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Session 4 Track 2: Optimizing Oligonucleotide Therapeutics: Translating PK/PD and Tissue Half-Life into Smarter Dose Selection and Risk Assessment
Session Chair(s)
Tod Harper, PhD
Scientific Director, Amgen, United States
Sree Rayavarapu, DVM, PhD
Toxicologist, FDA, United States
Oligonucleotide-based therapeutics present distinct nonclinical development challenges compared to traditional small molecules and biologics, including prolonged tissue persistence, complex pharmacokinetic/pharmacodynamic (PK/PD) relationships, and sequence-dependent off-target effects. All of these features can complicate nonclinical-to-clinical translation. This session will examine practical strategies for integrating translational PK/PD and nonclinical safety data to support more informed dose selection and rigorous risk assessment. Speakers will also address how current development approaches are being adapted in the absence of oligonucleotide--specific regulatory guidance.
Translational PK/PD Modeling Considerations for Oligonucleotide Therapeutics
Luc Rougee, Lilly and Company
Utilization of Oligonucleotides’ Half-Life to Optimize Dosing Regimen for Desirable Efficacy and Reduced CNS Toxicity
Meena, Stoke Therapeutics
Nonclinical Dose Selection, Safety Margins, and Translational Strategies for Oligonucleotide-Based Therapeutics
Tod Harper, Amgen Inc
Learning Objective : At the conclusion of this session, participants should be able to: - Compare key PK/PD and tissue half-life factors that drive dose selection for oligonucleotide therapeutics
- Apply nonclinical safety and translational PK/PD data to assess risk and justify starting-dose strategies
- Distinguish current challenges and practical approaches used when oligonucleotide -specific regulatory guidance is limited
Speaker(s)
Translational PK/PD Modeling Considerations for Oligonucleotide Therapeutics
Luc Rougee, MS
Director, Eli Lilly and Company, United States
Utilization of Oligonucleotides’ Half-Life to Optimize Dosing Regimen for Desirable Efficacy & Reduced CNS Toxicity
Meena Meena, PhD
SVP of Translational DMPK and Clinical Pharmacology, Stoke Therapeutics, United States
Nonclinical Dose Selection, Safety Margins, and Translational Strategies for Oligonucleotide-Based Therapeutics
Tod Harper, PhD
Scientific Director, Amgen, United States