Nonprofits partner to bring therapy for Wiskott-Aldrich syndrome to US
Deal also aims to create access to treatments for ultra-rare diseases like AADC
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Fondazione Telethon and Orphan Therapeutics Accelerator, both nonprofit biotech organizations, have signed a Memorandum of Understanding (MoU) to facilitate commercial access in the U.S. to the first gene therapy approved in the nation for the treatment of Wiskott-Aldrich syndrome.
The gene therapy, Waskyra (etuvetidigene autotemcel), was recently approved by the U.S. Food and Drug Administration for certain adults and children with the ultra-rare, life-threatening immunodeficiency. It was developed by Fondazione Telethon following more than two decades of research.
Beyond supporting access to the therapy for patients with Wiskott-Aldrich syndrome, the MoU aims to establish a sustainable, nonprofit path to market access for other advanced therapies for ultra-rare diseases, such as aromatic l-amino acid decarboxylase (AADC) deficiency. In such disorders, small patient populations often make these therapies unlikely to be commercialized by for-profit companies.
“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,” Craig Martin, CEO of Orphan Therapeutics Accelerator, said in a joint press release.
Fondazione Telethon will retain ownership of biologics license
Under the terms of the MoU, Fondazione Telethon will retain ownership of the biologics license. Orphan Therapies, a nonprofit subsidiary of Orphan Therapeutics Accelerator, will be exclusively responsible for commercialization activities in the U.S., including distribution, engagement with qualified treatment centers, and patient support services.
The agreement establishes what the organizations describe as a “patient access-focused alliance” between two mission-aligned nonprofits, each contributing complementary expertise to address the commercial and operational barriers that frequently prevent ultra-rare disease therapies from reaching patients.
Fondazione Telethon brings experience developed over several decades, including the prior development and commercialization of similar therapies in Europe. Orphan Therapeutics Accelerator contributes a team with deep expertise, established relationships across the U.S. market, and a specialized infrastructure designed specifically to support the commercial availability of advanced therapies for the smallest patient populations.
“The commercial and operational complexities of launching an advanced therapy in the ultra-rare space are immense,” said Beth White, chief operating officer 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.”
If implemented, the collaboration would mark the first commercialization of a gene therapy through a purely nonprofit partnership.
“The anticipated partnership with Orphan Therapeutics Accelerator — a [nonprofit] 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.”
