On May 11–13, 2023, the first Sirius.Biotech summit of drug developers was held at the Sirius Federal Territory. A distinctive feature of the event was that, over three days, representatives of leading biotech and pharmaceutical developers, manufacturers and scientists from fundamental research institutes of the Russian Academy of Sciences met for discussions at the forum venues. Two sessions of the program were devoted to the development and to preclinical and clinical studies of viral gene therapies for patients with orphan diseases. Among the eight selected mini-lectures was a report by the team of the University’s Department of Pathological Anatomy.

The department’s work, carried out jointly with a number of commercial and research organizations in Russia, is a preclinical study of the safety and efficacy of an original Russian drug developed on the basis of knowledge about the pathomorphogenesis of the disease and designed on a technological platform that uses adeno-associated viruses to correct one type of myopathy. The research group’s work is funded by the Ministry of Science and Higher Education of the Russian Federation (agreement No. 075-15-2021-1346).

For the University, it is important that, alongside experienced researchers, young lecturers of the Department of Pathological Anatomy, residents and students take part in the work. This creates a solid foundation for introducing high-quality morphological research not only into everyday practice but also into the way a pathologist thinks.