Genotarget, a biotech startup that is part of the Artgen Biotech ecosystem, has completed preclinical studies of GTDF102, a new gene therapy drug for the muscular dystrophy dysferlinopathy (LGMD type R2, Miyoshi myopathy).

Research continues. The data obtained must be confirmed and extended, manufacturing must be refined and a documentation package prepared. After that, Genotarget will submit a dossier to the Russian Ministry of Health to obtain permission for clinical trials of the drug in patients.

The studies were conducted in partnership with Belgorod State National Research University with grant support from the Russian Ministry of Science and Higher Education. The Avtsyn Research Institute of Human Morphology of the Petrovsky National Research Centre of Surgery and the Genetico Center also took part in the studies.

In studies in mice with a model of the disease, synthesis of normal dysferlin protein was achieved. Interim results have been published in the International Journal of Molecular Sciences. These data were obtained in animals: the safety and efficacy of the drug in humans can only be assessed in clinical trials.