Advances and Challenges in Oligonucleotide and mRNA Therapeutics: New Strategies and Regulatory Perspectives (S12)

Symposium Chair(s)

• Xihui Xu, PharmD, PhD

  Director
  Toxicology
  QurAlis Corporation
  Cambridge, Massachusetts, United States

• Jennifer Sisler, PhD

  Senior Advisor
  Toxicology
  Eli Lilly and Company, Inc.
  Indianapolis, Indiana, United States

Symposium Speaker(s)

• Belete Teferedegne, DVM, PhD, DABT

  Director
  Clinical Pharmacology and Safety Sciences
  AstraZeneca
  Gaithersburg, Maryland, United States

• Tom Zanardi, PhD, DABT

  Executive Director
  Toxicology
  Ionis Pharmaceuticals
  Carlsbad, California, United States

• Chun-Ting (David) Lee, PhD, MSPH

  Toxicologist
  Division of Pharmacology and Toxicology, Neurology, Office of Neuroscience, CDER
  US FDA

Educational Support Provided by: QurAlis Corporation

Oligonucleotides and mRNA therapeutics have advanced over the past 20 years, as well as the number of drugs approved. With the advancement of this field, there has been an increase in novel targets and delivery mechanisms. While the field has advanced in targeted knockdown, the challenges have also increased such as local tolerability and off-target toxicity. This symposium will focus on these advancements and strategies for different modalities, including GalNAc siRNAs, ASOs, mRNA vaccines. Each talk will focus on the development strategy including type of studies performed, dose selection and the use of surrogate molecules if appropriate for an IND-enabling package and beyond.

Presentations:

• 2:00 PM - 2:05 PM CT

  Welcome
  

  Symposium Chair: Xihui Xu, PharmD, PhD – QurAlis Corporation
• 2:05 PM - 2:40 PM CT

  Strategies and Development of GalNAc siRNAs
  

  Symposium Speaker: Jennifer Sisler, PhD – Eli Lilly and Company, Inc.
• 2:40 PM - 3:15 PM CT

  Nonclinical Safety Assessments of the Next Generation of mRNA Vaccines for SARS-CoV-2 and Other Respiratory Viruses
  

  Symposium Speaker: Belete Teferedegne, DVM, PhD, DABT – AstraZeneca
• 3:15 PM - 3:45 PM CT

  Break
• 3:45 PM - 4:15 PM CT

  Expanding the Reach of Antisense Therapeutics: Facing Challenges Associated with Nonclinical Safety Assessment for Local IT Administration
  

  Symposium Speaker: Tom Zanardi, PhD, DABT – Ionis Pharmaceuticals
• 4:15 PM - 4:45 PM CT

  Safety Assessment of Oligonucleotides Administered via the Intrathecal Route
  

  Symposium Speaker: Chun-Ting (David) Lee, PhD, MSPH – US FDA
• 4:45 PM - 5:00 PM CT

  Panel Discussion