Clinical Pharmacology – Biomolecular
2019 PharmSci 360
This presentation will review the basic pharmacokinetic properties of antisense oligonucleotides (ASOs) in humans and provide an overview of key clinical pharmacology studies and some findings as a platform. The session will also highlight some challenges and lessons learned in ASO drug development, as well as some recent breakthroughs in target tissue delivery which have transformed the platform. The value of PK/PD modeling and exposure/response analysis in ASO dose prediction and recommendation will also be discussed.