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Capital Hilton

28 oct 2024 8:30 a.m. - 30 oct 2024 12:40 p.m.

1001 16th Street NW, Washington, DC 20036-5794

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 9 Track 2: Extra/Non-Hepatic Delivery

Session Chair(s)

Jeffrey  Foy, PhD

Jeffrey Foy, PhD

Senior VP, Toxicology

PepGen Inc., United States

Sree  Rayavarapu, DVM, PhD

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)

Eileen  Blasi, MS, MSc

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

Jessica  Grieves, DVM, PhD

Nonclinical Development of Inhaled Oligonucleotides: Nothing Good Comes Easily

Jessica Grieves, DVM, PhD

Ionis Pharmaceuticals, United States

Director, Pathology and Nonclinical Development

Vignesh Narayan  Hariharan, PhD

Speaker

Vignesh Narayan Hariharan, PhD

University of Massachutes Medical School, United States

Instructor

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