Criteria for the application of the platform approach for mRNA vaccines and gene therapy products in regulatory practice

INTRODUCTION. The platform approach, which involves the use of an unchanged manufacturing process and standardized quality control methods, is recognized by leading regulatory authorities, such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), as an effective tool for accelerating market entry of innovative products, namely mRNA vaccines and gene therapy products. In this context, the implementation of this concept is relevant for the Russian Federation and the countries of the Eurasian Economic Union (EAEU), as it would accelerate pharmaceutical development, optimize quality control and process validation, and improve regulatory review during the registration of such products.

AIM. This study aimed to analyze international regulatory approaches to the application of platform technologies in the manufacturing and quality control of mRNA vaccines and gene therapy products based on viral vectors and genome editing systems, in order to provide a rationale for their implementation in the national legislation and regulatory documentation of the Russian Federation and the EAEU.

DISCUSSION. An analysis of publications from 2015 to 2025 in the PubMed, Google Scholar, and eLIBRARY.ru databases, as well as official documents from regulatory authorities, including the FDA, the EMA, the World Health Organization (WHO), the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), the Medicines and Healthcare products Regulatory Agency, and the Pharmaceuticals and Medical Devices Agency (PMDA), addressing the application of platform technologies in the manufacturing and quality control of medicinal products, was performed. It was found that the regulatory documentation of the Russian Federation and the EAEU lacks criteria for applying the platform approach to mRNA vaccines and gene therapy products, which hinders the accelerated introduction of innovative personalized medicinal products into clinical practice. International regulatory approaches (FDA, EMA, PMDA) were shown to accelerate development and process validation, and reduce the scope of preclinical studies and clinical trials through the reuse of data on previously manufactured and approved products (including data on the manufacturing process and safety profiles for mRNA vaccines and gene therapy products). The main unresolved issues remain the amount of supporting data and the need for a first approved product before platform technology status can be granted. The need to develop unified regulatory requirements for relevant experience and process validation was substantiated, which would allow the benefits of the platform approach to be realized while maintaining a high level of safety and efficacy of medicinal products during regulatory review.

CONCLUSIONS. The platform approach, based on an unchanged manufacturing process and standardized quality control methods, can accelerate pharmaceutical development, reduce the extent of preclinical studies and clinical trials, and optimize the regulatory review of mRNA vaccines and gene therapy products. To implement this approach in the Russian Federation and the EAEU, it is necessary to incorporate provisions into the regulatory documentation allowing the use of standardized modules within a single technological platform, which would accelerate the development and market launch of gene therapy products and personalized products, and increase their affordability.

1. Skerritt JH, Tucek-Szabo C, Sutton B, Nolan T. The platform technology approach to mRNA product development and regulation. Vaccines (Basel). 2024;12(5):528. https://doi.org/10.3390/vaccines12050528

2. Urnov F, Kassim S, Musunuru K, et al. Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: Key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher. Cytotherapy. 2025;27(10):1151–63. https://doi.org/10.1016/j.jcyt.2025.06.010

3. Ogawa T, Sunyi J, Kawachi K, et al. Regulatory approaches for platform-based vaccine development in Japan: Insights from PMDA’s experience with COVID-19 and RSV vaccines. Vaccine. 2026;76:128315. https://doi.org/10.1016/j.vaccine.2026.128315

4. Musunuru K, Grandinette SA, Wang X, et al. Patient-Specific in vivo gene editing to treat a rare genetic disease. N Engl J Med. 2025;392(22):2235–43. https://doi.org/10.1056/NEJMoa2504747

5. Bagdasarova DV, Bolotina LV, Leukhina IA, et al. mRNA technologies in treatment of oncological diseases: global experience of clinical trials and implementation in the Russian Federation. P.A. Herzen Journal of Oncology. 2025;14(6):69–77 (In Russ.). https://doi.org/10.17116/onkolog20251406169

