Workshop A: Navigating Complexity of Conjugated Oligonucleotide Formulation to Ensure Efficient Brain Delivery
Time: 7:30 am
day: Pre-Conference Workshop Day Track A
Details:
Synthesis and formulation of conjugated oligonucleotides can be very different to naked oligonucleotides and require a proper control strategy to be set in place prior. How can we leverage experience from protein and biologic formulation development to better navigate conjugate formulation for siRNAs, ASOs & RNA aptamers in the CNS
- Sharing case studies in formulating more complicated modifications to the backbone using new starting material to
- gain insights into customized synthesis
- Understanding the changes in biophysical properties of oligonucleotides with conjugation
- Exploring use cases of more challenging modifications and conjugations e.g. formulating oligonucleotides with 2’ sugar positioning
- Developing and validating analytical methods capable of determining impurities in complex modified and conjugated oligonucleotides
- Optimizing formulation to avoid aggregation of oligonucleotides and protecting safety
