Panel Discussion: Beyond Transferrin Receptors: Evaluating the Next Generation of CNS Oligonucleotide Delivery Strategies
8:30 am - Thursday 20th August 2026
- Reassessing the limitations of TfR1-mediated delivery, including broad peripheral expression, systemic sink effects, tolerability concerns, and limited cell-type specificity and discussing whether alternative receptor targets such as CD98, IGF1R and peptide ligands can deliver improved CNS selectivity
- Comparing emerging delivery modalities including antibody shuttles, peptide-mediated carriers, lipid nanoparticles (LNPs), viral vectors and other non-viral platforms, evaluating their ability to improve biodistribution, intracellular uptake, manufacturability and clinical translation
- Exploring both physical and non-physical approaches to overcoming the blood-brain barrier, including focused ultrasound, microbubble-assisted BBB opening and antibody-mediated modulation, while balancing enhanced CNS penetration with safety, reversibility and translational feasibility
- Debating which delivery strategies are most likely to enable systemic, repeatable and disease-specific oligonucleotide delivery for the next generation of CNS therapeutics
