Fondazione Telethon announces CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)

ROME, Nov. 14, 2025 /PRNewswire/ -- Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency.

Fondazione Telethon is the first non-profit organization to have successfully led the full pathway from laboratory research to regulatory approval, collaborating with industry partners when available to bring gene therapies from discovery to patients.

Developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, the therapy represents a major scientific and clinical achievement, offering new hope for patients affected by this condition.

"This milestone demonstrates that academic research, when guided by a strong sense of responsibility towards patients and conducted to the highest industrial standards, can truly change the natural history of rare diseases," said Ilaria Villa, General Director of Fondazione Telethon. "We are proud that the work started in our laboratories is now reaching European patients, reaffirming the value of a research model that bridges science and care."

"Making therapies truly available is essential to offering families a real chance at treatment," added Dr. Alessandro Aiuti, Deputy director clinical research at SR-Tiget, Chief of Pediatric Immunohematology at IRCCS Ospedale San Raffaele and Full Professor of Pediatrics at Università Vita-Salute San Raffaele. "The true meaning of science lies in the impact it has on people's lives."

The therapy will be made available to patients at IRCCS Ospedale San Raffaele, a recognized center of excellence in gene therapy for this and other diseases, where the clinical trial phase was conducted.

The BLA for the same gene therapy for WAS remains under review by the U.S. Food and Drug Administration (FDA).

Fondazione Telethon will continue to collaborate with regulatory authorities to make therapies available to all eligible patients.

About Wiskott-Aldrich syndrome (WAS) 

Wiskott-Aldrich syndrome is a rare genetic blood disorder that causes immunodeficiency and low platelet count, resulting from mutations in the WAS gene. The disease manifests from early childhood with recurrent and persistent infections, bleeding episodes, eczema, and an increased risk of developing autoimmune diseases and lymphomas.

It affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. Current treatment options include supportive therapies aimed at managing and preventing clinical manifestations. The only potentially curative option is hematopoietic stem cell transplantation, for which a compatible donor is not always available, and which is not without risks.

About Waskyra™ (etuvetidigene autotemcel) gene therapy for Wiskott-Aldrich syndrome

Waskyra consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene.

Once corrected, the stem cells are reinfused into the patient, who undergoes chemotherapy beforehand to prepare the bone marrow to receive them. It has been shown that Waskyra reduces the frequency of severe and moderate bleeding events and serious infections in patients with WAS compared with the period prior to treatment. In cases where transplantation from a compatible family donor is not possible, gene therapy represents a potential therapeutic option for eligible patients, whose safety and efficacy have been evaluated.

About Fondazione Telethon

Fondazione Telethon is an Italian non-profit biomedical organization committed to advancing research on rare and complex genetic diseases. For over 35 years, it has supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.

Logo: https://mma.prnewswire.com/media/2823541/Fondazione_Telethon_Logo.jpg