Critical Path Institute Grants $249,719 to Odylia Therapeutics to Propel Gene Therapy for USH1C-Related Vision Loss

In an ambitious effort to combat a rare genetic disorder known as Usher Syndrome Type 1C (USH1C), the Critical Path Institute (C-Path) has awarded a $249,719 research grant to Odylia Therapeutics, a nonprofit biotechnology organization headquartered in Atlanta. The initiative, spearheaded by Ashley Winslow, Ph.D., CEO and Chief Scientific Officer of Odylia, is supported through C-Path’s Bridging Research and Innovation in Drug Development Grants (BRIDGe) program, a pivotal component of its Translational Therapeutics Accelerator. This funding marks a significant milestone in the pursuit of novel treatments for USH1C-associated vision loss, an ailment that represents an urgent unmet medical need.

USH1C is an autosomal recessive disorder manifesting early with bilateral congenital deafness, balance disturbances stemming from vestibular dysfunction, and notably, vision degeneration that progresses into retinitis pigmentosa during adolescence. Despite advances in auditory management through cochlear implants, no therapeutic options currently exist to maintain or restore vision impaired by retinal cell deterioration in these patients. Odylia Therapeutics aims to fill this critical void by developing a gene therapy specifically targeting the causative mutation in the USH1C gene, intending to halt or reverse retinal degeneration and preserve sight.

The therapy utilizes an adeno-associated virus (AAV) delivery vector engineered to transport a functional copy of the human USH1C gene directly into retinal cells. This approach capitalizes on the safety and efficacy profile of AAVs, widely studied as gene therapy vehicles for ocular diseases due to their low immunogenicity and ability to mediate long-term expression in post-mitotic retinal neurons. Administered via intravitreal or subretinal injection, this vector is designed to restore usherin protein function, crucial for retinal maintenance and cellular function, thus intervening at the genetic root of vision loss in USH1C patients.

Prior proof-of-concept studies funded by the Usher 2020 Foundation have demonstrated effective transduction of target retinal cells with the therapeutic vector. These preclinical experiments confirmed the expression of human USH1C transcript and usherin protein in retinal tissue, providing compelling evidence of the gene therapy’s potential to address the underlying pathology. Encouraged by these findings, the current C-Path grant supports critical next steps, including scalable Good Manufacturing Practice (GMP) production of the AAV vector to ensure batch reproducibility, purity, and potency necessary for clinical application.

Toxicology and biodistribution studies are a focal point of this funded phase, as these assessments are indispensable to evaluate the safety profile of the gene therapy candidate. Such studies will investigate potential off-target effects, immune responses, and vector dissemination beyond ocular tissues, establishing comprehensive risk profiles that meet regulatory standards. These data will serve as foundational components for Investigational New Drug (IND) applications, paving the way for future human clinical trials aiming at vision restoration for USH1C patients.

Dr. Ashley Winslow, who brings over 15 years of experience in medical genetics and drug development, has been instrumental in guiding this promising gene therapy project from concept to translational application. Her expertise spans academia and industry, with postdoctoral research conducted at Massachusetts General Hospital and Harvard Medical School, equipping her with unique insights into genetic diseases and therapeutic innovation. Under her leadership, Odylia Therapeutics focuses on rare disease drug development through collaborative partnerships and cutting-edge scientific methodologies.

“The grant from C-Path is transformative for our work on USH1C-associated vision loss,” Dr. Winslow stated. “Our mission at Odylia Therapeutics is to address neglected rare genetic conditions by developing therapies that target disease mechanisms directly. This project reinforces our commitment to bringing tangible, hope-inspiring solutions to patients and families facing the daunting challenge of vision impairment.”

C-Path’s Translational Therapeutics Accelerator aligns with its broader mission to catalyze drug development by bridging academic discoveries and pharmaceutical expertise to accelerate innovative treatments. Maaike Everts, Ph.D., Executive Director of the accelerator, emphasized this alignment: “This project exemplifies the translational science we strive to support — innovative, mechanism-driven approaches targeting the root cause of disease. Our funding and collaborative model aim to expedite the journey from lab bench to patient bedside for therapies that remain elusive in the current treatment landscape.”

Critical Path Institute, established in 2005 amid the FDA’s Critical Path Initiative, remains a globally influential nonprofit organization dedicated to enhancing drug development efficiency. By nurturing more than 20 disease-specific consortia and fostering cross-sector collaborations, C-Path has become a nexus for drug development science involving regulators, industry, academia, and patient advocacy groups. Its Translational Therapeutics Accelerator uniquely empowers academic researchers to navigate the drug development process with expert guidance and regulatory insight rarely accessible to early-stage projects.

Odylia Therapeutics, founded as a nonprofit biotechnology enterprise, concentrates its efforts on addressing rare genetic diseases through innovation, collaboration, and patient engagement. Beyond its flagship USH1C gene therapy program, Odylia currently advances three gene therapy candidates targeting other rare conditions, demonstrating a commitment to filling therapeutic gaps overlooked by traditional commercial pipelines. Its Brydge Solutions program further assists early-stage biotech companies, researchers, and patient groups to facilitate translational research and accelerate drug development.

The Odylia-C-Path collaboration represents a promising convergence of scientific rigor, strategic funding, and translational expertise poised to unlock novel treatment avenues for a disease that profoundly impacts both hearing and vision. As USH1C patients and families await breakthroughs beyond cochlear implantation, this gene therapy initiative offers hope for preserving sight and enhancing quality of life through cutting-edge genetic medicine.

To learn more about Critical Path Institute’s Translational Therapeutics Accelerator, interested parties are encouraged to visit c-path.org/programs/trxa or contact trxa@c-path.org. Additional information about Odylia Therapeutics and its research portfolio can be found at odylia.org, where ongoing efforts to democratize rare disease drug development are detailed.

News Publication Date: May 20, 2026

Web References:
https://c-path.org/programs/trxa
https://odylia.org/
https://odylia.org/research-and-development/
https://odylia.org/brydge-solutions/
https://criticalpathinstitute.createsend1.com/t/y-i-awujht-hllrhunuh-i/