FTELE - Fondazione Telethon

Fondazione Telethon is a major Italian charity founded in 1990 and supported through fundraising. Its mission is to advance biomedical research towards the cure of rare genetic disease and its vision is to convert the results of excellent research into therapies available for all patients. Fondazione Telethon investment model comprises competitive quality-certified peer-reviewed Calls to fund extramural research conducted in Italian research institutes, ad hoc initiatives to sustain strategic special projects, such as the Telethon Undiagnosed Disease Program, and two peer-reviewed institutes to foster intramural research, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and Telethon Institute of Genetics and Medicine (TIGEM). This model is supported by specific competencies in technology transfer, clinical development, advanced therapies manufacturing, regulatory affairs, and by partnerships with the industry and venture capitals, which allowed Fondazione Telethon to develop six cell and gene therapies for rare genetic diseases.

Role within JOIN4ATMP

Fondazione Telethon is the leader of the Rare Non-Cancer Diseases pillar that will encompass all Work Packages, ensuring that rare genetic diseases pecularities are considered in all the activities of the project. Fondazione Telethono will also contribute expertise in translational project management & regulatory affairs, clinical trials, business development and technology transfer to the JOIN4ATMP initiative, offering real use-cases of hurdles and solutions adopted in the development of Advanced Therapy Medicinal Products for rare genetic diseases. Specifically, it will co-lead activities i) to explore new incentives and financing approaches for academic and SME-produced ATMPs clinical development, ii) to design a platform model to accelerate GMP manufacture of ATMPs based on the same technology and mechanism of action, and iii) to define new frameworks for value assessment, pricing and reimbursement schemes for ATMPs.

Main contact(s)

Stefano Benvenuti

Head of Public Affairs, FTELE WP4 reference person

Sean Russell

PrimeRA Pharma Partners, Managing Partner, FTELE WP3 reference person

Martina Cesani

Head of Quality Assurance, FTELE WP2 reference person

Mario Barilani

Research Program Manager, FTELE project manager