Foundation Fighting Blindness Shares 4-Year RUSH2A Data

The Foundation Fighting Blindness has announced the public release of de-identified four-year data from participants in RUSH2A, an ongoing natural history study focused on individuals with mutations in the USH2A gene. Mutations in this gene are known to cause Usher syndrome type 2A or nonsyndromic retinitis pigmentosa (RP).

Advancing Research Through Data Accessibility

According to the foundation, the goal of RUSH2A (NCT03146078) and other sponsored natural history studies is to help researchers and therapy developers identify new, sensitive, and reliable clinical trial endpoints and to inform the design of future trials targeting inherited retinal diseases (IRDs).

“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, senior vice president of clinical and outcomes research at the Foundation Fighting Blindness.

Data Supporting USH2A Treatment Development

Zuhal Butuner, OD, MSc, MBA, chief medical officer of Sepul Bio, emphasized the value of RUSH2A in shaping therapeutic development for USH2A-related conditions.

“The RUSH2A study has been a cornerstone to our ongoing evaluation of how we can develop effective treatments for USH2A-related conditions,” Butuner said. “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.”

Study Design and Scope

The RUSH2A study has followed more than 100 patients with USH2A mutations for over four years and will continue for a total of nine years. Data is being collected using a combination of:

• Best-corrected visual acuity (BCVA)

• Microperimetry

• Full-field sensitivity

• Optical coherence tomography (OCT)

The study is conducted through the foundation’s Clinical Consortium, a network of more than 40 clinical sites operating under standardized protocols and equipment. This consortium includes leading clinical experts in inherited retinal diseases, working collaboratively to ensure consistency and reliability in data collection.