Since the discovery of deoxyribonucleic acid (DNA) in the 19th century, the global scientific community has succeeded in elucidating the mechanisms governing heredity and variability. The ability to introduce localized changes into the human genome has been a goal of medicine ever since DNA became known as the fundamental unit of heredity. In the late 20th century, following the deciphering of the DNA molecule, various DNA editing methodologies began to emerge, the most promising and successful of which to date is gene therapy (GT).

Gene therapy holds the promise of becoming a powerful tool for transitioning medicine to a fundamentally new level of development through human genome editing. In cases where traditional pharmacotherapeutic methods have failed to achieve the desired results, GT may offer an effective treatment option [1]. The fifty-year journey of GT from theory to clinical application has demonstrated the complexity of the practical implementation of this concept at various stages [2], ranging from defining the concept and addressing research design and ethical issues to evaluating therapeutic efficacy. Gene and cell therapy represent innovative directions in medicine aimed at treating diseases by targeting the body’s genetic material and cellular structures to correct the root causes of pathology. This approach offers high therapeutic potential but requires rigorous scientific, legal, and ethical oversight. Gene therapy acts directly on the genetic basis of a disease and can be implemented by introducing a functional copy of a gene to compensate for a lost or defective function, inactivating a pathologically active gene, or performing targeted DNA editing to correct mutations; the delivery of genetic material is accomplished using viral or non‑viral vector systems that enable transgene entry into target cells and its expression. Cell therapy, in turn, is based on introducing autologous or allogeneic cells into a patient’s body; these cells either directly replace damaged cells and tissues or exert a therapeutic effect through the secretion of biologically active factors and immunomodulation. In some cases, cells are pre‑genetically modified, allowing both approaches to be combined, as in CAR‑T therapy (chimeric T‑lymphocytes). Thus, the mechanisms of action of gene and cell therapy are aimed not at symptomatic treatment but at eliminating the root causes of disease or restoring disrupted biological functions, which fundamentally distinguishes them from traditional pharmacological methods.

According to Russian legislation, gene therapy medicinal products are medicinal products whose pharmaceutical substance is a recombinant nucleic acid or contains a recombinant nucleic acid enabling the regulation, repair, replacement, addition, or deletion of a genetic sequence [3].

Gene therapy, as defined by the U.S. Food and Drug Administration (FDA), is a medical intervention based on the modification of the genetic material of living cells. This branch of medicine aims to correct impaired gene functions, reduce their activity, or introduce new genes that enable the body to acquire missing functions. Genetic therapy of germ cells and early‑stage embryos is technically possible [4] but is primarily restricted by ethical norms and is not legally controlled. Within this method of targeting the cell’s genetic apparatus, a wide variety of agents are used, ranging from whole viruses that deliver DNA sequences into the cell nucleus to inorganic gels. Currently, gene therapy is a field that exists mainly in research laboratories, and its clinical application remains largely experimental. The indications for gene therapy are quite broad and include the potential treatment of diseases caused by recessive gene disorders (cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia), acquired genetic diseases such as cancer, and certain viral infections such as HIV.

At the same time, the legal regulation of genome editing should be understood as a system of legal instruments—legal relationships, self‑regulatory acts, individual directives, judicial practice, intellectual property, administrative regulations, and criminal law norms—through which regulatory and organizational influence is exerted on the entire set of relationships concerning human genome editing in order to order, develop, and protect them in accordance with international standards and social needs [5]. Thus, this issue is interdisciplinary, requiring consideration from the perspectives of legal questions, ethical support, and clinical feasibility.

Gene therapy products are regulated in different countries taking into account both medical and ethical risks, as they involve intervention into human genetic material. Legally, in the United States, EU countries, and the Russian Federation, gene therapy is subject to strict state control, mandatory clinical trials, and long‑term patient follow‑up, while genome editing that could affect future generations is prohibited or under moratorium in most states. Ethical concerns relate to possible long‑term consequences, the need for fully informed consent, the inadmissibility of using gene technologies for human “enhancement,” and limited access to treatment due to its high cost. Despite the existence of international bioethical principles, uniform global regulatory standards for gene therapy have not yet been developed, reflecting differences in legal systems and cultural approaches.

