Design Features of Nonclinical Studies for In Vivo Gene Therapy Medicinal Products. Part 2: Pharmacokinetic and Toxicological Studies
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.
Fields of Study & Subjects
Virus-based gene therapy research
CAR-T cell therapy research
RNA Interference and Gene Delivery
Publication details
- Journal / source
- Innovative medicine of Kuban
- Publisher
- Scientific Research Institute - Ochapovsky Regional Clinical Hospital No 1
- Volume
- 11
- Issue
- 1
- Pages
- 148–156
- Publication date
- 1 March 2026
- Language
- RU