We work everyday for:

Every life counts

Mostly overlooked by large public and industrial investments, orphans of research and drugs: rare genetic diseases taken individually are not statistically significant. But can we say the same for somebody’s life?

Why do we study genetic diseases?

Research can give real hope

By funding research we can provide answers and therapies to people who otherwise, in many cases, would not have any hope.

Learn more about our mission.

Latest news

Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union
Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches. Coordinated by Luigi Naldini, director of the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET)...
Beta Thalassemia: clinical trial provides encouraging evidence for efficacy of gene therapy
Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only. This is the conclusion from the first clinical study of gene therapy in bo...
A new ingredient to potentiate gene therapy in stem cells
Gene therapy has reached significant clinical milestones but its application remains difficult for a number of disorders and cell types for which basic knowledge to develop more efficient protocols and strategies is still lacking. Now, a new study published in Cell Stem Cell* by the research team...
Restore Health – Therapies that cure
On 18 May, the European Commission (EC) announced that the FET Flagship RESTORE, which was initiated by the Berlin-Brandenburg Center for Regenerative Therapies (BCRT), has been selected to enter the next phase of the FET Flagship competition. A successful FET Flagship will eventually receive ar...