Organoids in Pharmacology: From Disease Modeling to Predictive Drug Discovery

Organoid technologies have emerged as transformative tools in biomedical research, enabling the generation of three-dimensional, multicellular systems that recapitulate key structural and functional features of human tissues. Derived from primary cells or induced pluripotent stem cells, organoids provide physiologically relevant platforms for disease modeling and the study of disease progression, as well as for investigating therapeutic response. In the context of drug discovery and screening, organoids are increasingly recognized as powerful drug discovery tools, offering clear advantages over traditional two-dimensional cultures and animal models by preserving tissue-specific architecture, cellular heterogeneity, and human-specific biology. These attributes position organoids as highly valuable translational platforms, bridging the gap between preclinical studies and clinical outcomes. Organoid-based platforms are becoming central components of next-generation human-relevant experimental systems and New Approach Methodologies (NAMs), with growing adoption across academia and industry for evaluating drug efficacy, toxicity, and mechanisms of action.Despite significant advances, key challenges remain in fully utilizing the potential of organoids for drug discovery and screening. These include variability in organoid generation and maturation, limited scalability and throughput, and the need for standardized benchmarking against clinical and in vivo data. Furthermore, integrating organoids with complementary technologies, such as microphysiological systems (MPS), high-content imaging, multi-omics profiling, and computational modeling, remains an active area of development.This Research Topic aims to bring together interdisciplinary efforts that advance organoid-based platforms toward greater predictive power, reproducibility, and translational relevance. We seek contributions that address both fundamental and applied aspects of organoid use in pharmacology, including innovations that improve model fidelity, enable higher-throughput screening, and enhance mechanistic insight into drug responses. By highlighting emerging technologies, analytical frameworks, and case studies, this collection will help define best practices and identify strategies to bridge the gap between in vitro findings and clinical outcomes.This Research Topic welcomes submissions that explore the use of organoids in drug discovery and screening across diverse biological systems and disease areas. Topics of interest include, but are not limited to: organoid model development and characterization; scalability and automation for screening applications; integration with microphysiological systems and other NAMs; high-content imaging and multi-omics analyses; computational modeling and data integration; disease modeling and target validation; precision medicine applications; drug efficacy and toxicity testing; and benchmarking against clinical or in vivo datasets.We encourage a range of manuscript types, including Original Research Articles, Reviews, Mini Reviews, Methods, and Perspective articles. Contributions that emphasize translational relevance, reproducibility, and cross-platform comparison are particularly encouraged. Interdisciplinary studies that combine experimental and computational approaches, or that demonstrate applications in precision medicine and patient stratification, are especially welcome. Topic editors: Leonardo D'Aiuto, Amelia Versace, Mark T Miedel Source: https://www.frontiersin.org/research-topics/81229/organoids-in-pharmacology-from-disease-modeling-to-predictive-drug-discovery