Roundtable Discussion: Into the Thick of It: Oligonucleotides into the Brain with Depth, Precision & Safety
- Achieving deep brain oligonucleotide exposure while managing acute and chronic neurotoxicity
- The role of imaging and PK/PD modelling in optimising CNS dosing strategies
- Limitations of transferrin receptor-mediated delivery and emerging alternative shuttle approaches
- Balancing regional coverage, cell-type specificity, and peripheral exposure risks
- Translational and safety considerations impacting clinical development and regulatory confidence