Roundtable Discussion: Advancing Dose Selection & Translational PK/PD Modeling for Oligonucleotide Therapeutics & Brain Shuttles in the CNS

Time: 1:30 pm
day: Day 2

Details:

• Leverage translational PK/PD modeling to inform dose selection and understand exposure-response relationships in the CNS for oligonucleotide-based therapies
• Explore brain shuttle and Transferrin Receptor mediated uptake and modeling strategies to assess CNS exposure and PK ‘hit’
• Integrate intrathecal distribution data into predictive frameworks to optimize therapeutic delivery and biodistribution
• Case examples and lessons learned from neuroscience and oligonucleotide programs to support clinical translation and decision making