FDA-Approved WAS Gene Therapy Hits Market in Nonprofit Model

Fondazione Telethon and Orphan Therapeutics Accelerator have signed a memorandum of understanding (MoU) outlining a pathway to US commercial access for etuvetidigene autotemcel (brand name Waskyra), an ex-vivo gene therapy targeting Wiskott-Aldrich syndrome (WAS), a rare inherited immunodeficiency (1). The therapy was approved by FDA earlier in December 2025 (2), positioning the agreement as a practical test case for whether nonprofit organizations can address long-standing access and sustainability challenges associated with advanced therapies for extremely small patient populations.

Advanced gene therapies for ultra-rare diseases face a distinct set of technical and economic constraints. These include patient-specific or small-batch manufacturing, extended vein-to-vein timelines, specialized quality control testing, and the need for tightly coordinated logistics between manufacturing facilities and transplant-capable treatment centers (3). When combined with limited commercial volume, these factors often undermine traditional return-on-investment models, even after regulatory approval has been achieved (4).

Why does nonprofit gene therapy commercialization matter for ultra-rare diseases?

Under the memorandum, Fondazione Telethon would retain the biologics license application for etuvetidigene autotemcel, while a nonprofit subsidiary of Orphan Therapeutics Accelerator would serve as the exclusive commercialization partner in the United States. If finalized, the arrangement would represent what the organizations describe as the first instance of a gene therapy being commercialized entirely through a nonprofit collaboration (1).

“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, in a company press release (1). “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.”

From an industry perspective, the model is notable for separating regulatory ownership from commercialization execution while keeping both functions within mission-driven organizations. The framework emphasizes cost transparency, operational efficiency, and long-term supply continuity rather than revenue maximization (1). For developers of advanced therapies, this approach may offer an alternative pathway for assets that have cleared clinical and regulatory hurdles but lack a viable commercial sponsor (5).

How could this agreement affect gene therapy manufacturing and distribution?

The memorandum outlines plans for US distribution and logistics, including the establishment of networks of specialty distributors and qualified treatment centers. For ex-vivo gene therapies, this setup requires the synchronization of leukapheresis, vector transduction, release testing, cryogenic transport, and patient conditioning at clinical sites. Each step introduces operational risk and cost that must be managed at low scale.

According to the organizations, centralizing these activities within a nonprofit commercialization entity is intended to reduce duplication, standardize processes, and maintain compliance with current good manufacturing practice requirements without relying on high-volume throughput. The agreement also includes patient services and support programs aimed at coordinating referrals, facilitating site readiness, and supporting reimbursement navigation, all of which are critical to uptake in ultra-rare indications (1).

“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, in the release (1). “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.”

What does this model signal for the future of advanced therapy access?

WAS results from mutations in the WAS gene and presents in early childhood with immunodeficiency, thrombocytopenia, eczema, and elevated risk of infection, autoimmunity, and malignancy. While hematopoietic stem cell transplantation remains a potentially curative option, donor availability, conditioning-related toxicity, and graft-versus-host disease limit its applicability. An approved gene therapy in this indication introduces a new treatment modality but also intensifies the need for reliable, long-term access mechanisms (1).

“The commercial and operational complexities of launching an advanced therapy in the ultra-rare space are immense. 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, in the release (1). “We look forward to finalizing the definitive agreement and quickly moving toward making this therapy available.”

If successful, this nonprofit commercialization framework could inform future strategies for gene and cell therapies that fall outside conventional market dynamics. For the biopharmaceutical industry, the collaboration underscores a growing recognition that alternative access and manufacturing models may be required to translate scientific advances into durable patient access in ultra-rare diseases (1,4).

References

1. Fondazione Telethon. 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. Press Release. Dec. 15, 2025.
2. FDA. FDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome. Press Release. Dec. 9, 2025.
3. Lee N. K.; Chang, J. W. Manufacturing Cell and Gene Therapies: Challenges in Clinical Translation. Ann. Lab. Med. 2024, 44 (4), 314–323. DOI: 10.3343/alm.2023.0382
4. Fox, T. A.; Booth, C. Improving Access to Gene Therapy for Rare Diseases. Dis. Models Mech. 2024, 17 (6), dmm050623. DOI: 10.1242/dmm.050623
5. Jarosławski, S.; Toumi, M. Non-Profit Drug Research and Development: The Case Study of Genethon. J Mark Access Health Policy 2018, 7 (1), 1545514. DOI: 10.1080/20016689.2018.1545514