TELETHON INITIATIVE FOR THE DISCOVERY OF DRUG TARGETS (TIDID)

There are about 6,000 known human genetic disorders. While most of these are rare, they collectively represent about 10% of the overall number of pathologies affecting mankind. Because of their individual rarity, genetic diseases have historically been neglected by the profit-driven structure of the systems for drug development. The Telethon Initiative for the Discovery of Drug targets was aimed at, and has now achieved, the setting up a complete non-profit-driven programme of drug development for the treatment of genetic diseases. The search for pharmacological approaches to genetic diseases is justified because: a) a large body of knowledge has been accumulating over the past decades on the molecular defects underlying genetic diseases; and b) powerful drug-discovery technologies are now available to the pharmaceutical industry to exploit this knowledge towards the development of drugs. The TIDID initiative is sustained by the integration of two complementary scientific partner Institutions, the Department of Cell Biology and Oncology, at the Consorzio Mario Negri Sud Research Institute, and SienaBiotech, and it is aimed at exploiting our basic knowledge gained on molecular mechanisms of genetic diseases, for the development of pharmacological treatments for these diseases. The ongoing efforts of TIDID, an initiative that was started up in 2006, are towards establishing a small- to medium-scale model for a process that has now shown the required success to begin to extend it in the future. In these first two years, we have set up appropriate procedures to establish the feasibility of the approach, applying it to a limited number of diseases, including Lowe syndrome and cystic fibrosis. The positive results obtained so far lead to the idea that the model can now be gradually extended to other tractable genetic diseases