Wiskott-Aldrich Syndrome

Overview

Rare immunodeficiency causing recurrent infections and severe bleedings, treated with hematopoietic stem cell gene therapy; approved in both the US and EU after Fondazione Telethon completed the full regulatory path as the first charity to do so. Wiskott-Aldrich syndrome patients suffer from recurrent infections and severe bleedings; after gene therapy, patients progressively stopped having these events, and the product was approved in both the US and Europe. Fondazione Telethon drove the Wiskott-Aldrich syndrome gene therapy to full marketing registration in both the US and EMA over the past 2 years before 2026, making it the first charity to complete this entire commercialization pathway. (Segment 2 (05:00-10:00))