Objective: To analyze available international legislative acts pertaining to the development, application, and ethical principles of GT use that are found in open literature sources.

A search query was formulated for the Russian legal reference system “Consultant Plus” (https://www.consultant.ru, accessed 03/02/2026); the English‑language website of the United Nations (UN) with published declarations (https://www.un.org/ru/documents/decl_conv/declarations/human_genome.shtml, accessed 23/01/2026) and the FDA website (https://www.fda.gov, accessed 06/02/2026); as well as the English‑language database of the National Archives (https://www.ecfr.gov) and the European Union database (https://eur‑lex.europa.eu, accessed 18/01/2026). For the Consultant Plus system, the query was formulated as “gene therapy”. For the UN and FDA websites, as well as for the EU and U.S. National Archives databases, the query was formulated as “regulatory acts” + “gene therapy”. The following eligibility criteria were developed and applied:

Inclusion criteria: a current regulatory act having the status of a legislative act, regulating the development, clinical research, manufacturing, and application of gene therapy medicinal products, the text of which is published in Russian or English.Exclusion criteria: publications devoted exclusively to clinical aspects of using gene therapy products; administrative and judicial practice data; commentary on legislation; drafts of legal acts; regulatory acts in which gene therapy is an exclusion criterion.

The search results are presented in a diagram (Fig. 1).

Fig. 1. Literature source search sequence and results

During the writing of this article, 321 publications were found (including regulatory acts in which gene therapy is an exclusion criterion or is merely mentioned). After excluding 260 publications that were devoted exclusively to clinical aspects of using gene therapy products; constituted administrative or judicial practice data; or represented commentary or draft legislation, 61 publications remained. After reading the full texts, a further 34 regulatory acts were excluded (gene therapy only mentioned or used as an exclusion criterion), leaving 27 regulatory acts for analysis. All regulatory acts used are current as of the end of January 2026, and amendments made up to the time of writing have been taken into account. Five acts are international; the remaining 22 are part of domestic legislation.

International documents. At present, the field of international biological law is actively developing, and a distinct legal framework for the regulation of human genome editing technology is emerging. Among the relevant international legislative acts are the UNESCO Universal Declaration on the Human Genome and Human Rights [6], the UNESCO International Declaration on Human Genetic Data [7], the UNESCO Universal Declaration on Bioethics and Human Rights [8], and the WHO (2021) Guidelines on Human Genome Editing [9].

In 2019, the WHO (World Health Organization) Advisory Committee on the Governance and Oversight of Human Genome Editing Activities was established to develop a regulatory framework for genome editing activities. The legal acts presented below are international ethical and legal instruments of a recommendatory nature, aimed at regulating the rapidly evolving field of biomedicine and genetics. They are not legally binding but serve as an important foundation for the formation of national legislation and international standards in the field of human rights protection in genetic research.

