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Considerations for assessment of reproductive and developmental toxicity of oligonucleotide-based therapeutics

Joy Cavagnaro1, Cindy Berman, Doug Kornbrust

  • 11 Access BIO , Boyce, Virginia.

Nucleic Acid Therapeutics
|August 20, 2014
PubMed

Insights

This white paper outlines strategies for assessing reproductive toxicity of therapeutic oligonucleotides (ONs). It emphasizes product-specific considerations for designing safety evaluation programs due to limited class experience.

Area of Science:

  • Pharmacology
  • Toxicology
  • Drug Development

Background:

  • Therapeutic oligonucleotides (ONs) represent a novel class of drugs with unique chemical and biological properties.
  • Assessing the reproductive and developmental risks associated with ONs requires tailored toxicological study designs.

Purpose of the Study:

  • To summarize the consensus on strategies for evaluating potential reproductive and/or developmental toxicities of therapeutic oligonucleotides.
  • To provide guidance on designing science-based reproductive safety evaluation programs for ONs.

Main Methods:

  • The white paper consolidates the current consensus from the Reproductive Subcommittee of the Oligonucleotide Safety Working Group.
  • Key considerations for study design include chemical characteristics, mechanism of action, and optimal exposure assessment.

Main Results:

  • Specific best practices for all oligonucleotide classes cannot yet be defined due to limited experience.
  • The paper provides "points to consider" for designing reproductive safety evaluations based on product attributes.

Conclusions:

  • A product-specific, science-based approach is crucial for evaluating the reproductive safety of therapeutic oligonucleotides.
  • Careful consideration of ON chemical characteristics, mechanism of action, and exposure is essential for study design.

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