Non-profit biotech company Orphan Therapeutics Accelerator (OTXL) announced on Monday that it has signed a Memorandum of Understanding (MoU) with Fondazione Telethon, an Italian non-profit biomedical organisation, to facilitate US commercial access to an ex vivo gene therapy for the treatment of Wiskott-Aldrich syndrome.

The therapy, developed by Fondazione Telethon, has just received US Food and Drug Administration (FDA) approval for marketing in the United States.

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 commercialisation partner for the therapy in the US, with Fondazione Telethon holding the Biologics License Application (BLA). This would mark the first commercialisation of a gene therapy via a purely non-profit collaboration, with the added goal of establishing a viable path to market access for other advanced therapies for very small groups of patients that would not otherwise be commercialised by for-profit companies.

Ilaria Villa, Fondazione Telethon CEO, said: "The anticipated partnership with Orphan Therapeutics Accelerator -- a non-profit organisation closely aligned with us in mission and values -- strengthens our shared commitment to demonstrating that alternative commercialisation models are possible, even for ultra-rare diseases that do not attract traditional industry interest. This collaboration is expected to help bring to the United States a therapy developed in our SR-Tiget laboratories, the result of more than 20 years of Italian research."