Workshop D: Navigating Complexity of Conjugated Oligonucleotide Formulation to Ensure Efficient Brain Delivery

Time: 3:00 pm
day: Pre-Conference Workshop Day Track D

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

Speakers: