Fondazione Telethon ETS

Telethon Foundation is an Italian non-profit organization that has been supporting bio-medical research on rare and genetic diseases for more than 30 years. The vision of the Telethon Foundation is to translate research results into therapies available to patients. Telethon, besides being an Italian organization, is committed to treat any patient in the world affected by a rare genetic disease and believes that fighting genetic diseases is a worldwide challenge. Over the years Telethon Foundation has invested more than 620 euro million funding over 2800 research projects involving more than 1600 scientists.
Every year Telethon Foundation launch a call for projects in the field of RD research open to all Italian not for profit research centers. Moreover Telethon runs 2 research institutes, the Telethon Institute for Genetic Medicine (TIGEM) and the San Raffaele Telethon Institute for Gene Therapy (SR‐Tiget). In 2023 FTELE became the Marketing Authroization Holder of Strimvelis.

Regulation on orphan medicinal products - Indirect contact with the Commission through IRDiRC
Pharma strategy - indirect contact through the Alliance for Regenerative Medicine
Personalized medicine - indirect contact with the Commission through ICPerMED
implementing acts of HTA regulation

http://www.telethon.it

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FT does not have an office in Brussels. However its staff is often involved in informal consultation with the EC on Rare Disease research policies and Orphan drug development. FT now has 1 person dedicated to Public Affairs whose main focus is on national policies; therefore the lobbying activities at EU level remain below 50% of his effort. In addition FT has a Regulatory Experts engaged in some EMA consultation and regulatory focused intiatives (10%).

Data not provided by Register Secretariat due to GDPR

Data not provided by Register Secretariat due to GDPR

Non-governmental organisations, platforms and networks and similar