In a landmark decision on December 10, 2025, the FDA approved WASKYRA (etuvetidigene autotemcel), marking a transformative milestone in the treatment of Wiskott-Aldrich syndrome—a rare, life-threatening genetic blood disorder. This approval represents not only a significant advancement in gene therapy but also a historic achievement for the non-profit sector, as Fondazione Telethon became the first non-profit organization to successfully shepherd an ex vivo gene therapy from laboratory research through the complete regulatory pathway to FDA approval.
Wiskott-Aldrich syndrome is a rare genetic disorder caused by mutations in the WAS gene, which encodes a crucial pro...
