Foundation Fighting Blindness Provides Public Access to Data From its RUSH2A Natural History Study
Foundation News
RUSH2A has captured extensive data over four years from patients with USH2A mutations causing non-syndromic retinitis pigmentosa and Usher syndrome type 2A.
COLUMBIA, Maryland, November 13, 2025 – The Foundation Fighting Blindness, a driving force in the global development of treatments and cures for blinding diseases, is now providing open access to de-identified four-year data from individuals participating in RUSH2A, an ongoing natural history study for people with mutations in the USH2A gene causing Usher syndrome type 2A or non-syndromic retinitis pigmentosa (RP). The goal of RUSH2A and other natural history studies sponsored by the Foundation 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. With improved endpoints and trial designs, therapy developers have a better opportunity to gain regulatory approval for their treatments.
“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,” said Todd Durham, PhD, SVP, Foundation Fighting Blindness. “As the global leader in driving retinal disease research, the Foundation is uniquely empowered to launch programs like RUSH2A, which are having a broad, paradigm-changing impact on advancing more treatments toward the finish line.”
“The RUSH2A study has been a cornerstone to our ongoing evaluation of how we can develop effective treatments for USH2A-related conditions,” said Dr. Zuhal Butuner, Chief Medical Officer, Sepul Bio, a business unit of Théa. “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. We are proud to partner with the Foundation Fighting Blindness in leveraging the RUSH2A data to drive forward our USH2A program and bring innovative RNA therapies to the community as quickly and effectively as possible.”
RUSH2A has followed more than 100 USH2A patients for more than four years and will be extended for a total of nine years. Investigators are collecting natural history data using several functional and structural measures, including best-corrected visual acuity (BCVA), microperimetry, full-field sensitivity, optical coherence tomography, and, as part of the extension, virtual-reality based mobility. RUSH2A 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 Jaeb Center for Health Research is the coordinating center for the Consortium.
The Foundation has invested more than $3 million in RUSH2A thus far. Co-funders of the study include BlueRock Therapeutics, Restore Vision, and Sepul Bio.
Instructions for requesting data access to the RUSH2A data set are available at: https://public.jaeb.org/ffb/view/RUSH2A
The RUSH2A study investigators have published 16 peer-reviewed papers to date (https://public.jaeb.org/ffb/pubs). The following research paper from the journal TVST reviews four-year results and recommendations from RUSH2A: Endpoints and Design for Clinical Trials in USH2A-Related Retinal Degeneration
About the Foundation
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 $954 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit FightingBlindness.org for more information.
Media Contact:
Chris Adams
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CAdams@FightingBlindness.org
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