Organoid

Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges

Authors: Daisong Wang, Remi Villenave, Nadine Stokar-Regenscheit & Hans Clevers in Nature Reviews Drug Discovery.

Nov 14, 2025

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Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges

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Abstract

Organoids are 3D structures derived from stem cells that recapitulate key architectural and functional aspects of the corresponding tissue. Compared with conventional 2D cell lines, human organoids provide experimental models that more closely reflect human physiology. Their ability to capture the complexity and heterogeneity of human tissues enables the study of disease mechanisms, drug efficacy and toxicity. When generated from patient material, organoids also allow the assessment of individual drug responses. In this Review, we explore the utility of organoids in drug discovery. We outline current methodologies for generating and maintaining organoids, examine their applications in disease modelling, drug screening and safety evaluation, and consider regulatory aspects and the challenges for their broader adoption in drug discovery.

Wang, D., Villenave, R., Stokar-Regenscheit, N. et al. Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges. Nat Rev Drug Discovery (2025).

https://doi.org/10.1038/s41573-025-01317-y

WORC Update (He/Him)

Head of community, WORC.Community (A Caterpillar Hill Limited venture).

Thank you for joining us here at WORC! This platform has the sole purpose of bringing Organoid and Organ-on-a-chip researchers together! Easily connect, share content, ideas and experiences in this transformative area of science. The idea for this community came after speaking with many of you expressing the need for joined up thinking and making long lasting connections.