Gene Expression | Modulating with Oligonucleotide-Based Therapies - 2021 World Medical Innovation Forum
May 2–4, 2022

Gene Expression | Modulating with Oligonucleotide-Based Therapies

Gene Expression | Modulating with Oligonucleotide-Based Therapies

Thursday, May 20, 2021

4:20 PM – 4:45 PM
Hot Topics

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?
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
Bob Brown, PhD
  • EVP R&D and CSO, Dicerna Pharmaceuticals
Brett Monia, PhD
  • CEO, Ionis
Alfred Sandrock, MD, PhD
  • EVP, R&D and CMO, Biogen
  • Q&A

    04:50 PM – 05:05 PM

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