Wiskott-Aldrich gene therapy benefits last up to 13 years

The New England Journal of Medicine published long-term results on 24 September 2026 for etuvetidigene autotemcel, the gene therapy for Wiskott-Aldrich syndrome developed at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan. The inherited disorder leaves children with failing immune defences and dangerous bleeding.

The therapy takes a patient’s own blood stem cells and adds a working copy of the WAS gene with a lentiviral vector. The paper, led by Francesca Ferrua with Alessandro Aiuti as senior author, follows 27 patients treated at a median age of 2.6 years, with follow-up between 5.7 and more than 13 years. Survival was 96% at one and five years. Severe infections and moderate-to-severe bleeding fell markedly, and every patient had stopped immunoglobulin replacement by the latest visit. More than 80% attend school or kindergarten, and about half take part in sports, according to IRCCS Ospedale San Raffaele and Fondazione Telethon.

Fondazione Telethon obtained marketing authorization for the therapy, sold as Waskyra, in the European Union and the United States between December 2025 and January 2026.