N-Acetylcysteine amide

The commitment directs $775,000 to the RD Fund, to be matched dollar for dollar, and $225,000 to a Foundation Fighting Blindness research award, advancing approaches that can help patients regardless of the gene causing their retinal disease COLUMBIA, Md. and RALEIGH, N.C., June 17, 2026 /PRNewswire/ -- The Foundation Fighting Blindness and its venture arm, the RD Fund (Retinal Degeneration Fund), today announced a $1 million commitment from A Race Against Blindness (RAB) to advance gene-agnostic therapeutic approaches for inherited retinal diseases. The commitment directs $775,000 to the RD Fund and $225,000 to a Foundation Fighting Blindness research award. The $775,000 to the Fund will be matched dollar for dollar through the Gordon and Llura Gund Foundation Challenge, unlocking $1.55 million in impact, a donor match that doubles new and increased gifts to the RD Fund. A Race Against Blindness was founded in 2023 by Dr. Stephen and Kristina Johnston after their son was diagnosed with retinitis pigmentosa (RP) caused by Bardet-Biedl syndrome (BBS). Since then, the family-run nonprofit has directed millions of dollars toward research for RP and BBS, with a focus on pediatric inherited retinal diseases and on moving promising therapies from concept to clinic with urgency. "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," said Dr. Stephen Johnston, co-founder of A Race Against Blindness. "Gene-agnostic approaches offer hope to the broadest possible community, including children with Bardet-Biedl syndrome like our son. By directing our support to the RD Fund and to the Foundation's research, and seeing the Fund portion matched, we can put more resources behind the science that reaches the most families the fastest." Advancing Gene-Agnostic Approaches Across the RD Fund Portfolio The $775,000 directed to the RD Fund will support gene-agnostic therapeutic approaches across the Fund's portfolio companies, which are developing treatments designed to help patients regardless of the specific gene mutation causing their disease. The RD Fund intentionally balances its portfolio between gene-specific and gene-agnostic strategies, including neuroprotective and antioxidant approaches that slow disease progression and vision-restoration strategies such as optogenetics, which aims to restore light sensitivity to the retina independent of the underlying gene defect. One example is Nacuity Pharmaceuticals, a clinical-stage portfolio company developing a neuroprotective, gene-agnostic candidate. Nacuity's lead candidate, NPI-001, is an oral tablet formulation of N-acetylcysteine amide that targets oxidative stress, a process that damages photoreceptors across many forms of retinal degeneration. Rather than correcting a single gene, NPI-001 is designed to boost 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. "The partnership with A Race Against Blindness reflects the conviction the RD Fund 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 community, including the RP and BBS1 families represented by A Race Against Blindness." Funding Gene-Agnostic Innovation at the Foundation Fighting Blindness The remaining $225,000 of the commitment will complete funding for a Foundation Fighting Blindness Career Development Award supporting Dr. Thomas Mendel at The Ohio State University Wexner Medical Center in Columbus, Ohio. The five-year, $375,000 award funds research into a new way to deliver retinal gene therapy that could apply across many inherited retinal diseases. Dr. Mendel is testing a surgical approach in preclinical models that delivers 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 approach aims to deliver therapy faster and with less inflammation, without compromising retinal function. Because the delivery method is gene-agnostic, it has the potential to improve treatment for conditions including Bardet-Biedl syndrome, choroideremia, Leber congenital amaurosis, Stargardt disease, Usher syndrome, and multiple forms of retinitis pigmentosa. Dr. Mendel's work is mentored by Andy J. Fischer, PhD, and Cagri Besirli, MD, PhD. "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. "Whether through the RD Fund's portfolio or a Foundation-supported researcher like Dr. Mendel, this commitment from A Race Against Blindness accelerates approaches that do not depend on a single gene, and that gives hope to families across the entire community." 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, a track record that made it a natural partner for A Race Against Blindness. About A Race Against Blindness A Race Against Blindness is a family-run nonprofit co-founded in 2023 by Dr. Stephen and Kristina Johnston after their son was diagnosed with retinitis pigmentosa caused by Bardet-Biedl syndrome. Through innovative fundraising and a focus on pediatric inherited retinal diseases, the organization has directed more than $5 million toward research for retinitis pigmentosa and Bardet-Biedl syndrome, working to move promising therapies from concept to clinic with urgency. For more information, visit araceagainstblindness.org. About the Foundation Fighting Blindness Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised nearly $1 billion 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. About the RD Fund The Retinal Degeneration Fund (RD Fund) is a mission-driven venture fund established by the Foundation Fighting Blindness to invest in companies developing innovative therapies for inherited retinal diseases and dry age-related macular degeneration. The RD Fund was designed to bridge the funding gap between discovery research and confirmatory clinical trials. For more information, visit . Media Contact: Chris Adams Foundation Fighting Blindness 410-423-0585 [email protected] SOURCE Foundation Fighting Blindness 21% more press release views with Request a Demo

重磅进展：Usher综合征迎来广谱口服新药，Ⅲ期临床试验正式启动 Usher综合征俗称“聋-盲绝症”，属于罕见遗传性疾病，患者同时存在先天性耳聋与视网膜色素变性，最终可致视力、听力双重丧失。过往治疗方案多针对特定基因型开发，适用人群受限。 由药企研发的NPI-001口服片剂，以抗氧化应激为作用机制，可提升体内谷胱甘肽水平，阻断活性氧对视网膜细胞的损伤。药物可穿透血-视网膜屏障、血脑屏障，作用靶点明确，且不依赖致病基因型，覆盖所有类型患者。 前期Ⅱ期临床数据证实，该药物能将视网膜光感受器细胞凋亡速度减缓50%以上。目前该药全球Ⅲ期临床试验已开启，采用随机、双盲、安慰剂对照设计，进一步验证长期疗效与安全性。作为首款临床获益明确的Usher综合征小分子药物，其研发落地将填补此类罕见病的治疗空白。 参考文献 1. Whatley M, Francis A, Ng ZY, et al. Usher Syndrome: Genetics and Molecular Links of Hearing Loss and Directions for Therapy[J]. Front Genet, 2020, 11:565216. 2. Clinicaltrials.gov. NCT04355689、NCT07290530 临床试验公开数据 3. Nacuity Pharmaceuticals. NPI-001药物研发及适应症公告 #Usher综合征 #罕见病 #视网膜色素变性 #眼科新药 #临床试验