Exploring Opportunities for Systemic Antisense Delivery with Receptor-Mediated BBB Shuttles
Time: 8:00 am
day: Pre-Conference Day
Details:
Due to their large size and virus-like nature eliciting immune defences, oligonucleotide therapeutics have been restricted in their ability to be delivered systemically and cross the blood-brain barrier, but what if they could be conjugated to brain-penetrating proteins and shuttled across as cargo?
Push the boundaries of ASO delivery beyond intrathecal injection and uncover new opportunities for the application of shuttle-mediated systemic delivery in this forward-thinking workshop covering:
- Emerging examples of existing shuttle platforms being applied to oligonucleotides to achieve efficacious delivery across the blood-brain barrier
- Considerations for the potential of peptide-based oligonucleotide conjugates leveraging blood-brain barrier receptors to internalize the oligonucleotides
- Next steps for successful translation of these animal and NHP studies
