Muthiah Manoharan
Seminars
Wednesday 2nd December 2026
Translating Lessons from GalNAc to the Brain Exploring How Oligonucleotide Chemistry Shapes CNS Delivery and Distribution
1:30 pm
- Applying key learnings from the GalNAc platform to CNS-targeted delivery systems to improve brain exposure and therapeutic performance
- Understanding how oligonucleotide backbone chemistry, from negatively charged siRNAs to neutral PMOs, influences conjugate design and biodistribution
- Evaluating the impact of drug-to-antibody ratio and conjugation architecture, including 1:1, 4:1 and 8:1 formats, on delivery efficiency and CNS distribution profiles
- Leveraging chemical modifications to optimize shuttle performance, enhance target engagement and overcome barriers to effective brain delivery
