Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US

Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts

CAMBRIDGE, Mass. and ROME, Dec. 15, 2025 /PRNewswire/ -- Orphan Therapeutics Accelerator (OTXL), a patient-centered non-profit biotech focused on bringing promising clinical-stage treatments for ultra-rare conditions to patients, and Fondazione Telethon (FT), an Italian non-profit with a 30-year+ legacy of advancing research on rare and complex genetic diseases, today announced the signing of a Memorandum of Understanding (MoU) to facilitate US commercial access to an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome, which was developed by Fondazione Telethon and has just received approval for US marketing by the US Food and Drug Administration (FDA).