Gene Therapy and Oncolytic Viruses – Russian Scientists' 2025 Achievements

Photo Source: Kurchatov Institute

Representatives of scientific and medical centers presented their results at a meeting of the Council for Implementation of the Federal Scientific and Technical Program for Genetic Technologies Development. Under this program, five genomic research centers based at federal scientific institutions received 1.5 billion rubles in 2025 for their projects.

Parkinson's Disease Therapy and Orphan Diseases

The Center for Genetic Reprogramming and Gene Therapy, established at the Federal Center for Brain and Neurotechnologies (FMBA), successfully synthesized patent-clean serotypes of adeno-associated viruses (AAV) as genes and produced them as viral particles for the first time.

AAV vectors are the foundation of modern gene therapy. They underpin drugs like Zolgensma for treating spinal muscular atrophy and Luxturna for hereditary forms of blindness. Obtaining patent-clean serotypes opens possibilities for creating domestic gene therapy drugs, including treatment for the orphan disease spinal muscular atrophy.

Additionally, the center developed a drug for Parkinson's disease therapy, which is planned to enter preclinical trials.

Oncolytic Viruses Against Cancer

The Center for High-Precision Genetic Technologies for Medicine, bringing together the Engelhardt Institute of Molecular Biology, the Institute of Gene Biology RAS, Pirogov Russian National Research Medical University, and the FSCC of Physical-Chemical Medicine (FMBA), obtained an echovirus-1 strain with enhanced oncolytic activity.

The new strain can selectively damage melanoma and breast cancer cells while demonstrating an improved safety profile. Oncolytic viruses represent a promising avenue for targeted therapy, allowing reduced toxicity in cancer treatment. Marus Media covered cancer vaccines in more detail in a dedicated review.

Personalized Medicine

The Center for Predictive Genetics, Pharmacogenetics and Personalized Therapy at the Petrovsky Russian Research Center of Surgery developed pharmacogenetic diagnostic panels for selecting individualized treatment for patients with cardiovascular and oncological diseases, mental disorders, pain syndromes, and for treating various pediatric conditions.

All panels are specifically adapted for Russia's population, distinguishing them from foreign counterparts. Researchers also identified the candidate gene TRPM4, recognized as one of the key genes associated with cardiac conduction system diseases.

Biosafety and Infection Control

The Center for Biological Safety and Technological Independence, uniting the State Research Center for Applied Microbiology and Biotechnology (Rospotrebnadzor), the Vector Center, and the Central Research Institute of Epidemiology, studied the effectiveness of capsular polysaccharide depolymerases – a promising class of antimicrobial agents against superbugs – in treating sepsis.

The center also developed a test system capable of simultaneously detecting 27 respiratory viral pathogens from a single sample within 24 hours. Platform solutions for rapid detection of other pathogens were also created. The center's staff numbers 155 people, 84 of whom are young scientists.

Russia's Federal Program for Genetic Technologies Development was approved in 2019 and will run at least until 2030. The program establishes special national genomic centers – consortia of scientific and educational organizations working to implement breakthrough genetic technologies into clinical and industrial practice, including healthcare.

Each center has industrial partners intending to apply the technology in practice. In medical development, most partners are domestic pharmaceutical companies.

Source: Government of the Russian Federation