SC2: Examining the Safety and Toxicity of Nucleic Acid Therapeutics

MONDAY, MARCH 16 | 6:30 – 9:30 PM

Nucleic acid drugs continue to deliver on their promise to become a third therapeutic modality, in addition to small molecules and biologics. Several antisense oligonucleotide drugs have been on the market for some time, while the first RNAi approval was granted in 2018. In addition, numerous mRNA and CRISPR therapeutic programs have entered clinical stages. Despite the common “nucleic acid” component, the mechanisms of action and of non-specific effects differ for each of these drug types.


  • Different types of nucleic acid-based drugs
  • Mechanisms of actions and non-specific effects
  • Current approaches to address non-specific and potentially toxic effects

Aimed at both novice and advanced nucleic drug developers, the course will:

  • Introduce and explain the differences between various types of nucleic acid drugs
  • Summarize our current understanding of the origins of non-specific and potentially toxic effects
  • Provide direction on how to minimize the potential toxic effects of nucleic acids drugs

Jeffrey Foy, PhD, Senior Director, Toxicology, Dicerna Pharmaceuticals

Shawna Weis, PhD, Senior Director, Regulatory Toxicology, Wave Life Sciences

Xiao Shelley Hu, Senior Director, Head of DMPK and Clinical Pharmacology, Wave Life Sciences

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