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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
Principal Consultant, Aclairo Pharmaceutical Development Group, Inc., United States
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.
Speaker(s)
Kendall Frazier, DVM, PhD
, Retired, United States
Examination of Glomerular Toxicity Risk Across ASO Platforms and Trials
Padma Kumar Narayanan, DVM, PhD, MS
Executive Director, Ionis Pharmaceuticals, Inc., United States
Understanding the Mechanism for Dose-Dependent Thrombocytopenia Associated with 2' MOE ASOs
Ivan Zlatev, PhD
Principal Scientist, Research, Alnylam Pharmaceuticals, Inc., United States
Safety Evaluation of 2'-Deoxy-2'-Fluoro Nucleotides in GalNAc-siRNA Conjugates
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