The Universal Declaration on the Human Genome and Human Rights (1997) is of fundamental importance. It was the first to establish the idea of the human genome as the common heritage of humanity and to emphasize the primacy of human dignity over scientific and commercial interests. The document introduces basic ethical principles: the inadmissibility of discrimination based on genetic characteristics, the prohibition of the commercialization of the genome, the need for informed consent, compensation for harm, and international cooperation. Of particular significance is the prohibition of practices contrary to human dignity, including reproductive cloning.The International Declaration on Human Genetic Data (2003) is more applied in nature and focuses on the collection, storage, use, and transfer of genetic information. Although the document does not directly regulate genome editing, it establishes strict requirements for the protection of privacy, confidentiality, and security of genetic data, which directly impacts gene therapy and research practices.The Universal Declaration on Bioethics and Human Rights (2005) is comprehensive and interdisciplinary. It integrates the principles of previous documents and expands them to include issues of human responsibility for the environment, biodiversity, and the sustainable use of biological and genetic resources. The Declaration emphasizes the need for a balance between scientific progress, human rights, and the protection of nature.The WHO Guidelines on Human Genome Editing (2021) establish a system of ethical and regulatory principles for the governance of genome editing technologies (including CRISPR/Cas9 technologies—targeted genome editing techniques based on the use of a guide RNA and Cas enzyme for precise cutting and subsequent alteration of DNA sequences), affirm the priority of respect for human dignity, protection of rights and personal autonomy, and establish a prohibition on the clinical application of interventions aimed at heritable human genome modifications and the creation of “designer babies”. “Designer babies” is an ethically and normatively problematic concept used to denote the hypothetical practice of intentional editing of an embryo’s genome to select or enhance non‑medical traits that would be inherited by subsequent generations.

Collectively, these documents form a unified international bioethical approach according to which the development of genetics and biomedicine is permissible only under strict observance of human rights, respect for human dignity, and prevention of abuse of scientific achievements. Their overarching meaning is that scientific progress cannot be an end in itself and must serve humanity, not threaten it. UNESCO proceeds from the idea that the human genome is not an object of free market or unconstrained experimentation but a value requiring special protection. At the same time, the importance of international cooperation, research transparency, and the formation of common ethical standards in the context of the global development of genomic technologies is emphasized.

Experience of the United States. As of the writing of this article (February 2026), 12 gene therapy products and 36 cell therapy products have been registered in the United States; the list is publicly available on the FDA website [10]. The regulation of gene therapy in the United States is primarily focused on protecting patient health and ensuring the clinical safety of innovative treatments. The regulatory framework covers all stages of the life cycle of gene therapy products—from preclinical studies and first‑in‑human applications to long‑term follow‑up after therapy completion. The main legislative acts are:

The Federal Food, Drug, and Cosmetic Act [11], FDA guidance documents [12].The United States Code [13] regulates the conduct of clinical trials of new drugs, including gene therapies.The National Institutes of Health [14] establishes ethical standards for conducting state‑funded research, including gene therapy.

The key legislative act is the Federal Food, Drug, and Cosmetic Act (FD&C Act), under which gene therapy is classified as a drug or biological product. From a medical standpoint, this Act establishes the requirement for a preliminary assessment of the potential risks and benefits of therapy before human application. The central element is the Investigational New Drug (IND) application procedure, without which clinical studies cannot be conducted. This procedure aims to minimize risks to patients and allows the FDA to assess preclinical data on toxicity, biodistribution, immunogenicity, and anticipated therapeutic efficacy.

The mechanisms for implementing requirements for gene therapy products are detailed in 21 CFR Part 312, which defines the conduct of Phase I–III clinical trials. Of particular importance for clinical practice are the requirements for study protocols, patient inclusion and exclusion criteria, monitoring of adverse drug reactions, and immediate reporting of serious side effects to the regulator. These provisions ensure continuous medical oversight of clinical trial participants.

FDA guidance documents on gene therapy play a significant role in the clinical development of gene therapy, shaping clinical and manufacturing practice standards. The “Guidance for Industry: Human Gene Therapy” focuses on specific medical risks associated with the use of gene constructs, including the possibility of immune reactions, oncogenicity, and unpredictable effects of long‑term gene expression. In the medical context, the requirement for long‑term (up to 15 years) post‑treatment patient follow‑up is important, reflecting the principle that safety prevails over the speed of innovation implementation.

The FDA Guidance on Chemistry, Manufacturing, and Controls (CMC) has direct clinical significance because the stability, purity, and manufacturing of a gene therapy product directly affect treatment efficacy and the risk of adverse events. Strict requirements for adherence to Good Manufacturing Practice (GMP) standards and quality control of vector systems aim to reduce the likelihood of contamination, dose variability, and unpredictable clinical effects.

