Posted by Michael Wonder on 15 Nov 2025
CHMP positive opinion for Waskyra, a gene therapy for the treatment of Wiskott-Aldrich syndrome
14 November 2025 - Fondazione Telethon announces the positive opinion issued by the CHMP of the EMA, recommending marketing authorisation in the European Union for Waskyra, an ex vivo gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening primary immunodeficiency.
Fondazione Telethon is the first non profit organisation 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.
