Optimizing Both Conjugate & Oligonucleotide Design for Greater Target Cell Affinity & Blood-Brain Barrier Permeability of siRNA Payload
Time: 11:00 am
day: Day 1
Details:
- Focus on ligand optimization over high throughput screenings to improve the balance of affinity for target cell with permeability across the blood-brain barrier
- Modify PK/PD profiles to reduce peripheral tissue distribution by improving receptor specificity to the blood-brain barrier
- Explore alternate regions beyond the FAB region for Transferrin Receptor mediated transcytosis across the blood-brain barrier
- Discuss the impact of the blood-brain barrier on distribution to different brain regions as compared to intrathecal injection as illustrated through ISH imaging analysis
