Odylia Therapeutics drives commitment to rare disease drug development through Rare Disease Day event

Industry leaders gather to discuss innovative solutions for accelerating the development of therapeutics

/EIN News/ -- BOSTON, MA, April 10, 2025 (GLOBE NEWSWIRE) -- 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.”

Transforming Drug Development with Purpose

Odylia’s unique mission-based approach seeks to accelerate the development of treatments for rare diseases by removing traditional roadblocks in the drug development process. Through its Brydge Solutions initiative, Odylia provides hands-on expertise and preferred service providers to patient groups, academic researchers and biotechs to navigate the complex path to clinical trials for rare diseases.

The organization is currently advancing a therapy for RPGRIP1-associated retinal dystrophy, a rare inherited retinal disorder, with two additional gene therapies in development, and more than 15 partnerships through Brydge Solutions.

Odylia is reshaping the traditional biotech model by prioritizing therapeutic impact over market size. The company’s nonprofit framework ensures that promising therapies aren’t abandoned due to shifting corporate priorities or financial viability concerns.

As the biotech industry grapples with economic pressures and capital constraints, Odylia’s model offers a viable path forward for developing high-impact therapies that might otherwise be deprioritized. The company is actively seeking collaborations with biotech firms, investment partners, and patient groups to continue expanding its portfolio.

About Odylia Therapeutics

Odylia is a nonprofit biotech focused on bringing life-altering and life-saving treatments to those with rare diseases. With expertise in both preclinical and clinical drug development for rare diseases, Odylia seeks to change how drugs are developed by focusing on the science and patient needs rather than the commercial potential. Odylia partners with patient communities, researchers, clinicians, industry and financial donors to bring promising therapeutics to clinical trials. In addition to three gene therapies in development, Odylia partners with rare disease patient groups, early-stage gene therapy companies and academic researchers to further rare disease drug development efforts through Odylia’s Brydge Solutions initiative. Patient groups, companies and researchers interested in learning more about Odylia Therapeutics’ services can email info@odylia.org and follow Odylia on LinkedIn (Odylia Therapeutics), Instagram (@OdyliaTx), and YouTube (@OdyliaTx). For additional information, visit odylia.org.

Contact Information

Odylia Therapeutics
Ashley Winslow, Ph.D., CEO, CSO
awinslow@odylia.org