Consortium on Advanced Stem Cell-Based Models in Drug Discovery and Development
A global organization of industry and academic scientists working with regulatory experts to establish standards, develop tools, and build the collaborative infrastructure needed to accelerate responsible integration of stem cell-derived models into preclinical stages of drug development. This effort aligns with recent policy and regulatory shifts toward new approach methodologies (NAMs) and greater emphasis on human-based research.
Impact and Working Groups
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Drug Discovery and Efficacy
Harnessing human stem cell-derived models—including iPSC, ESC, and tissue stem cell-derived organoids, cancer organoids, 2D cultures, including their use in microphysiological systems — to advance preclinical drug discovery and efficacy assessment. By leveraging the physiological relevance and patient-specific nature of stem cell-based models, the conrotium can improve translational accuracy, reduce late-stage drug attrition, and accelerate the development of safe and effective therapeutics.
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Translational Safety and Pharmacokinetic Modeling
Advancing the development, validation, and application of human stem cell-derived in vitro models to assess drug safety assessment. The consortium aims to integrate cutting-edge stem cell-based technologies with mechanistic toxicology and metabolic profiling to better model human biology in safety assessments.
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Precision Medicine
Advancing the development and application of patient-specific stem cell-based models, including induced pluripotent stem cells (iPSCs) and organoids as transformational tools for personalized healthcare.
The consoritum will also identify technical, biological, ethical, and translational challenges and propose collaborative strategies to integrate stem cell-based technologies into precision and personalized medicine pipelines across discovery, development, and preclinical implementation.
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Model Validation: Benchmarking, Reproducibility, and Standards
Dedicated to establishing robust, transparent, and reproducible validation frameworks for stem cell–based models used in biomedical research, drug development, and regulatory science. As these models become increasingly sophisticated and widely adopted, there is a critical need for independent evaluation criteria that ensure their biological relevance, reliability, reproducibility, and suitability for overall and tissue-specific model validation guidelines.
