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Human organoids: fit for drug discovery?

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Item Type:Review
Title:Human organoids: fit for drug discovery?
Creators: Wittich, Annika, Krieg, Kim, Gribbon, Philip, Metzger, Jakob J. ORCID logoORCID: https://orcid.org/0000-0001-6813-4425, Prigione, Alessandro and Pless, Ole
Abstract:Organoids are self-organizing three-dimensional (3D) in vitro tissues derived from pluripotent stem cells (PSCs) that recapitulate key structural and functional features of human organs. Their multicellular architecture and physiological relevance make them promising new approach methodologies (NAMs) for disease modeling, drug discovery, and toxicity testing. However, their reliability and scalability for compound screening remain under evaluation. This review summarizes current human PSC-derived organoid screening strategies, highlighting available readouts, related machine learning methods, and their potential advantages over traditional screening models. We also discuss major challenges, including assay robustness, throughput limitations, and the need for standardized protocols. Advancing validated and scalable approaches will be essential for integrating organoids into pharmaceutical development and improving the translational success of drug candidates.
Keywords:Drug Discovery, High-Throughput Screening Assays, Organoids, Pluripotent Stem Cells, Preclinical Drug Evaluation
Source:Stem Cell Reports
ISSN:2213-6711
Publisher:Elsevier / Cell Press
Volume:21
Number:6
Page Range:102898
Date:9 June 2026
Official Publication:https://doi.org/10.1016/j.stemcr.2026.102898
PubMed:View item in PubMed

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