Non-profit deal to bring US commercial access to gene therapy

Italian non-profit rare and complex genetic diseases specialist Fondazione Telethon (FT) and Orphan Therapeutics Accelerator (OTXL), a non-profit biotech focused on bringing promising clinical-stage treatments for ultra-rare conditions to patients, have announced the signing of a Memorandum of Understanding (MoU).

This agreement facilitates 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 from the US Food and Drug Administration (FDA). 

The MoU establishes the foundation for a distribution and access agreement under which Orphan Therapies, a non-profit subsidiary of OTXL, would become the exclusive commercialization partner for the therapy in the USA, with Fondazione Telethon holding the Biologics License Application (BLA).