The results of preclinical trials of the first domestic drug for gene therapy of limb-girdle muscular dystrophy were presented at the summit of drug developers “Sirius.Biotech-2024”.
Alexey Deikin, Director of the Joint Center for Genetic Technologies of BSU, told about the successful preclinical trial of the drug for the treatment of rare diseases at the summit of drug developers in Sirius. The partners of the research were Genotarget LLC (Ivan Yakolev) and Artgen-Biotech PJSC (Artur Isaev, Moscow).
In his report, Alexey Deikin noted that the drug for the treatment of neuromuscular diseases is being created under the grant from the FSTP program for the development of genetic technologies. As the head of the cluster “Development of genetic technologies” of the strategic project “Science of the XXI century” of “Priority-2030” program, he drew attention to the fact that along with the development of gene therapy drugs a Clinic of gene therapy is planned to be created at the university.
“We expect that due to such a successful result the university will become one of the emerging centers of full-cycle development in Russia with access to the use of gene therapy drugs not only, for example, for children with spinal muscular atrophy, but also for adults. The University has increased its competencies and we have created a laboratory for modeling and gene therapy. More than 800 students have been trained in the Molecular Biology program, 10 residents and 20 postgraduates are also being trained in these areas,” – he added.
It should be noted that junior researchers of the Research Institute for Pharmacology of Living Systems – postgraduates of the Medical Institute Evgeny Patrakhanov, Nikita Zhunusov and Vladimir Pokrovsky took part in the summit. The young scientists are directly involved not only in the creation of genotherapeutic drugs, but also in the execution of the project on creation of a line of rabbits producing heat shock protein (Hsp 70) for the needs of the pharmaceutical industry within the Research Center “Innovative Solutions in Agro-industrial Complex” and a number of other developments in the field of genetic technologies. These include work with laboratory animals to create various models of human diseases.
The important role of Belgorod State University in the creation of genetic models of diseases was emphasized in the reports of the representatives of industrial partners of BSU – Alexander Karabelsky, head of the “Gene Therapy” direction of the scientific center of translational medicine of Sirius University, and Ivan Galkin, researcher at Marlin Biotech LLC.
The information was prepared with the support of the grant of the Ministry of Education and Science of Russia under the federal project “Popularization of science and technology” № 075-15-2024-572 from 13.05.2024.
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