Genetic diseases, including muscular dystrophies are rarely curable directly. Muscular dystrophy can restrict the flexibility and mobility of joints. Up to now the different therapies of muscular dystrophies relies on classical treatment, assistive devices that can improve quality and sometimes length of life of patients with muscular dystrophy. The R&D result is based on eradicating of the initial step of muscle dystrophy by inhibition of the proteolytic cleavage of bulk muscle proteins actin and myosin, which in turn provides substrates for the main proteolytic pathways, calpain, lysosomal and the ubiquitin-26S proteasome.
This R&D achievement therefore very promising. The present state of R&D activity requires an initial investment or subsidy to complete the development of the product. One version of the product can be a nutraceutical supplement. This product can reach the market in the fourth year and with an average pharmaceutical-level product plant, the mass production can be solved.
There can be other solutions too for implementing the R&D result. In case of a registered medicine the procedure of the approval and licensing (i.e.: FDA approval)takes at least five years. So in this valorization plan we did not take into consideration this option. Another possibility is to contract with a pharmaceutical company for licensing agreement. In this case the investment for finalizing the development is a bit less – according to the calculation of the project owner it can be € 460 thousand) and after the third year the IPR can be sold for the company.