Foundation Fighting Blindness releases 4-year RUSH2A data to public

Foundation Fighting Blindness has granted public access to de-identified 4-year data from individuals participating in RUSH2A, an ongoing natural history study for patients with mutations in the USH2A gene, which causes Usher syndrome type 2A or nonsyndromic retinitis pigmentosa (RP).

According to the foundation, the goal of RUSH2A (NCT03146078) and other natural history studies it sponsors is to enable researchers and therapy developers to identify new, sensitive, and reliable clinical trial end points and inform clinical trial designs for emerging inherited retinal disease (IRD) therapies.

Todd Durham, PhD, senior vice president of clinical and outcomes research, Foundation Fighting Blindness, commented on the data, saying, “The public release of RUSH2A data is removing barriers for everyone in the research community who is developing sight-saving therapies for inherited retinal diseases.”¹

“The RUSH2A study has been a cornerstone to our ongoing evaluation of how we can develop effective treatments for USH2A-related conditions. In an area of high unmet need, like USH2A, natural history studies can play a critical role in guiding clinical research. Incorporating key findings from the RUSH2A study into our ongoing USH2A clinical program has enabled the Sepul Bio team to lay down the foundational road map toward a potential treatment effect for those individuals with RP or nonsyndromic RP due to variants in exon 13 of the USH2A gene,” added Zuhal Butuner, OD, MSc, MBA, chief medical officer of Sepul Bio.¹

According to the foundation, RUSH2A has followed more than 100 USH2A patients for more than 4 years and will be extended for a total of 9 years. Natural history data are being collected using best-corrected visual acuity (BCVA), microperimetry, full-field sensitivity, and optical coherence tomography (OCT). The trial is being conducted through the foundation’s Clinical Consortium, a network of more than 40 clinical sites with standardized protocols, equipment, and clinical IRD experts.

The Clinical Consortium is a not-for-profit alliance of retinal disease specialists and geneticists “committed to accelerating the development of efficacious treatments for IRDs.”

Additionally, the foundation has helped identify over 330 genes linked to retinal disease and launch over 45 clinical trials for potential treatments. The foundation states that it leverages its “unsurpassed clinical, technical, and analytical expertise to advance rigorous clinical research, promote the creation of meaningful programs, and engender impactful collaboration in search of treatment breakthroughs for these diseases.”

Individuals seeking 4-year data from the RUSH2A trial can request access from the foundation online.

Reference

1. Foundation Fighting Blindness provides public access to data from its RUSH2A natural history study. News release. Foundation Fighting Blindness. November 13, 2025. Accessed November 17, 2025. https://www.fightingblindness.org/news/rush2a-public-access-3115