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Session 2B: Assessing the Implications of Oligonucleotide Uptake into Cells
Session Chair(s)
Xuan Chi, 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)
Jeffery Engelhardt, DVM, PHD
Vice President, Pathology and Nonclinical Drug Safety, Ionis Pharmaceuticals, Inc., United States
Co-Chair
Kendall Frazier, DVM, PHD
, Retired, United States
Renal and Macrophage Uptake of Systemically Administered Oligonucleotides
Jeffery Engelhardt, DVM, PHD
Vice President, Pathology and Nonclinical Drug Safety, Ionis Pharmaceuticals, Inc., United States
Neuronal Uptake of Intrathecally Administered Oligonucleotides
James M. Willard
Pharmacologist, DCaRP, CDER, FDA, United States
A Report from the ESTP Expert Panel on Adversity of Lysosomal Accumulation
Panel Discussion
, All Session Speakers, United States
Vidhya Gopalakrishnan, PHD
Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc, United States
Panelist
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