Serpin inhibitors for the treatment of prion and prion like diseases

To date, despite numerous active efforts, there are no drugs available for the cure of the neurodegenerative diseases collectively referred as prion and prion-like disorders. Symptomatic treatment is the only available option, including the administration to patients of anti-psychotics, such as quetiapine and clonazepam, to treat myoclonus and of selective serotonin re-uptake inhibitors (SSRIs) to treat depression. Indeed, through this invention, the technology transfer office of a research center based in the North of Italy and internationally renowned, is presenting a new class of small molecules. These small molecules are acting as SERPINA3 inhibitors, which are able to reduce PrPSc accumulation in prion-infected cell lines, thereby representing a new therapeutic treatment against prion and prion-like diseases. The development of specific inhibitors of SERPINA3 with an activity in the nano-molar range is expected. The characterization of structural models of the binding of the small molecules with the protein should allow the identification of novel inhibitors with improved potency. The research center is looking for licence agreements or joint ventures with industry (pharma companies) or financial agreements with investors interested in developing new drugs.