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

Rafael, a patient with ADA-SCID who received gene therapy.

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

+3000 research projects funded across Italy
+1900 scientists supported through national and international collaborations
+3000 scientific publications in leading international journals
+160 patients treated with Telethon-developed gene therapies
2 approved gene therapies + one nearing FDA and EMA approval 
5 spin-off companies launched from our research

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

Together, they employ over 500 scientists working across gene therapymolecular 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. 

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