A hope twelve thousand kilometres long
The story of Amari, who came from the small Caribbean Island of Trinidad to Milan to receive gene therapy for his rare genetic disorder of the immune system.
Our numbers
Since 1990 we fund research on rare genetic conditions- 741 Millions of euros invested
- 1.916 Researchers
- 3.118 Projects funded
What we do
15.12.25
Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US
Orphan Therapeutics Accelerator and Fondazione Telethon today announced the signing of a Memorandum of Understanding to facilitate US commercial access to an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome.
10.12.25
Fondazione Telethon Announces FDA approval of Waskyra™ (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome
The therapy represents a major scientific and clinical achievement, offering new hope for patients affected by this condition.
05.12.25
Engineering peripheral immune tolerance: Tregs and beyond
Tregs and beyond: discover how SR-TIGET scientists are engineering peripheral immune tolerance for tomorrow’s precision therapies.
