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Session 2B: Assessing the Implications of Oligonucleotide Uptake into Cells
Session Chair(s)
Xuan Chi, MD, PhD
Supervisory Pharmacologist
CDER, FDA, United States
This session will examine the cellular uptake and lysosomal processing of single-stranded oligonucleotides by the most frequently affected tissues and cells following systemic administration (kidney and macrophages) and intrathecal administration (neurons). The histopathologic changes associated with uptake will be presented and discussed relative to assessing adversity of the findings. The conclusion of the European Society of Toxicologic Pathology (ESTP) expert panel on adversity of lysosomal accumulation relative to oligonucleotides will also be discussed.
Learning Objective :
Speaker(s)
Co-Chair
Jeffery Engelhardt, DVM, PhD
Ionis Pharmaceuticals, Inc., United States
Vice President, Pathology and Nonclinical Drug Safety
Renal and Macrophage Uptake of Systemically Administered Oligonucleotides
Kendall Frazier, DVM, PhD
GlaxoSmithKline, United States
Director, Cellular and Molecular Pathology
Neuronal Uptake of Intrathecally Administered Oligonucleotides
Jeffery Engelhardt, DVM, PhD
Ionis Pharmaceuticals, Inc., United States
Vice President, Pathology and Nonclinical Drug Safety
A Report from the ESTP Expert Panel on Adversity of Lysosomal Accumulation
James M. Willard
FDA, United States
Pharmacologist, DCaRP, CDER
Panel Discussion
All Session Speakers, United States
Panelist
Vidhya Gopalakrishnan, PhD
Quark Pharmaceuticals, Inc, United States
Senior Vice President, Pharmaceutical Development