomery, PhD

miRagen Therapeutics, Inc., United States

Director, Research

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