Design Features of Nonclinical Studies of In Vivo Gene Therapy Medicinal Products. Part 1: Pharmacological studies
https://doi.org/10.35401/2541-9897-2025-10-4-113-120
Abstract
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.
Supporting agencies: The study was carried out within the framework of FSBI "SCEEMP" No. 056-00001-25-00 for applied scientific research (Research State Registration Number No. 124022200093-9).
About the Authors
O. A. Rachinskaya
Scientific Centre for Expert Evaluation of Medicinal Products
Россия
Россия
Olga A. Rachinskaya, Cand. Sci. (Bio.), Leading Expert, Laboratory of Biomedical Cell Products
Petrovskii bulvar 8/2, Moscow, 127051
E. V. Melnikova
Scientific Centre for Expert Evaluation of Medicinal Products
Россия
Россия
Ekaterina V. Melnikova, Cand. Sci. (Bio.), Head of the Laboratory of Biomedical Cell Products
Moscow
V. A. Merkulov
Scientific Centre for Expert Evaluation of Medicinal Products
Россия
Россия
Vadim A. Merkulov, Dr. Sci. (Med.), Professor, Deputy General Director
Moscow
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Review
For citations:
Rachinskaya O.A., Melnikova E.V., Merkulov V.A. Design Features of Nonclinical Studies of In Vivo Gene Therapy Medicinal Products. Part 1: Pharmacological studies. Innovative Medicine of Kuban. 2025;10(4):113-120. (In Russ.) https://doi.org/10.35401/2541-9897-2025-10-4-113-120
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