Telethon Foundation arrives in the United States with a new challenge that confirms the excellence of Made in Italy research. The American FDA (Food and Drug Administration) has in fact approved Waskyra, an ex vivo gene therapy intended for patients suffering from Wiskott-Aldrich syndrome (WAS), a rare and serious genetic immunodeficiency. The approval by the FDA follows the positive opinion of the CHMP of the European Medicines Agency (EMA) for the same therapy, which arrived a few weeks ago, and confirms the effectiveness of a model that has become unique in the world. The clinical trial phase was conducted at the IRCCS San Raffaele Hospital, a center of excellence in gene therapy for WAS and other diseases.
The success of the research
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Telethon Foundation is in fact the first non-profit organization to have successfully completed the development of a gene therapy, taking it from laboratory research to, today, regulatory approval in the United States. It is an important milestone that follows the path started in 2023, when the Foundation assumed responsibility for the production and distribution in the European Union of another gene therapy (for the treatment of ADA-SCID, the disease of the so-called “bubble children”). This decision gave rise to a non-profit model that guarantees patients access to advanced therapies in those cases in which the pharmaceutical industry and the market withdraw due to the impossibility of seeing their investment adequately remunerated. The new treatment for WAS was developed thanks to 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 pathology.
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Concrete response to patients’ needs
“We know that we are facing a new demanding challenge today” said the President of the Telethon Foundation Luca di Montezemolo. “We do it 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. It is an extraordinary achievement, not only for the Telethon Foundation and for the Made in Italy research which is 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 left alone.” “The approval of this gene therapy is a decisive step forward and a concrete response to patients’ needs” explained Alessandro Aiuti, Deputy Director of SR-Tiget, head of Pediatric Immunohematology at the IRCCS San Raffaele Hospital and full professor of Pediatrics at the Vita-Salute San Raffaele University. “Seeing years of scientific research and commitment translate into real therapeutic opportunities for people gives profound meaning to our work.”
