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.

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).

The MoU establishes the foundation for a distribution and access agreement under which Orphan Therapies, a non-profit subsidiary of Orphan Therapeutics Accelerator (OTXL), would become the exclusive commercialization partner for the therapy in the US, with Fondazione Telethon holding the Biologics License Application (BLA). This would mark the first commercialization 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 wouldn’t otherwise be commercialized by for-profit companies.

“The anticipated partnership with Orphan Therapeutics Accelerator — a non-profit organization closely aligned with us in mission and values — strengthens our shared commitment to demonstrating that alternative commercialization models are possible, even for ultra-rare diseases that do not attract traditional industry interest,” said Ilaria Villa, CEO of Fondazione Telethon.

This patient access-focused alliance represents a novel approach involving two unique, mission-aligned non-profits – each offering distinct strengths relevant to the model: FT brings the experience and capabilities, established over several decades, of having developed and commercialized one other similar product in Europe, and OTXL offers a team with deep, relevant experience and extensive relationships in the US market, and a specialized infrastructure built specifically to ensure commercial availability of advanced therapies for the smallest patient populations.

“This MoU is a demonstration of our shared commitment to ensuring that breakthrough therapies for devastating rare conditions reach patients who desperately need them, regardless of commercial scale,” said Craig Martin, CEO of Orphan Therapeutics Accelerator. “Fondazione Telethon’s dedication to making this therapy available is remarkable. We are honored to collaborate with them and look forward to the impact we will have together for Wiskott-Aldrich syndrome patients.”

The MoU outlines the parties' intent to finalize arrangements for:

"This MoU signals our readiness to deploy the Orphan Therapies model, a streamlined, mission-focused approach that allows us to manage intricate supply chain, distribution, treatment and other requirements in a highly efficient way,” said Beth White, COO of Orphan Therapeutics Accelerator. “We look forward to finalizing the definitive agreement and quickly moving toward making this therapy available.”

The Orphan Therapeutics Accelerator (OTXL) is a patient-centered non-profit biotech focused on obtaining and reinitiating development of promising “shelved” clinical-stage treatments for ultra-rare conditions and providing a scalable and sustainable commercialization path to get these treatments to patients. OTXL leverages its non-profit status, success-based agreements and incentives to obtain rights to shelved programs and complete development at deferred or lower cost via a network of affiliated CDMOs, CROs and other partners. When a program is approved and generates revenue, net proceeds are used to repay and reward contributing partners, companies, and institutions, with a portion returned to the Orphan Therapeutics Accelerator to fund additional programs. Our team is deeply familiar with challenges faced in developing and commercializing treatments for rare conditions and committed to overcoming them. For more information, please visit www.orphantxl.com.

Orphan Therapies is a wholly owned subsidiary of the Orphan Therapeutics Accelerator (OTXL), a non-profit focused on advancing shelved ultra-rare disease treatments through clinical trials. Orphan Therapies provides commercial access to approved treatments for very rare conditions where high costs and small patient populations make commercialization by traditional biotech companies unviable. Its streamlined, non-profit model enables operation with a primary focus on sustaining and scaling access to treatments, while generating revenues that support OTXL’s ongoing mission to deliver more treatments to patients. For more information, please visit www.orphantherapies.com.

Wiskott-Aldrich syndrome is a rare genetic blood disorder that causes immunodeficiency and low platelet count, resulting from mutations in the WAS gene. The disease manifests from early childhood with recurrent and persistent infections, bleeding episodes and eczema, and is associated with an increased risk of developing autoimmune diseases and lymphomas.  It affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. Current treatment options include supportive therapies aimed at managing and preventing clinical manifestations. The only potentially curative option is hematopoietic stem cell transplantation, for which a compatible donor is not always available, and which is not without risks.