A Race Against Blindness Commits $1 Million to Advance Gene-Agnostic Therapies for Inherited Retinal Diseases
核心洞察
A Race Against Blindness (搜索) has committed $1 million to the Foundation Fighting Blindness and its RD Fund (搜索) to advance gene-agnostic therapeutic approaches for inherited retinal diseases.
The commitment directs $775,000 to the RD Fund (搜索), matched dollar-for-dollar through the Gordon and Llura Gund Foundation Challenge, unlocking $1.55 million in total impact.
The remaining $225,000 funds a Career Development Award supporting Dr. Thomas Mendel's research into a novel gene-agnostic retinal gene therapy delivery method at The Ohio State University.
A Race Against Blindness (搜索) (RAB), a family-run nonprofit founded in 2023, has committed $1 million to the Foundation Fighting Blindness and its venture arm, the RD Fund (搜索) (Retinal Degeneration Fund), to accelerate gene-agnostic therapeutic approaches for inherited retinal diseases. The announcement, made on June 17, 2026, directs $775,000 to the RD Fund and $225,000 to a Foundation Fighting Blindness research award, with the RD Fund portion matched dollar-for-dollar through the Gordon and Llura Gund Foundation Challenge, unlocking a total of $1.55 million in impact.
The commitment reflects an urgent push to develop treatments that can benefit patients across the spectrum of inherited retinal diseases, irrespective of the specific gene mutation underlying their condition. A Race Against Blindness (搜索) was established by Dr. Stephen and Kristina Johnston after their son was diagnosed with retinitis pigmentosa (搜索) (RP) caused by Bardet-Biedl syndrome (搜索) (BBS). Since its founding, the organization has directed millions of dollars toward research for RP and BBS, with a particular focus on pediatric inherited retinal diseases.
"As parents, we know families cannot wait for a separate therapy to be developed for every one of the thousands of mutations that cause retinitis pigmentosa (搜索)," said Dr. Stephen Johnston, co-founder of A Race Against Blindness (搜索). "Gene-agnostic approaches offer hope to the broadest possible community, including children with Bardet-Biedl syndrome (搜索) like our son."
Strengthening the RD Fund Portfolio
The $775,000 directed to the RD Fund (搜索) will support gene-agnostic therapeutic approaches across the Fund's portfolio companies. The RD Fund intentionally balances its investments between gene-specific and gene-agnostic strategies, including neuroprotective and antioxidant approaches that slow disease progression, as well as vision-restoration strategies such as optogenetics, which aims to restore light sensitivity to the retina independent of the underlying gene defect.
A prominent example is Nacuity Pharmaceuticals (搜索), a clinical-stage portfolio company developing NPI-001, an oral tablet formulation of N-acetylcysteine amide. Rather than correcting a single gene, NPI-001 targets oxidative stress—a process that damages photoreceptors across many forms of retinal degeneration—by boosting glutathione, one of the body's most powerful natural antioxidants, to help protect retinal cells regardless of the underlying mutation. The U.S. Food and Drug Administration has granted NPI-001 Fast Track, Breakthrough Therapy, and Orphan Drug designations for the treatment of retinitis pigmentosa (搜索).
"The partnership with A Race Against Blindness (搜索) reflects the conviction the RD Fund (搜索) was built to reward," said Rusty Kelley, PhD, managing director of the RD Fund. "Their support, matched by the Gordon and Llura Gund Foundation, allows us to diversify and strengthen our portfolio of gene-agnostic approaches that could one day benefit the entire retinitis pigmentosa (搜索) community."
Advancing Novel Gene Therapy Delivery
The remaining $225,000 of the commitment completes funding for a Foundation Fighting Blindness Career Development Award supporting Dr. Thomas Mendel at The Ohio State University Wexner Medical Center. The five-year, $375,000 award funds preclinical research into a novel method of delivering retinal gene therapy that could apply across many inherited retinal diseases.
Dr. Mendel's approach involves delivering gene therapy onto the surface of the retina, rather than beneath it, combined with insulin added to the therapy to accelerate its uptake into retinal cells. The method aims to deliver therapy faster and with less inflammation, without compromising retinal function. Because the delivery method is gene-agnostic, it holds potential to improve treatment for conditions including Bardet-Biedl syndrome (搜索), choroideremia, Leber congenital amaurosis, Stargardt disease, Usher syndrome, and multiple forms of retinitis pigmentosa (搜索).
"Some of the most powerful ideas in our field are the broad-reaching ones that can help many patients across the spectrum of inherited retinal diseases," said Amy Laster, PhD, chief scientific officer of the Foundation Fighting Blindness. "This commitment from A Race Against Blindness (搜索) accelerates approaches that do not depend on a single gene, and that gives hope to families across the entire community."
Broader Funding Momentum
In a separate announcement on August 3, 2026, A Race Against Blindness (搜索) also launched a new Research Grants Program to further advance breakthrough science for inherited retinal diseases and Bardet-Biedl Syndrome (搜索). The program invites universities, hospitals, nonprofit research organizations, and biotechnology companies to submit proposals for innovative research, with funding priorities spanning novel therapeutics, translational and preclinical research, clinical studies, biomarker discovery, and quality-of-life technologies. Applications will be accepted on a rolling basis and reviewed through an independent scientific review process.
To date, A Race Against Blindness (搜索) has committed $5.1 million to move AXV-101 toward clinical study and $1 million to expand gene-agnostic therapeutic approaches. As the world's leading private funder of retinal degenerative disease research, the Foundation Fighting Blindness has played a key role in driving the development of more than 60 retinal disease drug candidates now in clinical trials.