Telethon experience and its efforts towards the registration of new advanced therapies

Abstract

Telethon, since its establishment in 1990, raised a total of 382 million euros to finance external (research project, fellowships, research services and core facilities) and internal programs (TIGEM Institute, HSR-TIGET Institute, DTI Institute). Overall, Telethon research projects have addressed more than 400 genetic disorders and complex diseases (Fig. 1). A new Telethon Strategic Plan has been drafted for 2006–2010, and states the willingness to become an internationally recognised charity that progresses excellent laboratory research into clinical results to be made available to patients, by increasing the flexibility of funding and promoting relationships. One of Telethon major commitments is to transfer the results in preclinical research into clinical practice, by promoting translational research. This strategy includes the support of the clinical development for the most promising pre-clinical projects (Telethon Technology Transfer Office, Farmindustria Initiative) and the undertaking of regulatory activities (Fig. 2).