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Capital Hilton

Oct 28, 2024 8:30 AM - Oct 30, 2024 12:40 PM

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

Executive Director, Toxicology, Nimbus Therapeutics, 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

Eileen Blasi, MS, MSC

Senior Director of Toxicology, Avidity Biosciences, United States

Development of a Novel Muscle-targeted Antibody Oligonucleotide Conjugate for the Treatment of Myotonic Dystrophy Type 1: Toxicology and Regulatory Approaches and Considerations

Jessica  Grieves, DVM, PHD

Jessica Grieves, DVM, PHD

Director, Pathology and Nonclinical Development, Ionis Pharmaceuticals, United States

Nonclinical Development of Inhaled Oligonucleotides: Nothing Good Comes Easily

Vignesh Narayan  Hariharan, PHD

Vignesh Narayan Hariharan, PHD

Instructor, University of Massachutes Medical School, United States

Speaker

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