Fondazione Telethon wins EU nod for Waskyra gene therapy

European Commission has granted marketing authorization to Fondazione Telethon’s Waskyra, an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome, a rare and severe genetic immunodeficiency.

The EC’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. The therapy was also approved by the U.S. FDA in December 2025.

Fondazione Telethon is the first nonprofit organization to successfully complete the development of a gene therapy, taking it from laboratory research to regulatory approval in both the U.S. and the EU.

Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

The therapy consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene. By restoring functional WAS protein expression in affected cells, Waskyra addresses the underlying cause of the disease.