Introduction. Gene and cell therapies represent one of the fastest growing areas of modern medicine, offering new opportunities for the treatment of diseases with limited or ineffective conventional therapeutic options.
The aim of this article is to review the main regulatory aspects, principles, types and most significant clinical applications of Advanced Therapy Medicinal Products (ATMPs), with a focus on gene therapies, somatic cell therapies and tissue engineering products.
The methodological approach is aimed at systematizing and critically analyzing the available scientific data on the main types of ATMPs. A documentary method was also applied to establish a regulatory framework and main challenges in the implementation of advanced therapy medicinal products in clinical practice.
Results. The mechanisms of action of gene therapies are analyzed, including the use of viral and non-viral vectors for the delivery of therapeutic genes, as well as the role of modern gene editing technologies. Key aspects of cell therapies are presented, including autologous and allogeneic approaches, cell modification methods, and challenges related to manufacturing, standardization, and quality control. Special attention is paid to the clinical application of these therapies in oncology, hematology, and rare hereditary diseases, where significant therapeutic progress has been reported. The article also discusses the main risks and limitations of gene and cell therapies, including issues of safety, immunogenicity, long-term follow-up, and high cost. The regulatory framework in the European Union is reviewed, emphasizing the importance of strict control, pharmacovigilance, and a multidisciplinary approach in the development and implementation of ATMPs. In conclusion, gene and cell therapies are emerging as a key element of personalized medicine, with the potential to change therapeutic strategies for a number of severe and chronic diseases. Their future development depends on the accumulation of long-term clinical data, the improvement of manufacturing technologies, and the provision of sustainable access for patients.
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