EU gene therapy filing marks new milestone for charity

Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, has submitted a Marketing Authorization Application (MAA) for the gene therapy etuvetidigene autotemcel to the European Medicines Agency (EMA).

The application is for the treatment of patients with Wiskott-Aldrich syndrome (WAS), a rare genetic disease of the immune system.

After positioning itself as the first charity in the world to assume responsibility for the production and distribution of a drug - the gene therapy for the treatment of adenosine deaminase (ADA-SCID) - this submission to the EMA represents a new milestone that confirms the foundation's commitment to making therapies discovered through the work of its researchers available to patients.