The research program has two main goals: increasing the technological know-how necessary to design and deliver RNA-based and gene therapy medicinal products and identifying promising candidate drugs/genes in five major areas of human diseases (genetic diseases, cancer, metabolic/cardiovascular diseases, neurodegenerative disorders and inflammatory/infectious diseases).
The past few decades have seen a dramatic shift in the view of how human disorders can be treated. Molecularly personalized treatments, developed through a global rethinking of pharmacology, have become an ambitious goal for greatly increasing the efficacy of cures. The need for highly specific new drugs stems from the astounding advancements in the understanding of the molecular and cellular events responsible for human diseases. Indeed, large molecular diversity is not the exclusive hallmark of rare, inherited disorders but underlies the pathogenesis of the most common human diseases. The case of cancer is exemplary: it has become clear that neoplastic diseases with a similar clinical and phenotypical appearance, may differ from each other for the set of oncogenes and tumor suppressor genes involved through different pathogenic mutations, and innovative approaches targeting specifically the altered protein, or even the genetic mutations, show high efficacy. Thus, the development of drugs specific for a wide range of targets has become not only the principle of treatment of rare disorders, often neglected in the priorities of pharmaceutical companies, but also the basis of a global new approach to cure, appropriately defined “precision medicine”. For this task, it is necessary to explore therapeutic strategies that go beyond the laborious identification of small chemical molecules fitting critical regulatory domains of enzymes, transporters and channels. Rather, a change in paradigm is needed, with the development of a class of drugs that share common synthetic and delivery platforms and can act, in principle, on any class of proteins with unprecedented accuracy. Clearly, the extraordinary power and flexibility of nucleic acids have made these molecules the ideal tools for this task, with a virtually limitless breadth of applications. The impact of RNA- based vaccines in the containment of the COVID-19 pandemic has provided direct, impressive evidence of how RNA-based drugs for specific targets can be rapidly and effectively developed.