inovmedИнновационная медицина КубаниInnovative Medicine of Kuban2541-9897Scientific Research Institute – Ochapovsky Regional Clinical Hospital No. 110.35401/2541-9897-2025-10-4-113-120inovmed-1380Research ArticleОБЗОРЫREVIEWSОсобенности дизайна доклинических исследований препаратов генной терапии in vivo. Часть 1: фармакологические исследованияDesign Features of Nonclinical Studies of In Vivo Gene Therapy Medicinal Products. Part 1: Pharmacological studieshttps://orcid.org/0000-0001-8377-9205РачинскаяО. А.RachinskayaO. A.

Рачинская Ольга Анатольевна, к. б. н., ведущий эксперт лаборатории биомедицинских клеточных продуктов

127051, Москва, Петровский б-р, д. 8, стр. 2 

Olga A. Rachinskaya, Cand. Sci. (Bio.), Leading Expert, Laboratory of Biomedical Cell Products 

Petrovskii bulvar 8/2, Moscow, 127051 

Rachinskaya@expmed.ruhttps://orcid.org/0000-0002-9585-3545МельниковаЕ. В.MelnikovaE. V.

Мельникова Екатерина Валерьевна, к. б. н., начальник лаборатории биомедицинских клеточных продуктов

Москва  

Ekaterina V. Melnikova, Cand. Sci. (Bio.), Head of the Laboratory of Biomedical Cell Products 

Moscow

https://orcid.org/0000-0003-4891-973XМеркуловВ. А.MerkulovV. A.

Меркулов Вадим Анатольевич, д. м. н., профессор, заместитель генерального директора 

Москва 

Vadim A. Merkulov, Dr. Sci. (Med.), Professor, Deputy General Director

Moscow 

Научный центр экспертизы средств медицинского примененияРоссияScientific Centre for Expert Evaluation of Medicinal ProductsRussian Federation202530122025104113120Copyright © Рачинская О.А., Мельникова Е.В., Меркулов В.А., 20252025Рачинская О.А., Мельникова Е.В., Меркулов В.А.Rachinskaya O.A., Melnikova E.V., Merkulov V.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.innovmedkub.ru/jour/article/view/1380

Статья посвящена особенностям дизайна доклинических исследований (ДКИ) и проведению фармакологических исследований препаратов генной терапии (ГТЛП) in vivo, основанных на введении векторов с рекомбинантными нуклеиновыми кислотами в организм пациента для коррекции генетических нарушений и терапии онкологических заболеваний. К 2025 г. в мире зарегистрировано около 20 in vivo ГТЛП, включая три в РФ. Отсутствие единых регуляторных требований к ДКИ обусловлено быстрым развитием технологий и ограниченным опытом экспертной оценки. В работе подчеркивается необходимость гармонизации требований ЕАЭС со стандартами ЕС к проведению ДКИ in vivo ГТЛП, приводится общая схема ДКИ, указываются риски, связанные с использованием этой группы препаратов, и отмечаются основные аспекты проведения фармакологических исследований, выявленные в результате анализа экспертных отчетов мировых производителей ГТЛП. 

The article focuses on the specific aspects of nonclinical studies (NCS) and the cunduct of pharmacological investigations of in vivo gene therapy medicinal products (GTMPs) which involve the administration of vectors with recombinant nucleic acids into the patient’s body for the correction of genetic disorders and treatment of oncological diseases. As of 2025 approximately 20 in vivo GTMPs have been authorized worldwide, including 3 in the Russian Federation. Due to the rapid technological advances and limited expertise in regulatory assessment, harmonized requirements for preclinical studies are lacking. The article emphasizes the necessity of harmonizing the Eurasian Economic Union regulations with the European Union standards conducting preclinical in vivo studies of GTMPs, the general outline of the NCS is provided, the risks associated with the use of this class of medical products are indicated, and the key aspects of pharmacological studies identified as a result of the analysis of public assessment reports from global GTMP developers, are noted. 

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The authors declare that there are no conflicts of interest present.