The U.S. National Institutes of Health has developed a set of guidelines for research involving recombinant or synthetic nucleic acid molecules, including gene therapies (Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules). Under these guidelines, institutions receiving NIH grants are required to comply with safety, ethical review, and biosafety control requirements for research.

Thus, from a medical perspective, the regulatory framework for gene therapy in the United States represents a system oriented toward evidence, clinical safety, and long‑term patient follow‑up. This approach ensures the controlled introduction of gene therapy technologies into clinical practice and reduces the risks associated with the use of high‑tech biomedical interventions.

Experience of the European medical community. In the European Union, gene therapy falls under the category of Advanced Therapy Medicinal Products (ATMPs) and is centrally regulated through the European Medicines Agency (EMA). This centralized procedure ensures a uniform standard for assessing the safety, efficacy, and quality of gene therapy products across all EU countries. The EMA also provides scientific support to developers in organizing pharmacovigilance systems and risk management after product market entry.

The main EU regulatory acts include:

Directive 2001/83/EC [15] defines gene‑based products as using genetic material for the treatment or prevention of human diseases; establishes requirements for clinical trials, manufacturing, safety, and adherence to ethical principles.Directive 2009/120/EC [16] specifies the scientific and technical requirements for gene therapy medicinal products, establishes registration and licensing procedures, and regulates the use of recombinant genes to achieve therapeutic, prophylactic, or diagnostic effects.Regulation (EU) No 536/2014 [17] governs the conduct of clinical trials, including gene therapy products, establishing uniform application procedures, requirements for participant safety monitoring, informed consent, and compliance with ethical standards.The Convention on Human Rights and Biomedicine (Oviedo, 1997) [18] permits the application of gene therapy exclusively for therapeutic and prophylactic purposes, provided that interventions are limited to human somatic cells and that the principles of primacy of human dignity, informed consent, and proportionality of risk and benefit are strictly observed, while simultaneously prohibiting interventions in the genome aimed at modifying hereditary characteristics. The Additional Protocol on the Prohibition of Human Cloning (1998) reinforces this approach, excluding any form of reproductive human cloning as incompatible with respect for the person.The Additional Protocol on Biomedical Research (2005) [19] of the Council of Europe regulates biomedical research involving human subjects, including research and clinical trials in the field of gene therapy, establishing requirements for scientific validity, informed voluntary consent, independent ethical review, the priority of the subject’s interests and dignity, and restrictions on interventions involving increased or unjustified risk.Regulation (EC) No 1394/2007 on advanced therapy medicinal products (ATMPs) establishes the legal framework for the development, evaluation, registration, and supervision of gene therapy products, defining them as a special category of medicinal products and setting out requirements for quality, safety, efficacy, pharmacovigilance, and protection of patient rights.Regulation (EU) 2016/679 (GDPR) [General Data Protection Regulation] establishes the legal framework for the processing of personal data in the European Union, including genetic and medical data used in research and clinical practice of gene therapy, enshrining the principles of lawfulness, data minimization, purpose limitation, confidentiality, enhanced protection of sensitive data, and expanded rights of patients as data subjects.

Thus, EU legislation creates a comprehensive system of control and support for gene therapy, combining scientific, technical, clinical, and ethical standards for the safe introduction of innovative treatment methods.

Experience of the United Kingdom. In the United Kingdom, the regulation of gene therapy represents a strict multi‑level system combining medical, bio‑regulatory, ethical, and human rights oversight. Gene therapy is considered a form of high‑tech medical care aimed at treating diseases, not at non‑medical enhancement of human characteristics. Clinical application of gene therapy in the UK is permitted only for somatic cells, while any interventions aimed at modifying the germline and transmitting genetic modifications by inheritance remain prohibited.

The central role in state regulation of gene therapy is played by the Medicines and Healthcare products Regulatory Agency (MHRA), which evaluates, authorizes, and supervises clinical trials and gene therapy medicinal products. The regulator verifies the compliance of such products with quality, safety, and efficacy requirements and ensures subsequent pharmacovigilance.

