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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
Representative Invited
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.
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)
Representative Invited
Senior Director, Oligo ADME Strategy, GlaxoSmithKline, United Kingdom
Translational PK/PD Modeling Considerations
Representative Invited
SVP of Translational DMPK and Clinical Pharmacology, Stoke Therapeutics, United States
Role of Tissue Half Life in Designing Clinical Studies
Tod Harper, PhD
Scientific Director, Amgen, United States
Nonclinical Dose Selection, Safety Margins, and Translational Strategies for Oligonucleotide-based Therapeutics: Results from an IQ DruSafe ONT Working Group Cross-Industry Survey