#Italy #Rome #WAS #Waskyra™ #Telethon

Fondazione Telethon Announces Positive Opinion for Waskyra™ Gene Therapy

Date: November 14, 2025
Location: Rome, Italy

In a groundbreaking development, Fondazione Telethon has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). This endorsement is for Waskyra™, an innovative ex vivo gene therapy designed specifically for treating Wiskott-Aldrich Syndrome (WAS). This rare yet severe primary immunodeficiency condition predominantly affects males, bringing forth significant challenges, including recurrent infections and bleeding complications.

Wiskott-Aldrich Syndrome, characterized by mutations in the WAS gene, affects around 1 in 250,000 live male births. Symptoms typically begin in early childhood, leading to recurrent infections, low platelet counts, eczema, and an elevated risk of autoimmune diseases and lymphomas. Traditional treatment has mainly focused on managing symptoms, with the only curative option being hematopoietic stem cell transplantation—which can be both risky and dependent on donor availability.

Waskyra™ represents a major shift in the treatment landscape for WAS, embodying years of dedicated research and collaboration between Fondazione Telethon and industry partners. Developed at the renowned San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, the therapy involves administering autologous CD34+ hematopoietic stem cells that have been modified to express the WAS gene through a lentiviral vector. Patients receive these genetically corrected cells following a chemotherapy regimen intended to prepare their bone marrow, significantly enhancing their ability to address the disease effectively.

Dr. Ilaria Villa, General Director of Fondazione Telethon, expressed pride in this achievement.