Telethon lands in the US: first time a non-profit organisation gets the OK to distribute a therapy

The Telethon Foundation lands in the United States with a new challenge that confirms the excellence of research made in Italy. For the first time in the world, a non-profit organisation has received authorisation to distribute a therapy resulting from its own research and development in the United States. The US Food and Drug Administration (FDA) has approved an ex vivo gene therapy (Waskyra) for patients suffering from Wiskott-Aldrich syndrome (Was), a rare and severe genetic immunodeficiency. The Fda's approval follows the positive opinion of the European Medicines Agency's (EMA) Chmp for the same therapy, which came a few weeks ago, and confirms the effectiveness of a model that has become unique worldwide.

The Telethon Foundation is in fact the first non-profit organisation to have successfully completed the development of a gene therapy, taking it from laboratory research to regulatory approval in the United States today. This is an important milestone that follows on from the path started in 2023, when the Foundation took over responsibility for the production and distribution in the European Union of another gene therapy (for the treatment of Ada-Scid, the disease of so-called 'bubble babies'). This decision kick-started a non-profit model that provides patients with access to advanced therapies in cases where the pharmaceutical industry and the market withdraw due to an inability to see an adequate return on their investment. The new treatment for WAS was developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan and represents not only an important scientific and clinical milestone but also a new hope for patients born with this disease.

"We know that today we face a new demanding challenge," said Telethon Foundation President Luca di Montezemolo. "We do so with courage and confidence, aware that it is the only way to remain faithful to our mission, which is not to leave people born with rare genetic diseases alone. This is an extraordinary achievement, not only for the Telethon Foundation and for the research made in Italy, which has been confirmed as excellent, but for the global community of patients. An important, almost historic change, but thanks to the generosity of our donors we are sure that we will not be alone". "The approval of this gene therapy is a decisive step forward and a concrete response to the needs of patients," explained Alessandro Aiuti, Deputy Director of SR-Tiget, Head of Paediatric Immunohaematology at IRCCS Ospedale San Raffaele and Full Professor of Paediatrics at the University Vita-Salute San Raffaele. "Seeing years of scientific research and commitment translated into real therapeutic opportunities for people gives a deep meaning to our work." The clinical trial phase was conducted at IRCCS Ospedale San Raffaele, a centre of excellence in gene therapy for WAS and other diseases. The FDA approval represents further recognition of Fondazione Telethon and consolidates its role as a reference in the international arena, both in research on rare genetic diseases and in the field of advanced therapies.