6. Kopein DS, Poroshin GN, Khamitov RA. Implementation of the quality-by-design concept for an adeno-associated viral vector-based gene therapy. Biological Products. Prevention, Diagnosis, Treatment. 2025;25(2):141–55 (In Russ.). https://doi.org/10.30895/2221-996X-2025-580

7. Bulcha JT, Wang Y, Ma H, et al. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6(1):53. https://doi.org/10.1038/s41392-021-00487-6

8. Young P. Treatment to cure: Advancing AAV gene therapy manufacture. Drug Discov Today. 2023;28(7):103610. https://doi.org/10.1016/j.drudis.2023.103610

9. Stoops J, Morton T, Powell J, et al. Treg cell therapy manufacturability: current state of the art, challenges and new opportunities. Front Immunol. 2025;16:1604483. https://doi.org/10.3389/fimmu.2025.1604483

10. Whitley J, Zwolinski C, Denis C, et al. Development of mRNA manufacturing for vaccines and therapeutics: mRNA platform requirements and development of a scalable production process to support early phase clinical trials. Transl Res. 2022;242:38–55. https://doi.org/10.1016/j.trsl.2021.11.009

11. Zhang G, Tang T, Chen Y, et al. mRNA vaccines in disease prevention and treatment. Signal Transduct Target Ther. 2023;8(1):365. https://doi.org/10.1038/s41392-023-01579-1

12. Li M, Wang Z, Xie C, et al. Advances in mRNA vaccines. In: International Review of Cell and Molecular Biology. Elsevier; 2022. P. 295–316. https://doi.org/10.1016/bs.ircmb.2022.04.011

13. Pardi N, Hogan MJ, Porter FW, Weissman D. mRNA vaccines — A new era in vaccinology. Nat Rev Drug Discov. 2018;17(4):261–79. https://doi.org/10.1038/nrd.2017.243

14. Schrieber SJ, Putnam WS, Chow ECY, et al. Comparability considerations and challenges for expedited development programs for biological products. Drugs RD. 2020;20(4):301–6. https://doi.org/10.1007/s40268-020-00321-4

15. Castellanos MM, Gressard H, Li X, et al. CMC strategies and advanced technologies for vaccine development to boost acceleration and pandemic preparedness. Vaccines (Basel). 2023;11(7):1153. https://doi.org/10.3390/vaccines11071153

16. Madabushi R, Seo P, Zhao L, et al. Review: Role of model-informed drug development approaches in the lifecycle of drug development and regulatory decision-making. Pharm Res. 2022;39(8):1669–80. https://doi.org/10.1007/s11095-022-03288-w

17. Brooks PJ, Ottinger EA, Portero D, et al. The platform vector gene therapies project: Increasing the efficiency of adeno-associated virus gene therapy clinical trial startup. Hum Gene Ther. 2020;31(19– 20):1034–42. https://doi.org/10.1089/hum.2020.259

18. Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229–36. https://doi.org/10.1038/s41586-020-1978-5

19. Chan AYL, Chan VKY, Olsson S, et al. Access and unmet needs of orphan drugs in 194 countries and 6 areas: A global policy review with content analysis. Value Health. 2020;23(12):1580–91. https://doi.org/10.1016/j.jval.2020.06.020

20. Drennen J, Anderson C. Comparability protocols: Chemistry, manufacturing and controls information. NIR News. 2003;14(3):14–5. https://doi.org/10.1255/nirn.720

21. Sanyal G, Särnefält A, Kumar A. Considerations for bioanalytical characterization and batch release of COVID-19 vaccines. npj Vaccines. 2021;6(1):53. https://doi.org/10.1038/s41541-021-00317-4

22. Vaganova OA, Smirnov RS, Lenin SA, et al. Monoclonal antibodies: Development of universal (platform) methods for the assessment of high and low-molecular weight impurities. Regulatory Research and Medicine Evaluation. 2025;15(3):330–46 (In Russ.). https://doi.org/10.30895/1991-2919-2025-15-3-330-346