Whole-Brain Targeting: Overcoming Biological and Technological Barriers to Optimize CNS Nucleic Acid Delivery
03 Sept 2026
Drug Delivery
- Do you view whole brain distribution, cellular uptake or endosomal escape as the dominant bottleneck to achieving maximal efficacy for CNS-targeted nucleic acid therapies?
- Getting ASOs to deeper brain regions by CSF delivery has been challenging; what technologies, delivery platforms (i.e. antibodies, peptides, lipids, synthetic moieties) or medical devices do you see as potentially unlocking penetration to the deeper brain regions?
- How might brain anatomy and the location of different perivascular cells influence delivery to neurons?
- What are the pros and cons of IV delivery vs intra-CSF delivery of ASOs and how can we best address the major challenges with each?
- What new technology in the field, including AI/ML, are you most excited about, or do you think has the most potential to drive impact in this field?
