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We are proud to announce that AAVantgarde, a spin-off of our Telethon Institute of Genetics and Medicine (TIGEM), co-founded by its Director Alberto Auricchio and Fondazione Telethon has won the first edition of the Nicolais Award for the Valorization of Research in Life Sciences, awarded by Network PerfeTTO. This recognition, established in memory of Prof. Luigi Nicolais, celebrates excellence in transferring research to the business world. And this is exactly where a fundamental piece of our work lies: the birth and growth of realities like AAVantgarde testify to the crucial role of the Business Development Office of Fondazione Telethon. Accepting the award in Milan, presented by Prof. Alessandro Sannino and Prof. GIULIO POMPILIO was Annamaria Merico, Head of Business Development at Fondazione Telethon. Our sincere thanks to Network PerfeTTO for this recognition, which drives us to continue building bridges between cutting-edge research and industrial development. Congratulations to the whole team! 👏

Fondazione Telethon has built a unique nonprofit model to take pioneering research from the laboratory to people living with rare genetic diseases. A model that turns scientific progress into real therapies, brings Italian biotech innovation to the world, and contributes to advancing the life sciences. Bringing a gene therapy developed through our research to the United States and Europe is a powerful example of how far this model can go. Fondazione Telethon. Research that goes all the way.

A historic milestone for rare disease research and an innovative model to make therapies accessible to those who need them most. Together with Orphan Therapeutics Accelerator, we are proud to announce that UCSF Benioff Children's Hospitals is the first Qualified Treatment Center in the United States authorized to administer WASKYRA® (etuvetidigene autotemcel), the gene therapy developed by our researchers for the treatment of Wiskott-Aldrich Syndrome (WAS). Approved by the FDA in December 2025, WASKYRA® represents an extraordinary dual breakthrough: 🔬 It is the first gene therapy available for this ultra-rare and life-threatening immunodeficiency. 🤝 It is the first advanced therapy medicinal product (ATMP) to be commercialized entirely through a non-profit collaboration. As our CEO, Ilaria Villa, emphasizes: "This milestone reflects the continuous and successful evolution of the Fondazione Telethon model. After decades of pioneering research, we recognized the need for new and collaborative solutions to overcome the market barriers that prevent urgently needed therapies from reaching people with rare genetic diseases." Excellent science does not stop at the laboratory door. It fulfills its ultimate purpose only when it reaches the patient. Here's the complete article: https://lnkd.in/enq2Gb67

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The future of advanced therapies, told by those shaping it: in this video, a student from the Science and Technology for Advanced Therapies (STAT) PhD — run by IUSS - Scuola Universitaria Superiore Pavia in collaboration with Fondazione Telethon — shares their firsthand experience of the program. As Raffaella Di Micco notes, one year after STAT’s launch the assessment is highly positive: the program delivers an innovation- and internationalization‑oriented curriculum that guides researchers from basic science to clinical application. This testimony is a concrete example of that translational vision. Applications are now open for the National PhD Programme in Science and Technology for Advanced Therapies (STAT) – 42nd Cycle.

We are pleased to celebrate another meaningful step toward clinical translation, built on the value of research and the strength of collaboration. 🧬 In partnership with San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Genespire has shown durable preclinical efficacy for its liver-directed gene therapy approach in methylmalonic acidemia (MMA), with a single systemic administration leading to sustained therapeutic benefit in mouse models. The study, published in the Journal of Hepatology, also showed gene transfer efficiency exceeding 80% of the liver, further supporting the promise of this approach. This is a concrete example of how the research developed within Fondazione Telethon’s institutes can be transformed into innovation with real translational potential. Genespire, born as a spin-out of SR‑Tiget and founded with the contribution of Fondazione Telethon together with Ospedale San Raffaele, Alessio Cantore and #LuigiNaldini, perfectly embodies the path we aim to promote turning scientific excellence into tangible opportunities for patients. A result that reflects our mission and strengthens our commitment to advancing research toward the clinic, where it can make a real difference for people living with rare genetic diseases. Learn more: https://lnkd.in/deHiy7xT