Human pluripotent stem cell-derived organoids as translational platforms for drug discovery and regenerative medicine

Authors

DOI:

https://doi.org/10.66505/cbtt.v1i2.37

Keywords:

human pluripotent stem cells, stem cells, organoids, disease modeling, drug development, regenerative medicine, 3D culture, organ-on-chip, stem cell engineering

Abstract

Organoid technologies have quickly become vital experimental systems for modeling human tissues in vitro. These three-dimensional structures can reproduce aspects of tissue architecture, cellular diversity, and functional organization that are difficult to capture in traditional two-dimensional cultures. Advances in lineage-specification protocols and synthetic matrix engineering have substantially improved the reproducibility and maturity of hPSC-derived organoids. As a result, hPSC-derived organoids are being used more widely across biomedical research, including developmental studies, disease modeling, drug testing, and regenerative medicine. In this review, we analyze major hPSC-derived organoid systems across various organ types and discuss their growing importance in translational research. We focus on three emerging areas. First, organoids are increasingly used as human-relevant platforms for drug discovery, enabling assessment of therapeutic efficacy, mechanisms of action, and potential toxicity in physiologically relevant settings. Second, these systems are being studied as experimental foundations for regenerative medicine, supporting research on tissue repair, gene correction, and cell-based therapies. Third, advances in engineering, such as organoid-on-chip technologies, vascularization strategies, and multi-organoid culture systems, are enhancing the reproducibility, scalability, and functional maturity of organoid models. Despite rapid progress, several challenges remain. Organoid cultures can display variability, incomplete maturation, and limited representation of the full in vivo microenvironment. Continued integration of stem cell biology, biomaterials engineering, and computational analysis will be important for overcoming these limitations. With these advancements, hPSC-derived organoids are poised to become increasingly valuable platforms linking basic developmental biology with predictive pharmacology and regenerative medicine.

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Schematic illustrating the generation of human pluripotent stem cell (hPSC)-derived organoids from embryonic stem cells and induced pluripotent stem cells through somatic cell reprogramming. The figure highlights differentiation into organ-specific organoids representing ectoderm, mesoderm, and endoderm lineages and their applications in drug development and regenerative medicine.

Published

2026-05-12

How to Cite

1.
Wang Y, Nozhat Z, Duan T, Chen S, Ni Y, Wang Y, et al. Human pluripotent stem cell-derived organoids as translational platforms for drug discovery and regenerative medicine. Cancer Biome Target Ther. [Internet]. 2026 May 12 [cited 2026 Oct. 3];1(2):289-31. Available from: https://cancerbiometherapy.com/index.php/cbtt/article/view/37

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Review