Core Points - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Waskyra™, an ex-vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency [1][5] - Fondazione Telethon is the first non-profit organization to successfully navigate the entire process from laboratory research to regulatory approval, collaborating with industry partners to bring gene therapies from discovery to patients [2][10] - Waskyra™ represents a significant scientific and clinical achievement, providing new hope for patients affected by WAS [3][4] Company Information - Fondazione Telethon has been supporting impactful scientific research for over 35 years, focusing on developing innovative treatment methods for rare and complex genetic diseases [10] - The therapy was developed at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, which is recognized as a center of excellence for gene therapy [3][4] Therapy Details - Waskyra™ involves a one-time administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene [8] - The therapy has shown to reduce the frequency of severe and moderate bleeding as well as severe infections in patients with WAS compared to the period before treatment [9] - The current treatment options for WAS are limited, with hematopoietic stem cell transplantation being the only potentially curative option, which is not always available due to donor compatibility issues [7]