1:00 pm Workshop C: Harnessing ASO & siRNA Innovation for Cell Specific Delivery & Distribution
Time: 1:00 pm
day: Pre-Conference Workshop Day - Track A P2
Details:
Oligonucleotide therapeutics offer a powerful approach to modulating gene expression in the CNS, but precise cell-type targeting and broad distribution, remain key challenges. This workshop will explore how different oligonucleotide modalities, siRNA, ASOs, and aptamers, can be optimized for CNS penetration, cell-specific uptake, and long-lasting efficacy.
Join this session to:
- Understand how oligonucleotide stability and degradation impact long-term CNS exposure
- Investigate how ligand and conjugate modifications influence selective uptake in neurons, astrocytes, and microglia
- Assess how IT injection strategies can be refined to ensure broader and deeper brain distribution
- Evaluate current limitations in predicting oligonucleotide biodistribution and improving in vivo screening strategies
