Core Points - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the market authorization of Waskyra™, a gene therapy for Wiskott-Aldrich Syndrome (WAS) [1][12][13] - The therapy was developed over decades at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, representing a significant scientific and clinical advancement [3][4] - Waskyra™ offers new hope for patients suffering from WAS, a rare genetic blood disorder that leads to immunodeficiency and low platelet counts [7][9] Company Overview - Fondazione Telethon is the first non-profit organization to successfully navigate the entire process from laboratory research to regulatory approval, collaborating with industrial partners when necessary [2][4] - The organization has been dedicated to advancing research on rare genetic diseases for over 35 years, supporting impactful scientific work aimed at developing innovative treatments [11] Therapy Details - Waskyra™ involves a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been modified with a lentiviral vector carrying the WAS gene [9][10] - The therapy has shown to reduce the frequency of severe and moderate bleeding events and serious infections in patients with WAS compared to the pre-treatment period [10] - The therapy will be available to patients at the IRCCS Ospedale San Raffaele, a recognized center of excellence for gene therapy [5]