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Session 10 Track 2: Extra-Hepatic Targeting and Delivery
Session Chair(s)
Tae-Won Kim
Executive Director, Toxicology, Ionis Pharmaceuticals, Inc., United States
James Wild, PHD, MS
Pharmacologist, CDER, FDA, United States
Targeted delivery and activity of oligonucleotides in extra-hepatic tissues can enable localized exposures and reduce dose levels and limit systemic exposures and associated toxicities including off-target effects. This session will highlight diverse oligonucleotide programs and delivery strategies targeting multiple indications across tissues such as cardiac, adipose, ocular, and joint tissues. Key topics will include delivery technologies, nonclinical study design considerations and pharmacology, ADME, and toxicology data supporting extra-hepatic applications.
Learning Objective : At the conclusion of this session, participants should be able to:- Identify emerging approaches for targeting specific RNA messages or message cascades in tissues or organs outside of the liver
- Understand the key challenges and advantages of targeted oligonucleotide delivery and activity in diverse extra-hepatic tissues.
- Apply key nonclinical considerations (e.g., pharmacology, ADME, toxicology) to support extra-hepatic development
Speaker(s)
Ditte Jaehger, PHD
CEO & Co-Founder, Hazel Therapeutics, Denmark
Localized Delivery of Antisense Oligonucleotides for Joint Diseases
Pooja Dua, PHD
Senior Director, Alliance Management, OliX Pharmaceuticals, United States
Cell-penetrating Asymmetric siRNA Targeting MyD88 Suppresses Ocular Degeneration and Neovascularization
Sandor Batkai, DrMed
Medical Director, Clinical Development, Obesity & Cardiovascular, RNA-Based Ther, Resalis Therapeutics, Germany
Challenges of Anti-miR Development, with Particular Focus on Extra Hepatic Delivery to Adipose Tissues and Heart as Target Organs for Chronic Diseases