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Related Concept Videos

In vitro Mutagenesis01:16

In vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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Toxicity tests in animals are grounded on two main assumptions: first, the effects observed in laboratory animals can be extrapolated to humans, especially when adjusted for body surface area; second, high-dose exposure in animals is essential to identify potential human hazards from lower doses. This is based on the quantal dose-response concept, which faces the challenge of extrapolating results from relatively few test animals to much larger human populations. For example, a 0.01% incidence...
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Mutagenicity and carcinogenicity refer to the ability of drugs to cause genetic defects and induce cancer, respectively. The International Agency for Research on Cancer (IARC) classifies agents into four groups based on their carcinogenic potential. Group 1 agents are known human carcinogens; group 2A agents are probably carcinogenic to humans; group 3 agents lack data to support their role in carcinogenesis; and group 4 includes agents for which data support that they are not likely to be...

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Developmental and Reproductive Toxicity Testing Strategies for Oligonucleotides: A Workshop Proceedings.

Bethany R Hannas1, Sara M Bender2, Eileen Blasi3

  • 1Eli Lilly & Company, Indianapolis, Indiana, USA.

Nucleic Acid Therapeutics
|May 30, 2025
PubMed
Summary

This workshop focused on improving developmental and reproductive toxicity (DART) assessments for oligonucleotide (ONT) therapeutics. Key strategies discussed aim to harmonize global guidance for safer drug development.

Keywords:
developmental and reproductive toxicologyoligonucleotidesplatformregulatory guidancesurrogate

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Area of Science:

  • Pharmacology and Toxicology
  • Drug Development
  • Regulatory Science

Background:

  • Oligonucleotide (ONT) therapeutics represent a rapidly advancing class of drugs.
  • Robust safety assessment, particularly developmental and reproductive toxicity (DART), is crucial for ONT approval.
  • Existing DART assessment strategies may require adaptation for unique ONT properties.

Purpose of the Study:

  • To convene stakeholders to discuss and identify strategies for enhancing DART assessments of ONTs.
  • To foster consensus on best practices and address knowledge gaps in ONT DART evaluation.
  • To inform the development of harmonized international guidance for ONT safety assessment.

Main Methods:

  • A workshop format brought together regulators, industry experts, consultants, and contract research organizations.
  • Discussions included case studies, consensus-building on approaches, and identification of areas for further research.
  • Review of strategies for dose selection, dosing frequency, species selection, and alternative models.

Main Results:

  • Exploration of surrogate ONT use and assessment of exposure in placental and embryo/fetal compartments.
  • Discussion of weight-of-evidence approaches for comprehensive DART evaluation.
  • Identification of specific methods and case studies to optimize DART strategies for ONTs.

Conclusions:

  • The workshop provided valuable insights into optimizing DART strategies for oligonucleotide therapeutics.
  • The proceedings aim to guide the development of harmonized international regulatory expectations for ONT safety.
  • Collaborative efforts are essential for establishing robust and efficient DART assessments in this field.