Odylia Rare Disease Day Event Highlights Challenges in R&D for Orphan Drugs
Odylia Therapeutics event brings together leaders across biotech, pharma, venture capital, patient advocacy, and research sectors to address the financial and structural barriers that impede rare disease drug development.
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Odylia Therapeutics, a nonprofit biotech organization focused on accelerating drug development for rare diseases, recently hosted a Rare Disease Day event aimed at promoting new funding models and collaborative strategies to bring orphan drugs to market. According to the company, the event, held in in Boston, Mass., brought together leaders from the biotech, pharmaceutical, venture capital, patient advocacy, and research sectors.1
“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,” said Ashley Winslow, PhD, CEO, chief scientific officer, Odylia, in a press release. “Every dollar in rare disease research is precious. We need to ensure these funds translate into real therapeutic options. 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 key focus of the event was the increasing influence of patient groups in early-stage medication development. Odylia stated that it is working to provide these groups with tools and partnerships to help advance therapies from discovery to clinical trials.1
“Having been deeply involved in the rare disease community, I understand the complexities of drug development in this space,” said Jodi Cook, PhD, CEO, president, Skylark Bio, in the press release. “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.”
Currently, Odylia is developing a treatment for RPGRIP1-associated retinal dystrophy using a novel Anc80 vector technology developed by its co-founder, Luk Vandenberghe, PhD. The therapy is in late-stage preclinical development, with preparations underway for an investigational new drug (IND) submission. To support the program, Odylia is seeking approximately $3.5 million in funding.1,2
The company is also advancing a gene therapy for vision loss caused by mutations in the USH1C gene, associated with Usher Syndrome Type 1C. This program is being developed in collaboration with the Usher 2020 Foundation and the FAUN Foundation, alongside scientific experts across the United States and Europe.3
Through its Brydge Solutions partnerships, Odylia aims to reduce inefficiencies across the early discovery-to-clinic pathway, according to the company. The initiative provides strategic guidance and operational support to patient groups, researchers, and biotech firms, helping them maintain momentum while retaining control over their therapeutic programs.4
“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,” said Lain Anderson, managing director, L.E.K. Consulting, in the press release. “Odylia’s efforts to expedite this process for rare diseases where unmet needs are significant are truly commendable.”
References
1. Odylia Therapeutics drives commitment to rare disease drug development through Rare Disease Day event. Globenewswire. April 10, 2025. Accessed April 14, 2025. https://www.globenewswire.com/news-release/2025/04/10/3059743/0/en/Odylia-Therapeutics-drives-commitment-to-rare-disease-drug-development-through-Rare-Disease-Day-event.html
2. RPGRIP1 Gene Therapy Program (OT-004). Odylia. Accessed April 14, 2025. https://odylia.org/research-and-development/rpgrip1/
3. Treating Vision Loss in Usher Syndrome with an USH1C Gene Therapy. Odylia. Accessed April 14, 2025. https://odylia.org/research-and-development/ush1c-gene-therapy/
4. Brydge Solutions. Odylia. Accessed April 14, 2025. https://odylia.org/brydge-solutions/
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