Advanced Therapy Medicinal Products (ATMPs): fundamental concepts, classification, and translational challenges

Abstract

Advanced Therapy Medicinal Products (ATMPs) represent a transformative class of innovative therapies based on genes, cells, or engineered tissues that aim to modify, repair, or replace biological functions at a fundamental level. This review provides a foundational overview of ATMPs, addressing their scientific basis, regulatory classification, clinical translation, and key challenges for future development. The article outlines the four principal categories of ATMPs: gene therapy medicinal products, somatic cell therapy medicinal products, tissue-engineered products, and combined ATMPs, and discusses their distinct mechanisms of action and therapeutic applications. Recent clinical successes, including chimeric antigen receptor T-cell therapies, gene replacement therapies for inherited disorders, and tissue-engineered constructs for regenerative medicine, demonstrate the paradigm shift from symptomatic management towards disease modification or potential cure. However, the clinical implementation of ATMPs presents substantial challenges related to safety monitoring, long-term efficacy, complex manufacturing processes, regulatory evaluation, high treatment costs, and equitable patient access. Ethical considerations, including informed consent, long-term follow-up obligations, and global disparities in availability, further complicate their integration into routine clinical practice. Regulatory agencies have introduced adaptive pathways and accelerated approval mechanisms to facilitate timely patient access while maintaining rigorous standards of quality, safety, and efficacy. Continued technological innovation, coupled with sustainable healthcare policies and international collaboration, will be essential to realise the full therapeutic potential of ATMPs. As evidence accumulates from clinical trials and real-world use, ATMPs are expected to play an increasingly prominent role in modern medicine and the future of personalised healthcare.

Keywords

Advanced Therapy Medicinal Products / gene therapy / cell-based therapy / tissue engineering / regenerative medicine / translational medicine / regulatory science

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