Gene Expression | Modulating with Oligonucleotide-Based Therapies
Thursday, May 20, 2021
Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:
How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?
- Will oligonucleotides improve as a class that will make them even more effective? Are further advancements in backbone chemistry anticipated, for example.
- Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
- Are small molecules a threat to oligonucleotide-based therapies?
- Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides? Is there a place for multiple mechanism oligonucleotide medicines?
- Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?
Q&A04:50 PM – 05:05 PM