Design Features of Nonclinical Studies for In Vivo Gene Therapy Medicinal Products. Part 2: Pharmacokinetic and Toxicological Studies
https://doi.org/10.35401/2541-9897-2026-11-1-148-156
Abstract
The article focuses on the specific design features of nonclinical studies (NCS) of in vivo gene therapy medicinal products 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. This work highlights the key aspects of conducting pharmacokinetic and toxicological studies, identified through the analysis of expert reports from global gene therapy medicinal products manufacturers. These aspects include dose selection and route of administration; identification of specific studies applicable to all in vivo gene therapy medicinal products and the justification of study lists for products using different types of vectors; as well as duration of studies and the selection of appropriate models to demonstrate product safety and to detect potential adverse effects associated with their use.
Keywords
gene therapy products,
nonclinical studies,
pharmacokinetic studies,
toxicological studies,
vector,
transgene
Supporting agencies: The study was carried out within the framework of FSBI “SCEEMP” No. № 056-00001-25-01 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
Russian Federation
Russian Federation
Olga A. Rachinskaya - Cand. Sci. (Biol.), Leading Expert, Laboratory of Biomedical Cell Products, Scientific Centre for Expert Evaluation of Medicinal Products.
Petrovskii bulvar 8/2, Moscow, 127051
E. V. Melnikova
Scientific Centre for Expert Evaluation of Medicinal Products
Russian Federation
Russian Federation
Ekaterina V. Melnikova - Cand. Sci. (Biol.), Head of the Laboratory of Biomedical Cell Products, Scientific Centre for Expert Evaluation of Medicinal Products.
Moscow
V. A. Merkulov
Scientific Centre for Expert Evaluation of Medicinal Products
Russian Federation
Russian Federation
Vadim A. Merkulov - Dr. Sci. (Med.), Professor, Deputy General Director, Scientific Centre for Expert Evaluation of Medicinal Products.
Moscow
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For citations:
Rachinskaya O.A., Melnikova E.V., Merkulov V.A. Design Features of Nonclinical Studies for In Vivo Gene Therapy Medicinal Products. Part 2: Pharmacokinetic and Toxicological Studies. Innovative Medicine of Kuban. 2026;11(1):148-156. (In Russ.) https://doi.org/10.35401/2541-9897-2026-11-1-148-156
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