Telethon-Developed LVV Gene Therapy for Wiskott-Aldrich Syndrome Receives Positive CHMP Opinion

ROME, Nov. 14, 2025 — Fondazione Telethon has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion, recommending marketing authorization in the European Union for Waskyra™ (etuvetidigene autotemcel).

LVV-Powered Therapy Offers New Hope

Waskyra™ is an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency. The therapy was developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan and represents a major scientific and clinical achievement.

The gene therapy consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector (LVV) encoding the WAS gene. This LVV technology is central to the therapy’s function. Once corrected by the LVV, the stem cells are reinfused into the patient, aiming to reduce the frequency of severe bleeding events and serious infections.

“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.

LVV Success Marks Non-Profit Milestone

Fondazione Telethon is the first non-profit organization to have successfully led the full pathway from laboratory research to a positive regulatory opinion, collaborating with industry partners to bring this LVV gene therapy from discovery to patients.

Dr. Alessandro Aiuti, Deputy director of clinical research at SR-Tiget, noted, “The true meaning of science lies in the impact it has on people’s lives.” The therapy will be made available at IRCCS Ospedale San Raffaele, the center of excellence where the LVV clinical trial phase was conducted.

WAS is a genetic blood disorder that primarily affects males, causing immunodeficiency and low platelet counts. Current curative options, like hematopoietic stem cell transplantation, are limited by donor availability and risk. For eligible patients without a compatible family donor, this LVV gene therapy represents a crucial potential therapeutic option. The BLA for the same LVV gene therapy for WAS remains under review by the U.S. Food and Drug Administration (FDA).