Foundation Fighting Blindness releases 4-year RUSH2A data to public

The Foundation Fighting Blindness has allowed public open 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 causing Usher syndrome type 2A or non-syndromic retinitis pigmentosa (RP).

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

Todd Durham, PhD, SVP, 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.”

“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 roadmap towards a potential treatment effect for those individuals with RP or non-syndromic RP due to variants in exon 13 of the USH2A gene,” added Zuhal Butuner, OD, MSc, MBA, Chief Medical Officer of Sepul Bio.

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 is 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 states it has helped with the identification of over 330 genes linked to retinal disease and the launch of 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.”

Those seeking 4-year data from the RUSH2A can request access from the foundation online

References:

1. Foundation Fighting Blindness Provides Public Access to Data From its RUSH2A Natural History Study. Published November 13, 2025. Accessed November 17, 2025. https://www.fightingblindness.org/news/rush2a-public-access-3115