Odylia Therapeutics Announces New Pipeline Project: A Gene Replacement Therapy for NPHP1 Retinal Dystrophy

ATLANTA, May 5, 2025 /PRNewswire/ -- Odylia Therapeutics, a nonprofit (501(c)3) biotechnology company dedicated to advancing treatments for rare diseases, today announced its latest pipeline project: a unique and innovative co-development partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by mutations in the NPHP1 (nephrocystin-1) gene.

"We are incredibly pleased to make this announcement and to add yet another promising project to our pipeline," Odylia Chief Executive Officer and Chief Scientific Officer Ashley Winslow said. "NPHP1-associated retinal dystrophy is an area where there is a huge opportunity to build on existing science and to develop an effective treatment, with an accelerated path to clinical trials. The biology is promising, we are excited about the partnership model we have developed and are using with the NPHP1 Family Foundation, the team we have assembled is top-notch, and the path forward is clear."

Odylia also announced today that it is working with collaborators at Boston Children's Hospital, in the lab of Dr. Friedhelm Hildebrandt, a renowned expert on NPHP nephrocystin genes and their associated ciliopathies. Dr. Hildebrandt's team will be conducting the natural history, dosing and efficacy studies on two different NPHP1 mouse models – one with a more aggressive retinal phenotype and one with a milder phenotype. The former mouse was used in a successful gene replacement therapy proof of concept in the lab of Dr. Liangzhong Sun at Nanfang Hospital in Guangzhou, China.

NPHP1 gene mutations occur in one in 922,000 live births in the United States, according to the National Institutes of Health, and patients primarily present with nephronophthisis. Of that number, roughly six to ten percent will also develop retinal dystrophy, a blinding disease of the eye with extreme heterogeneity from person to person. The NPHP1 Family Foundation was established to fund and accelerate the development of treatments for this ultra-rare condition.

"As a parent, pretty much the worst thing you can hear is that your child is sick and there is no treatment for what they have," NPHP1 Family Foundation Co-Founder and parent of an afflicted child Caryn Alagno said. "To be able to change that circumstance and to work with a team of researchers with immense knowledge in this space gives us a massive amount of hopefulness for the future."

This innovative co-development collaboration is a reflection of the current drug development landscape, wherein patient groups and organizations supporting treatments for rare diseases look to non-traditional pathways to create treatments. Because rare conditions are believed to be of little to no commercial interest, these patients are overlooked by traditional pharmaceutical companies and left to build treatments themselves. Odylia's non-profit model, combined with the tenacity of parents and patient groups, creates a pathway for research and development that would otherwise not exist.

The NPHP1 project's preclinical work is slated to occur throughout the remainder of this year and the team is targeting an Investigational New Drug (IND) submission and subsequent clinical trial in late 2027.

To learn more about Odylia, the NPHP1 Family Foundation, or Dr. Hildebrandt's Lab – or to support this project by donating – please visit these resources:
www.odylia.org
www.nphp1.org
https://research.childrenshospital.org/

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.

Contact: Odylia Media
[email protected]

SOURCE Odylia Therapeutics