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Session 9 Track 2: Extra/Non-Hepatic Delivery
Session Chair(s)
Jeffrey Foy, PhD
Senior VP, Toxicology
PepGen Inc., United States
Sree Rayavarapu, DVM, PhD
Toxicologist
FDA, United States
This session will describe strategies for targeting extra-hepatic organs and will feature data from sponsor programs. Delivery technologies, nonclinical study design considerations, and pharmacology, ADME, and toxicology data will be discussed.
Learning Objective : At the conclusion of this session, participants should be able to:- Recognize current efforts to deliver oligonucleotide-based products to cells or organs outside of the liver
- Have a greater understanding of the challenges associated with delivering oligos to muscle, lung, and brain
Speaker(s)
Development of a Novel Muscle-targeted Antibody Oligonucleotide Conjugate for the Treatment of Myotonic Dystrophy Type 1: Toxicology and Regulatory Approaches and Considerations
Eileen Blasi, MS, MSc
Avidity Biosciences, United States
Senior Director of Toxicology
Nonclinical Development of Inhaled Oligonucleotides: Nothing Good Comes Easily
Jessica Grieves, DVM, PhD
Ionis Pharmaceuticals, United States
Director, Pathology and Nonclinical Development
Speaker
Vignesh Narayan Hariharan, PhD
University of Massachutes Medical School, United States
Instructor