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
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.
14.11.25
CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Announced the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome.
