CAMBRIDGE, Mass. — Fondazione Telethon and Orphan Therapeutics Accelerator have signed a memorandum of understanding aimed at establishing a non-profit commercial access model in the U.S. for an ex vivo gene therapy targeting Wiskott-Aldrich syndrome, a rare and life-threatening immunodeficiency.
The agreement follows recent U.S. Food and Drug Administration approval of the therapy, which was developed by Fondazione Telethon, an Italian non-profit with more than three decades of experience advancing research in rare genetic diseases. Orphan Therapeutics Accelerator, a U.S.-based patient-centered non-profit biotech, said the collaboration is designed to address a critical access gap for ultra-rare disease treatments that often fail to attract for-profit commercialization.
Under the memorandum, Orphan Therapies, a non-profit subsidiary of Orphan Therapeutics Accelerator, would become the exclusive commercialization partner for the therapy in the U.S., while Fondazione Telethon would retain the biologics license application. The organizations said the arrangement would represent the first commercialization of a gene therapy through a purely non-profit collaboration and could serve as a template for bringing other advanced therapies to patients with extremely small populations.
“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 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.”
The two organizations said the collaboration combines complementary strengths. Fondazione Telethon brings long-standing experience developing and commercializing a similar product in Europe, while Orphan Therapeutics Accelerator contributes U.S. market expertise, established relationships, and infrastructure designed specifically to ensure access to advanced therapies for very small 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 memorandum outlines plans to finalize agreements covering U.S. distribution and logistics, including the establishment of networks of specialty distributors and qualified treatment centers, as well as patient services and support programs aimed at ensuring access and assistance throughout the treatment process.
“The commercial and operational complexities of launching an advanced therapy in the ultra-rare space are immense,” said Beth White, COO of Orphan Therapeutics Accelerator. “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. We look forward to finalizing the definitive agreement and quickly moving toward making this therapy available.”