Research involving the use of human embryos is under the control of the Human Fertilisation and Embryology Authority (HFEA). The Human Fertilisation and Embryology Act 1990 [20] is the primary law regulating work with human embryos and reproductive technologies. Basic research on human embryos up to 14 days of development is permitted; however, implantation of genetically modified embryos for reproductive purposes is prohibited. This underscores a clear distinction between permissible scientific research and impermissible clinical practices.

Ethical review and protection of research participants are ensured by a system of independent oversight coordinated by the Health Research Authority. Special attention is given to informed voluntary consent, risk‑benefit assessment, and protection of vulnerable groups. Additionally, the processing of patients’ genetic data is regulated under the UK GDPR (United Kingdom General Data Protection Regulation), which provides enhanced protection of sensitive personal data. The normative act regulating ethical standards for the application of gene therapy is the UK Policy Framework for Health and Social Care Research [21], which defines uniform standards for ethical and scientific governance of biomedical research, including gene therapy research, with an emphasis on participant protection and public accountability.

The conduct of clinical trials of medicinal products for human use, including gene therapy products, is regulated by the Medicines for Human Use (Clinical Trials) Regulations 2004 [22]. It establishes requirements for: scientific validity; risk‑benefit assessment; informed voluntary consent; and regulatory approval of studies.

The Human Medicines Regulations 2012 [23] establish the legal regime for the circulation of medicinal products for human use in the UK, including: classification of gene therapy products as a special category; requirements for quality, safety, and efficacy; and pharmacovigilance after product authorization.

Thus, the regulation of gene therapy in the United Kingdom represents a comprehensive legal and ethical model aimed at supporting scientific progress while ensuring the primacy of human dignity, patient safety, and the prevention of heritable genetic interventions.

Experience of Japan. The PMD Act (Pharmaceuticals and Medical Devices Act, 2014) of Japan introduces special accelerated regulatory mechanisms for innovative therapies, including cell and gene‑based methods, providing for conditional and time‑limited approval based on early clinical data [24]. Regulation and assessment of safety, efficacy, and quality are carried out by the supervisory authority PMDA (Pharmaceuticals and Medical Devices Agency).

Experience of China. The regulation of gene therapy in China is characterized by a strict centralized state control model, established and significantly strengthened after 2018. Gene therapy is permitted only for therapeutic purposes and is subject to both pharmaceutical and biosafety regulation. Gene therapy standards applied in China are overseen by the National Medical Products Administration (NMPA).

The basic legal framework is set out in the Drug Administration Law of the PRC [25], 2019, which focuses on enhanced control of innovative interventions with an elevated risk level and covers all stages of the life cycle of gene therapy products: from extended preclinical safety, immunogenicity, and oncogenic potential assessment to phased clinical trials, strict GMP manufacturing requirements, and mandatory long‑term post‑registration patient monitoring. At the same time, the regulatory model directly excludes clinical editing of the human germline and any heritable genomic changes.

Experience of Russia. Russian legislation does not contain a unified definition of gene therapy: according to Federal Law No. 86‑FZ [26], gene therapy (genotherapy) is a set of genetic engineering (biotechnological) and medical methods aimed at introducing changes into the genetic apparatus of human somatic cells for the purpose of treating diseases; while according to Federal Law No. 61‑FZ [3], gene therapy medicinal products are medicinal products whose pharmaceutical substance is a recombinant nucleic acid or contains a recombinant nucleic acid enabling the regulation, repair, replacement, addition, or deletion of a genetic sequence.

In Russia, gene therapy products are in a stage of active development and regulation. To date, several gene therapy products have been registered and are used in the country (e.g., voretigene neparvovec, onasemnogene abeparvovec), and clinical trials of novel unique products are underway (e.g., ANB‑002 [27] for the treatment of hemophilia B is in Phase III clinical trial).

