Part 25 of our Bridging the Gap Series (Summary of the webinar session held in December 2025)
Editor’s note: Since this webinar, Waskyra has also received approval from the U.S. Food and Drug Administration (FDA), expanding access beyond Europe.
One of the hardest challenges in cell & gene therapy (CGT) is determining how programs for ultra-rare diseases move forward when there’s no clear commercial path. Many promising efforts stop well before reaching the clinic.
Waskyra is one example that didn’t. Developed by Fondazione Telethon, the gene therapy recently received a positive opinion from the European Medicines Agency (EMA) and has since been approved by the U.S. Food and Drug Administration (FDA), marking a major milestone for nonprofit-led innovation.
That experience became the focus of December’s Bridging the Gap webinar. The discussion centered less on the EMA decision itself and more on what it took to keep the program moving over time, from early research through review. The milestone was framed not as an isolated success, but as proof that nonprofit and mission-driven organizations can carry advanced therapies all the way to regulatory approval when commercial incentives fall short. Olga Bukatova, Associate Director of Business Development for CGT at Azenta Life Sciences, opened the session with Dr. Marsela Qesari, a CMC Manager at Fondazione Telethon, joined by Albert Ribickas, Assistant Director of the Cell Therapy Facility at Moffitt Cancer Center, Dr. Patrick Hainley, Chief and Director of the Cellular Therapy Program at Children’s National, Adam Clark, Director Operations at the Emily Whitehead Foundation, and Tom Whitehead, co-founder of the Emily Whitehead Foundation.
Fondazione Telethon – A Mission-Driven Engine for Rare Disease Innovation
To understand this outcome, it’s helpful to look at the organization behind it. Fondazione Telethon began more than 35 years ago as a national fundraising effort and has since become a central force in rare disease research in Italy. Crucially, its origins lie in large-scale Telethon fundraising campaigns broadcast on Italian national television, which mobilized millions of individual donors and embedded public participation at the core of its mission – a point underscored by panelist Patrick Hanley. Today, the foundation funds investigators across the country, operates two research institutes, and remains closely involved in programs that might otherwise lack a clear development path.
This structure is especially meaningful for conditions with very small patient populations, such as Wiskott-Aldrich syndrome, offering hope where traditional development pathways have often stalled, as Dr. Qesari noted, “For diseases with 10 to 15 new patients a year in all of Europe, there is simply no commercial incentive. Mission-driven organizations have to be the ones to carry these therapies forward.”
For patients and families, this distinction is critical. Regulatory approval does not only represent access – it represents continuity, consistency, and confidence that every patient will receive the same quality of therapy as the first.
Telethon has built the internal capabilities needed to support that mission, including expertise in CMC, regulatory affairs, quality, and supply chain, enabling continuity from discovery through approval – and, critically, beyond approval into manufacturing, distribution, and lifecycle management.
Why Nonprofit Development Matters – Advancing Therapies Beyond Commercial Limits
A consistent theme from the discussion was that many rare disease programs begin with promising science but struggle to move beyond early research. Telethon’s experience showed what can happen when an organization builds the structure, partnerships, and long-term commitment needed to stay with a therapy as it develops.
The panelists also noted that progress in ultra-rare disease often relies on organizations willing to advance therapies that may never draw commercial interest. Telethon’s work illustrates how a mission-driven group can help a program continue when the usual development paths aren’t available.
Building a Solid Foundation: CMC as a Compass for Success
CMC work played a central role in Waskyra’s progress. Telethon’s teams adopted a development mindset early and refined their approach as the program matured. They focused on:
- Documenting scientific rationale from the beginning to support regulatory review
- Choosing materials and processes suitable under GMP conditions
- Identifying which steps would be difficult to change downstream
Quality by design (QbD) helped the team align on risk and process considerations. Comparability was treated as an ongoing responsibility, supporting smoother transitions from small-scale work to GMP manufacturing, site changes, and process updates across the product lifecycle. A longstanding partnership with AGC Biologics added additional stability through scale-up.
Scaling with Purpose: From Bench to GMP
The panel spoke candidly about the challenges of scaling a therapy in a nonprofit setting. Moving from research-scale experiments to GMP manufacturing requires planning and realistic expectations. Manual steps are often part of early development, and automation may only be feasible later when resources allow.
Dr. Qesari described progress as a series of deliberate choices that keep the program aligned with its long-term needs, even when not every element can be optimized immediately.
Regulatory Considerations: From Complexity to Confidence in Approval
Telethon pursued submissions with both the EMA and FDA. Managing two regulatory pathways brought complexity, but it also strengthened the program. Feedback from both agencies helped clarify expectations early, identify gaps well before licensure, and reinforce that regulators are partners in bringing safe, effective therapies to patients.
Panelists also noted that distributed manufacturing models place greater emphasis on reproducible analytical methods and consistent reference materials.
Throughout the discussion, panelists emphasized that CMC is not simply a regulatory requirement. It is the mechanism that ensures reproducibility, consistency, and fairness – so that each patient receives the same therapy, regardless of when or where it is manufactured.
For other nonprofit organizations, academic centers, and mission-driven teams, Waskyra’s path offers a clear signal: sustained progress depends less on any single breakthrough than on early alignment, disciplined CMC strategy, and trusted partnerships. When those elements are in place, even programs without a traditional commercial path can advance with rigor and confidence.
Collaboration, Commitment, and the Path Forward
Tom Whitehead closed the session with a simple reminder: “When you go out with a mindset of collaboration, more patients benefit.”
Waskyra is more than a regulatory milestone. It reflects what’s possible when teams stay focused on the work at hand, make deliberate early choices, and build the relationships needed to move a therapy from discovery through delivery. For many in the CGT community, Telethon’s experience offers a practical path for programs that may never attract commercial interest but still matter deeply to the patients they’re meant to serve.
Key Takeaways
- Nonprofit, mission-driven organizations can carry gene therapies to full regulatory approval when traditional commercial incentives fall short, as demonstrated by Waskyra’s path to EMA and FDA approval.
- Early, disciplined CMC strategy is essential for long-term success, enabling smoother transitions from research to GMP manufacturing, regulatory review, and lifecycle management.
- Regulatory agencies function as partners, not just gatekeepers, with dual EMA and FDA engagement strengthening development plans and clarifying expectations well ahead of licensure.
- Comparability and quality by design are ongoing commitments, especially critical for distributed manufacturing models and programs that evolve over time.
- Collaboration across foundations, academic centers, clinicians, and industry partners accelerates progress, ensuring that therapies for ultra-rare diseases reach patients with consistency, confidence, and fairness.
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