In late August, Rossiya Segodnya hosted a roundtable discussion on the topic ‘Innovative Genetics: Research, Educational Programs, and Implementation in Production’. The event was held as part of the Scientific Belgorod project, with the support of the Ministry of Science and Higher Education of the Russian Federation.
As Maxim Patrushev, head of the Genomic Center of Kurchatov Institute R&D Center, noted ‘genetics is currently one of the priority areas of research in Russia... It is a cross-cutting industry with applications in numerous vital sectors of our economy and areas of our life. In 2019, the President of the Russian Federation initiated a federal scientific and technical program for the development of genetic technologies... There are four main areas of focus: medicine, biological security, agriculture, and industrial microbiology. The specific tasks to be solved are itemized.
Alexey Deykin, Director of the Joint Center for Genetic Technologies of the Belgorod State National Research University, discussed the ‘genetic square’, which the university has used to unite its divisions and research efforts across four areas: microbiology, plant breeding, animal breeding, and biomedicine. ‘In each of these areas, we have achieved significant results in the four incomplete years,’ he stated. ‘I would like to begin by thanking Genotarget's CEO, Ivan Yakovlev, and Artgen Biotech's Chief Science Officer, Roman Deyev. Their contributions have been instrumental in achieving remarkable outcomes in gene therapy and pharmacology.’
In 2024, Genotarget, a biotech startup within Artgen Biotech’s ecosystem, completed preclinical studies of its novel gene therapy drug, GTDF102, for the treatment of muscular dystrophy-dysferlinopathy (LGMD R2, Miyoshi myopathy). The research was conducted in partnership with the Belgorod State National Research University with grant support from the Russian Ministry of Education and Science.
Roman Deyev, Chief Science Officer of Artgen Biotech, highlighted the importance of developing treatments for genetic orphan diseases in his report. In 2011, Artgen Biotech became the first Russian company that managed to register and obtain authorization to use the gene therapy drug, Neovasculgen, for the treatment of lower limb ischemia. ‘Neovasculgen is currently the only registered gene therapy drug in the Russian Federation. Over eight years ago, Artgen Biotech initiated the development of a gene therapy for dysferlinopathy. In partnership with Genotarget LLC, we created the active substance for the prospective drug. Over the past two years, with the support of the Belgorod State National Research University and the Ministry of Science and Higher Education of the Russian Federation, significant advancements have been made.’
Roman Deyev explained that the construct is a genetic one combined with a viral capsid. ‘But imagine the challenge: to deliver the drug to all the muscle cells... 40 kilograms of muscle tissue. This is a significant challenge. To date, we have addressed it at the transgenic mutant mouse level, which serves as the model for this disease. We have completed the full cycle of preclinical studies, demonstrating that the genetic design is effective and induces changes in muscle tissue that halt disease progression. We have now started to make a dossier in order to move forward.’
‘Artgen Biotech is an ecosystem,’ Roman Deyev noted. ‘We are supporting and developing with a number of scientific groups approximately 30 potential constructs, with the goal of advancing about 10 of them into healthcare practice.’
Ivan Yakovlev, CEO of Genotarget, proceeded to address the subject of gene therapy for neuromuscular diseases, emphasizing the potential of existing solutions and the prospects for developing more effective approaches to treating these diseases. ‘Neuromuscular diseases, such as limb-girdle muscle dystrophies, Duchenne muscular dystrophy, and spinal muscle atrophy, represent a significant challenge for modern medicine and for the state,’ he stated. ‘Gene therapy offers a promising avenue for addressing the underlying genetic defects associated with these diseases. Our experience in developing a drug to treat dysferlinopathy, a form of limb-girdle muscle dystrophy, provides a solid foundation for creating effective solutions for other diseases.’
All of the current treatments for neuromuscular diseases that have reached the market are based on technologies that were the subject of extensive research and development approximately 10-20 years ago. Each of these treatments has its own set of limitations and drawbacks. For example, at the end of July Pfizer terminated a multi-year study on Duchenne muscular dystrophy due to a lack of conclusive data regarding the efficacy of their drug. This further highlights the necessity for a more comprehensive and contemporary methodology in the development of effective treatments for neuromuscular diseases, with a focus on leveraging the latest advancements in gene therapy.
In his presentation, Ivan Yakovlev highlighted the current obstacles to scaling up research and development of new gene therapy methods. ‘For example, the limited number of production sites is a significant challenge. Even preclinical research, let alone clinical research, requires a substantial amount of viral mass, as gene therapy for neuromuscular diseases necessitates relatively high dosages,’ he explained. ‘We will continue research and development to overcome existing barriers and create drugs that can improve the quality of life for patients with neuromuscular diseases.’
Please click on the link to view a video of the roundtable discussion ‘Innovative Genetics: Research, Educational Programs, and Implementation in Production’.