Federal Law No. 86‑FZ “On Biomedical Cell Products” was adopted in Russia in 2010; in addition to defining gene therapy, this law establishes the legal framework for the development, production, ethical aspects, clinical trials, and application of gene therapy.

The Ministry of Industry regulates the production of gene therapy products by addressing such matters as personnel safety, equipment requirements, and zoning of premises when working with biological materials [28].

An international regulatory act governing the rules for the use of raw materials for gene therapy production is Decision No. 100 of the Board of the Eurasian Economic Commission dated 11.08.2020 [29].

The procedure for registration and application of gene therapy products in Russia is regulated by Federal Law No. 61‑FZ “On Circulation of Medicinal Products”. According to this legislative act, the registration of gene therapy products does not differ from that of other medicinal products.

Federal Law No. 180‑FZ of 23.06.2016 “On Biomedical Cell Products” [30] regulates the development, testing, and circulation of cell products, including gene therapy. It provides for state registration, safety and efficacy review.

The main difference among national models of gene therapy regulation lies not so much in market access as in the depth and duration of pharmacovigilance: the EU, the United States, and Japan have institutionalized special long‑term follow‑up schemes (up to 15 years) with mandatory risk management plans, whereas in Russia and, to a lesser extent, China, pharmacovigilance is more centralized and less differentiated.

Table 1. Summary table of international and national regulatory acts on gene therapy

 Scope of applicationKey regulatory actsRegulatorPermissible target of interventionAccelerated registrationPharmacovigilance featuresProhibitions and restrictionsClinical trial phasesFeatures of early phasesInformed voluntary consent (IVC)European UnionRegulations (EC) No 1394/2007, 2016/679; Directives 2001/83/EC, 2009/120; Convention on Human Rights and Biomedicine; Additional Protocol on Biomedical ResearchEMASomatic cellsYes (conditional approval, accelerated assessment)Long‑term pharmacovigilance 5‑15 years, monitoring of delayed effectsProhibition of heritable human genome changesI–III (sometimes combined I/II)Enhanced requirements for preclinical data and biosafetyExtended IVC specifying long‑term and heritable risksUnited KingdomMedicines for Human Use (Clinical Trials) Regulations; Human Fertilisation and Embryology ActMHRA; HFEASomatic cellsYes (accelerated process)Extended pharmacovigilance, national clinical registries, long‑term follow‑upProhibition of implantation of genetically modified embryosI–III (adaptive designs)Early risk assessment, staged cohort expansionDetailed IVC + continuous patient informationUnited StatesFederal Food, Drug, and Cosmetic Act; U.S. Code Title 21 Part 312; Guidance for Industry: Human Gene Therapy Products Involving Human Genome EditingFDASomatic cellsYes (fast track, accelerated approval)Mandatory follow‑up up to 15 years, active post‑marketing studiesProhibition of clinical germline editingI–III (often I/II)IVC with enhanced safety requirementsParticularly detailed IVC specifying delayed effectsJapanPharmaceuticals and Medical Devices ActPMDASomatic cellsYes (conditional/time‑limited approval)Real‑world data collection as condition for maintaining approvalProhibition of reproductive application of genome editingI–III (early conditional assessment possible)Permitted with limited data under high medical needIVC emphasizing uncertainty of efficacyChinaDrug Administration Law of the PRCNMPASomatic cellsYes (priority review)Centralized oversight, focus on safety and delayed risksDirect prohibition of heritable genome interventionsI–III (strictly sequential)Enhanced biosafety requirementsMandatory written IVC, enhanced controlRussiaFederal Law No. 61‑FZ; Federal Law No. 86‑FZMinistry of Health of the RF; RoszdravnadzorSomatic cellsLimitedGeneral pharmacovigilance regime without specialized long‑term programsProhibition of heritable human genome changesI–IIIClassical clinical phase modelIVC under general clinical trial rules

