Ekta KADAKIA
Director, DMPK Biogen
Seminars
Friday 21st August 2026
Applications of Pharmacokinetics and Pharmacodynamics Principles for Optimizing the Design of Brain-Targeting Oligonucleotides
10:30 am
- Design of Brain-Targeting Oligonucleotides
- Intrathecal dosing enables effective CNS delivery of oligonucleotides, but limited brain biodistribution remains a key challenge, restricting their therapeutic potential
- PK/PD modeling and simulation provide a quantitative framework to integrate multiple oligonucleotide-related variables and predict target knockdown across the brain
- Model-guided optimization of oligonucleotide parameters such as the potency and tissue half-life provides an objective approach to lead optimization, to enhance their therapeutic application
