Odylia Therapeutics (Odylia) a nonprofit biotechnology company dedicated to expediting drug development for rare diseases, hosted a Rare Disease Day event to foster conversations about new funding models and collaborative strategies to bring treatments to market for rare diseases. The event, which took place in Boston, Massachusetts, underscores Odylia’s commitment to overcoming the financial and structural barriers that often stall rare disease drug development.

The gathering brought together leading biotech and pharmaceutical executives, venture capitalists, patient advocacy groups, and researchers to explore solutions for the funding and commercialization challenges that rare disease therapies face.

“We are thrilled to have a diverse group of industry leaders come together to share ideas about tackling the challenges of drug development for rare diseases. Every dollar in rare disease research is precious. We need to ensure these funds translate into real therapeutic options,” said Ashley Winslow, Ph.D., CEO and chief scientific officer at Odylia. “By thinking differently—through strategic nonprofit models, innovative funding approaches, and patient-driven development—we can accelerate progress for therapies that otherwise wouldn’t see the light of day.”

A major theme of the event highlighted the increasing role of patient groups in driving early-stage drug development. With more families and foundations directly funding research, Odylia’s work is both driving the development of the next generation of drugs for rare diseases and providing patient groups. Odylia provides the tools and partnerships necessary to enable patient groups to move their drug development programs from discovery to clinical trials.

Participants, including influential leaders like Lain Anderson, a managing director at L.E.K. Consulting, and Jodi Cook, Ph.D., CEO and president of Skylark Bio, underscored the importance of Odylia’s mission to remove barriers and accelerate the path from research to clinical application.

“At L.E.K. Consulting, I have advised numerous biopharma companies on their drug development journey and have seen firsthand how difficult this process can be, especially for smaller biotechs with limited resources,” Anderson said. “Odylia’s efforts to expedite this process for rare diseases where unmet needs are significant are truly commendable.”

Cook added, “Having been deeply involved in the rare disease community, I understand the complexities of drug development in this space. Skylark Bio’s work on novel therapeutics for rare hearing disorders is a testament to how challenging this path can be. Odylia’s mission to support others with their innovative approach and development efforts will undoubtedly lead to more therapeutics reaching patients who desperately need them.”