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.
Our impact in numbers
- 35+ years advancing research on rare genetic diseases
- 2 approved gene therapies + one nearing FDA and EMA approval
- 90+ patent families filed and managed through our technology transfer activities
- 5 spin-off companies launched from our research, fostering Italy’s biotech innovation ecosystem
At the core of our mission are two leading research institutes:
Together, they employ over 500 scientists working across gene therapy, molecular biology, and translational medicine — bridging the gap between laboratory innovation and patient care.
Our network extends through strategic partnerships with:
- Ospedale San Raffaele (Milan)
- Ospedale Universitario Federico II (Naples)
- Università degli Studi della Campania “Luigi Vanvitelli” (Caserta)
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.