• Use of small molecules to rescue folding defective proteins

    The use of CFTR (cystic fibrosis transmembrane conductance regulator) correctors for the treatment of rare and devastating genetic muscular diseases, namely sarcoglycanopathies and Brody myopathy, is the object of this invention. Sarcoglycanopathies and Brody myopathy are rare inherited striated muscle[...]

  • Small molecule inhibitors of OPA1 GTPase activity with anti-cancer properties

    Cancer is a complex disease resulting from the interplay between the tumor cell and its microenvironment, featuring impaired apoptosis and activation of quiescent blood vessels to induce neovascularization to support metastasis. Two of the yet unmet needs in anticancer therapy[...]

  • Bicyclic peptides for targeted therapy against cancer

    Since inhibitors based on small molecules and macromolecules are not selective and potent enough, new compounds, including bicyclic peptides, have been developed in recent years. Bicyclic peptides are new generation therapeutic molecules which exhibit properties typical of monoclonal antibodies (high[...]

  • A compound to mitigate the cytokine storm

    The body’s inflammatory response to fight infections and in tissue repair processes is crucial for normal functioning of the body. Yet, if not controlled, it can cause severe injury to the human body, as occurs during cytokine storm, a potentially[...]

  • siRNAEEC – Novel treatment to mitigate the Ectrodactyly-Ectodermal dysplasia- Clefting (EEC) associated blindness

    The Ectrodactyly-Ectodermal dysplasia-Clefting (EEC) syndrome is a rare genetic disorder due to mutations in the p63 gene and characterized by limb defects, orofacial clefting, ectodermal dysplasia, and ocular surface defects that lead to the loss of vision. Currently no specific[...]

  • New anti-tumoral approach: peptides targeting the hexokinase 2 protein

    Current antineoplastic treatments are not sufficient to cure cancer, in part due to lack efficacy and/or selectivity. Further, they often display significant side effects calling for new therapeutic approaches. The invention consists in the design, realization and administration of peptides[...]

  • A novel treatment for diabetic ulcers

    Diabetic chronic ulcers, a common complication of diabetes, are significant cause of morbidity and mortality in the Western world. Despite the considerable medical need, currently no specific and efficacious treatment for diabetic -and other chronic- ulcers exist. This is in[...]

  • A treatment for fibrotic disease

    Fibrosing diseases are chronic pathologies, with a different genesis, characterized by various types of cellular or extracellular infiltrates determining a progressive destruction of the parenchyma, which is replaced by fibrous tissue, causing progressive damage to organ anatomy. Fibrosing diseases are[...]

  • Therapeutic factors for the treatment of PolyQ diseases

    The polyglutamine (polyQ) diseases are a group of neurodegenerative disorders caused by expanded cytosine-adenine-guanine (CAG) repeats encoding a long polyQ tract in the respective proteins. To date, at least nine polyQ disorders have been described, see bellow for a list.[...]