Odylia Therapeutics Receives Translational Research Award from Foundation Fighting Blindness to Accelerate Development of Gene Therapy for RPGRIP1

Atlanta, GA, May 20, 2024 —  Odylia Therapeutics, a nonprofit biotechnology company, received a Translation Research Award for $1.5 million from the Foundation Fighting Blindness to accelerate the development of a novel gene therapy (OT-004) to treat vision loss caused by mutations in the RPGRIP1 gene.

Mutations in the RPGRIP1 gene cause rapid decline in function of the photoreceptor cells of the retina, resulting in a rare type of vision loss that can begin as early as infancy. Patients are usually diagnosed with one of three forms of retinal dystrophy: Leber congenital amaurosis (LCA) 6, cone rod dystrophy 13 (CORD13), or juvenile retinitis pigmentosa.OT-004, is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer of RPGRIP1 to treat vision loss in patients lacking a functional copy of the RPGRIP1 gene. OT-004 was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA in 2021. 

By becoming an awardee of this competitive funding, Odylia is recognized by the Foundation for the significant milestones they have reached with the RPGRIP1 Gene Therapy Program. During the execution of the Translational Research Award, Odylia will advance OT-004 through critical steps in the late-stage preclinical development for this vision loss gene therapy.

“We are thrilled to receive this award and excited to work with the Foundation more closely to advance this gene therapy toward clinical trials,” commented Ashley Winslow, CEO and CSO of Odylia Therapeutics. “This funding provides significant support for the program at a time when investment in rare disease genetic therapies is declining. We are thankful to the Foundation and to the patient community for continuing to partner with us in our efforts. In addition to advancing OT-004, we hope to leverage momentum from this award to secure the additional support needed to progress this gene therapy to clinical trials.”

“We are pleased to support Odylia through our translational research program to move its emerging RPGRIP1 gene therapy toward a human study,” says Chad Jackson, PhD, Senior Director of translational research at the Foundation Fighting Blindness. “They are meeting a critical unmet need by advancing research toward saving and restoring vision for people with challenging, progressive retinal diseases caused by RPGRIP1 mutations.”

OT-004 has been in development for several years, supported financially and through research and development activities by various partners. Since Odylia’s inception the nonprofit has championed the RPGRIP1 Gene Therapy Program ensuring it continues to move forward and the program is not shelved.

For media inquiries, please contact:

Odylia Media

Ashley Windslow, CEO & CSO

About Odylia Therapeutics
Odylia is a nonprofit biotech focused on bringing life-altering and lifesaving treatments to those with rare diseases. We have expertise in both preclinical and clinical drug development for rare diseases. Our vision is to change how drugs are developed for rare diseases by focusing on the science and patient needs, rather than the commercial potential. We partner with patient communities, researchers, clinicians, industry, and financial donors to bring promising therapeutics to clinical trials. In addition to our gene therapy development pipeline, we partner with rare disease patient groups, early-stage gene therapy companies, and academic researchers to further their rare disease drug development efforts through our Brydge Solutions program. Patient groups, companies, and researchers interested in learning more about Odylia Therapeutics’ services contact us at info@odylia.org and follow us on LinkedIn (Odylia Therapeutics), Instagram (@OdyliaTx), and YouTube (@OdyliaTx), and Facebook (Odylia Therapeutics). For additional information, visit https://odylia.org.


About OT-004
OT-004 is an investigational AAV gene therapy that utilizes the Anc80 AAV vector for efficient gene transfer. Anc80 is an ancestral adeno-associated virus discovered in 2015 in the lab of Luk H. Vandenberghe, at Massachusetts Eye and Ear. Dr. Vandenberghe’s laboratory discovered Anc80 through their efforts to identify safer, more effective viral vector-based delivery systems. Anc80 is predicted to be the ancestor of the broadly utilized AAV1, AAV2, AAV8, and AAV9, and can effectively transduce the retina, central nervous system, muscle, liver, and cells of the inner ear to name a few.

About the Foundation Fighting Blindness
Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $915 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.

About the Translational Research Program
The Translational Research Program awards accelerate the movement of preclinical research toward an Investigational New Drug filing and into clinical trials to provide a robust and diverse pipeline of potential therapies to fight inherited retinal diseases and dry age-related macular degeneration.