The regulation of the development and application of gene therapy is a complex and rapidly evolving field at the intersection of science, ethics, and law. The goal of regulation is to ensure the safety, efficacy, and ethical soundness of new treatments without stifling innovation. The regulation of gene therapy development and application represents a strict, multi‑level system designed to ensure patient safety, treatment efficacy, and ethical processes. It differs significantly from the regulation of classical drugs due to the unique characteristics and potential risks. The regulation of gene therapy application in modern legal systems is being formed as a comprehensive control model at all stages of the technology life cycle—from preclinical research to registration, manufacturing, and long‑term post‑registration surveillance. In all legislative frameworks analyzed in this article, gene therapy is recognized as permissible exclusively for therapeutic purposes and is regarded as a high‑risk medical technology requiring special regulatory and ethical mechanisms.

Clinical trials of gene therapy are characterized by enhanced requirements for the preclinical evidence base, the mandatory phased clinical testing, and the possibility of adaptive designs, with early phases and extended Phase III trials aimed at identifying long‑term effects being of key importance. It should be noted that clinical trials of gene therapy are often conducted on very small numbers of patients (sometimes tens or hundreds of individuals). At this stage, it is impossible to detect rare (<1%) or very delayed adverse effects, leading to market approval of drugs with an incompletely understood safety profile. Registration and market access are carried out either through the standard procedure or using accelerated and conditional approval procedures (Conditional Marketing Authorisation, CMA) for products with high clinical potential, while maintaining obligations for additional data collection after product authorization.

The manufacturing of gene therapy products is subject to strict Good Manufacturing Practice (GMP) requirements, driven by the high complexity and potential risks of such products. Quality control extends to all therapy components, primarily the vector systems for genetic material delivery: for viral vectors (adeno‑associated, lentiviral), verification of genetic stability, purity, absence of replication‑competent viruses, and reproducibility of the manufacturing process are mandatory, whereas for non‑viral systems the emphasis is on physicochemical stability and biocompatibility. In the case of cellular gene therapy products, GMP requirements additionally cover cell identification and traceability, control of viability, functional activity, contamination prevention, as well as validation of cultivation, storage, and transport processes. Overall, the manufacturing and quality control of gene therapy are regarded by regulators as critically important elements of safety and efficacy, and any changes to the technological process are subject to additional regulatory assessment.

A particularly important role is played by post‑registration surveillance, which for gene therapy transforms into long‑term pharmacovigilance using risk management plans, post‑registration safety studies, and specialized patient registries. Post‑registration follow‑up for gene therapy is not merely a continuation of clinical practice but a critically important and uniquely complex component of the entire product life cycle. This is due to the fundamental characteristics of gene therapy products themselves. Unlike an ordinary drug that is eliminated from the body, the active component of gene therapy (e.g., a functional gene integrated via a viral vector) may remain in the patient’s cells for decades. This means that adverse effects may also manifest many years after administration. Delayed risks may include insertional oncogenesis (risk of vector integration near an oncogene and triggering cancer)—a classic “delayed” risk requiring follow‑up of 10‑15 years or more; delayed immune responses to both the vector and the new protein now produced by the body; and loss of efficacy (gene expression may decline over time, requiring long‑term monitoring of disease markers).

Post‑registration surveillance for gene therapy is a large‑scale, long‑term, and costly research program integrated into clinical practice. It redefines the very approach to drug safety, shifting the focus from the short period of clinical trials to the patient’s entire lifetime. Thus, gene therapy is regulated not as a one‑time medical intervention but as a long‑term biomedical process requiring continuous monitoring, international coordination, and a balance between innovation and the protection of human rights.

The regulation of gene therapy is a dynamic and constantly evolving process. While the United States and the EU have already established but flexible regulatory processes, the Russian system is actively developing, striving to integrate international experience into the national legal framework.

[1] Regulation (EC) No 1394/2007 https://eurlex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2007:324:0121:0137:en:PDF

[2] Regulation (EU) 2016/679 (GDPR) https://www.ugmk-clinic.ru/media/uploads/2022/04/29/2016-679-27.pdf