Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges
Summary
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 ind
Content
# Human organoids as 3D in vitro platforms for drug discovery: opportunities and challenges
*Published: 2026 Mar*
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.
DOI: 10.1038/s41573-025-01317-y