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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 3 Track B: Toxicology Updates on Renal and Platelet Effects of ASOs and 2’-Fluoro Nucleotide Effects of siRNAs

Session Chair(s)

Ronald  Wange, PhD

Ronald Wange, PhD

Associate Director for Pharm/Tox, OND, CDER

FDA, United States

Jeffery  Engelhardt, DVM, PhD

Jeffery Engelhardt, DVM, PhD

Vice President, Pathology and Nonclinical Drug Safety

Ionis Pharmaceuticals, Inc., United States

This session will examine the renal accumulation of ASOs in animals, mechanisms of ASO-related glomerular injury, and the broader experience of clinical renal toxicity with the class. The current hypothesis on the pathogenesis of ASO-related thrombocytopenia in nonhuman primates and potential strain dependence will be discussed along with potential clinical ramifications. The safety studies of 2’-deoxy-2’-fluoro nucleotides in GalNAc-siRNA conjugates will also be discussed in view of toxicity concerns from previous drugs containing 2’-F-modified nucleobases, such as fialuridine.


Kendall  Frazier, DVM, PhD

Examination of Glomerular Toxicity Risk Across ASO Platforms and Trials

Kendall Frazier, DVM, PhD

Retired, United States

Padma Kumar  Narayanan, DVM, PhD, MS

Understanding the Mechanism for Dose-Dependent Thrombocytopenia Associated with 2' MOE ASOs

Padma Kumar Narayanan, DVM, PhD, MS

Ionis Pharmaceuticals, Inc., United States

Executive Director

Ivan  Zlatev, PhD

Safety Evaluation of 2'-Deoxy-2'-Fluoro Nucleotides in GalNAc-siRNA Conjugates

Ivan Zlatev, PhD

Alnylam Pharmaceuticals, Inc., United States

Principal Scientist, Research

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