Fondazione Telethon ETS

We choose to back research into rare diseases. It’s what makes us unique.

A collective heritage

Every year, the general public, enterprises, the media and institutions join forces with Telethon to invest time and resources in this enormous collective enterprise.

A track record of excellence

• 790 Milions invested
• 1.958 Researchers
• 3.186 Funded projects

Our mission

Fondazione Telethon is an Italian non-profit organization committed to advancing research on rare genetic diseases. For over 35 years, we have supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.

Often neglected by large public or industrial investment schemes, and the poor relation when it comes to research and drug treatments - rare genetic conditions are not statistically relevant when considered individually.

What is our response to this silent emergency?

We aim to: 

• develop innovative treatments and therapies, aiming to translate research into real-world health benefits. 
• fund cutting-edge research on genetic diseases, with a focus on rare and underserved conditions. 
• help shape regulatory frameworks that ensure high standards and sustainability in the development of gene and cell therapies. 
• raise awareness about the burden of genetic diseases and the importance of sustained scientific and societal commitment.

Our impact

Fondazione Telethon transforms research excellence into measurable outcomes for people affected by rare genetic diseases.

Through a model that integrates philanthropy, science, and innovation, we have built a sustainable ecosystem that advances discovery, accelerates clinical translation, and strengthens Italy’s leadership in the life sciences.

At the core of our mission are two leading research institutes: 

• Tigem – Telethon Institute of Genetics and Medicine (Pozzuoli)
• SR-Tiget – San Raffaele Telethon Institute for Gene Therapy (Milan) 

Together, they employ over 500 scientists working across gene therapy, molecular biology, and translational medicine — bridging the gap between laboratory innovation and patient care. 

From Discovery to Access 

Fondazione Telethon is a national leader in technology transfer, fostering collaborations with the biotech and pharmaceutical sectors to translate scientific discoveries into accessible therapies. 

Through licensing agreements, industrial partnerships, and venture creation, we ensure that research results are transformed into sustainable therapeutic solutions and reinvested into new innovation pathways. 

Looking Ahead 

Building on a solid legacy of excellence and accountability, Fondazione Telethon continues to invest in: 

• Innovative research platforms for next-generation therapies
• Advanced clinical programs to reach more patients
• Strategic alliances that expand global impact 

Our goal remains clear and constant: to transform the promise of science into cures — ensuring that no patient is left behind.