Organoids and organs-on-chips: Recent advances, applications in drug development, and regulatory challenges

Liangbin Zhou¹, Jingjing Huang², Cun Li², Qi Gu³, Gang Li⁴, Zhong Alan Li⁵, Jiankun Xu⁶, Jie Zhou⁷, Rocky S Tuan⁸

Affiliations

¹ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Musculoskeletal Research Laboratory, Department of Orthopaedics and Traumatology, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; Innovative Orthopaedic Biomaterials and Drug Translational Research Laboratory, Li Ka Shing Institute of Health Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China.
² Department of Microbiology, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
³ Key Laboratory of Organ Regeneration and Reconstruction, Beijing Institute for Stem Cell and Regenerative Medicine, Institute of Zoology, Chinese Academy of Sciences, Beijing, China.
⁴ Department of Otolaryngology-Head and Neck Surgery, Nanfang Hospital of the Southern Medical University, Guangzhou, China.
⁵ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Institute for Tissue Engineering and Regenerative Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: alanli@cuhk.edu.hk.
⁶ Musculoskeletal Research Laboratory, Department of Orthopaedics and Traumatology, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; Innovative Orthopaedic Biomaterials and Drug Translational Research Laboratory, Li Ka Shing Institute of Health Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: jiankunxu@cuhk.edu.hk.
⁷ Department of Microbiology, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China. Electronic address: jiezhou@hku.hk.
⁸ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Institute for Tissue Engineering and Regenerative Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: tuanr@cuhk.edu.hk.

PMID: 40220744
DOI: 10.1016/j.medj.2025.100667

Review

Organoids and organs-on-chips: Recent advances, applications in drug development, and regulatory challenges

Liangbin Zhou et al. Med. 2025.

. 2025 Apr 11;6(4):100667.

doi: 10.1016/j.medj.2025.100667.

Authors

Liangbin Zhou¹, Jingjing Huang², Cun Li², Qi Gu³, Gang Li⁴, Zhong Alan Li⁵, Jiankun Xu⁶, Jie Zhou⁷, Rocky S Tuan⁸

Affiliations

¹ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Musculoskeletal Research Laboratory, Department of Orthopaedics and Traumatology, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; Innovative Orthopaedic Biomaterials and Drug Translational Research Laboratory, Li Ka Shing Institute of Health Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China.
² Department of Microbiology, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
³ Key Laboratory of Organ Regeneration and Reconstruction, Beijing Institute for Stem Cell and Regenerative Medicine, Institute of Zoology, Chinese Academy of Sciences, Beijing, China.
⁴ Department of Otolaryngology-Head and Neck Surgery, Nanfang Hospital of the Southern Medical University, Guangzhou, China.
⁵ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Institute for Tissue Engineering and Regenerative Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: alanli@cuhk.edu.hk.
⁶ Musculoskeletal Research Laboratory, Department of Orthopaedics and Traumatology, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; Innovative Orthopaedic Biomaterials and Drug Translational Research Laboratory, Li Ka Shing Institute of Health Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: jiankunxu@cuhk.edu.hk.
⁷ Department of Microbiology, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China. Electronic address: jiezhou@hku.hk.
⁸ Department of Biomedical Engineering, Faculty of Engineering, The Chinese University of Hong Kong, Hong Kong SAR, China; Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science and Technology Park, Hong Kong SAR, China; Institute for Tissue Engineering and Regenerative Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong SAR, China. Electronic address: tuanr@cuhk.edu.hk.

PMID: 40220744
DOI: 10.1016/j.medj.2025.100667

Abstract

Organoids and organs-on-chips (OoCs) are rapidly evolving technologies for creating miniature human tissue models. They can mimic complex physiological functions and pathological conditions, offering more realistic platforms for disease modeling, drug screening, precision medicine, and regenerative therapies. The passing of the FDA Modernization Act 2.0 has reduced animal testing requirements for drug trials, marking a significant milestone in using advanced in vitro models such as organoids and OoCs for therapeutic discovery. Apart from technical and ethical challenges, regulatory issues persist in ensuring the reliability, scientificity, and applicability of these models in drug development. This perspective explores the concept, advancements, pros and cons, and applications of organoids and OoCs, particularly in drug research and development. It also examines global regulatory agencies' policies and actions on using these models in drug evaluation, aiming to guide industry standard setting and advance regulatory science.

Keywords: compliance challenges; drug discovery and development; organoids; organs-on-chips; regulatory perspectives.

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Conflict of interest statement

Declaration of interests The authors declare no competing interests.

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Organoids and organs-on-chips: Recent advances, applications in drug development, and regulatory challenges

Affiliations

Organoids and organs-on-chips: Recent advances, applications in drug development, and regulatory challenges

Authors

Affiliations

Abstract

Conflict of interest statement

Publication types

MeSH terms

LinkOut - more resources

Full Text Sources

Miscellaneous