N-Acetylcysteine amide

近日，总部位于冰岛雷克雅未克的临床阶段生物制药公司 Arctic Therapeutics（北极治疗公司） 宣布，其领衔在研口服小分子药物 AT-001 针对阿尔茨海默病（AD）的 IIa期临床试验已完成全部患者入组。这一里程碑标志着这款源自北欧罕见病研究的"疾病修饰型"疗法，正式向验证临床潜力迈出关键一步。 01 🧬 AT-001 是什么？ AT-001 是一款口服小分子药物，最初研发灵感来自对一种超罕见家族性痴呆症——遗传性胱抑素C淀粉样血管病（HCCAA） 的深入研究。与传统对症药物不同，AT-001 旨在实现疾病修饰（Disease-Modifying）： 通过破坏淀粉样蛋白（Aβ）斑块和 Tau 蛋白缠结形成与稳定所需的分子间相互作用，阻止有害聚集体生成并促进其溶解； 区别于目前已上市的抗Aβ单抗（如仑卡奈单抗、多奈单抗），它是口服给药，理论上依从性更好、无需静脉输注； 凭借在 HCCAA 适应症的潜力，AT-001 已获欧洲药品管理局（EMA）孤儿药资格认定（ODD），并获批在 HCCAA 中直接开展 IIb/III 期研究。 02 🔬 IIa期试验设计与最新进展 本次完成入组的是一项多中心、随机、双盲、安慰剂对照、剂量递增的 IIa 期临床试验，在冰岛和丹麦共 4 个研究中心开展，合作 CRO 为 Sanos Group： 项目 内容 样本量 计划入组约 40～50 例（随机分配至 AT-001 各剂量组及安慰剂对照组） 受试人群 50～85岁，诊断为轻度认知障碍（MCI）或轻度阿尔茨海默病患者 治疗周期 口服 AT-001，为期 12 个月，剂量逐步递增 安全性监测 每月一次实验室检查 生物标志物 每 3 个月检测血浆 pTau217、总 Tau、NfL 等（Quanterix Simoa 平台） 影像学评估 MRI（基线/中期/终点）+ PET（基线/终点）追踪脑萎缩与淀粉样负荷 最新状态：截至 2026 年中，AD 适应症的 IIa 期试验已完成全部患者入组（Full Enrollment），目前处于治疗随访阶段，顶线（Topline）数据尚未公布，预计最早于 2026 年底至 2027 年上半年读出。 03 🧪 另一战线：罕见病 HCCAA 推进更快 AT-001 在遗传性胱抑素C淀粉样血管病（HCCAA）——一种仅见于冰岛特定家系的超罕见脑血管淀粉样变性——中进度更为领先： 2025 年 11 月 EMA 正式授予 AT-001 孤儿药资格认定（ODD），用于治疗 HCCAA； EMA 已批准 AT-001 在 HCCAA 中直接开展 IIb/III 期关键性研究，是目前管线中推进最快的适应症。 若 HCCAA 的 IIb/III 期取得阳性结果，也将为 AT-001 向常见痴呆症（AD、血管性痴呆）的外推提供重要机制佐证。 04 🏢 关于 Arctic Therapeutics Arctic Therapeutics 成立于 2015 年，是美国费城儿童医院应用基因组学中心（CAG）的分拆企业，依托冰岛独特的人口家系数据和基因组学手段挖掘神经退行性疾病靶点。2025 年 1 月公司完成 2650 万欧元 A 轮融资，用于推进 AT-001（神经退行性疾病）及另一候选分子 AT-004（外用治疗痤疮，2026 年 2 月宣布首例患者入组）。 05 💡 行业视角 当前阿尔茨海默病药物研发正从"清除淀粉样蛋白"向"口服小分子、多靶点、早期干预"方向拓展。AT-001 若能在此 IIa 期研究中展现出良好的安全性及生物标志物改善趋势（pTau217 下降、PET 淀粉样负荷减少），将为口服疾病修饰疗法在 AD 领域的进一步开发增添重要证据。 不过需注意，IIa 期样本量有限（约 40～50 例），距确证性 III 期数据尚有时日，最终临床价值仍有待观察。 本文仅供医药行业资讯分享，不构成任何用药或投资建议。数据来源：Arctic Therapeutics 公告及公开医药媒体报道（截至 2026 年 7 月）。 往期回顾： 腺相关病毒（AAV）介导的基因疗法：抗衰老领域的新星 敲除内皮细胞SOX9可逆转衰老诱导的心脏功能障碍 同样是压力，为啥有的人没事，而你却焦虑得不行甚至会抑郁？科学家找到了相关的基因靶点 给细胞换个新电池？中国农大团队发现：异种线粒体移植可改善小鼠衰老表型 各种质粒构建、重组病毒递送载体（腺病毒/腺相关病毒/慢病毒等）、病毒样颗粒递送载体（VLP）、外泌体递送载体、细胞系改造（敲低/敲除/过表达/点突变等）、各类蛋白表达系统（大肠杆菌表达系统/杆状病毒表达系统/酵母表达系统/HEK293或CHO蛋白表达/无细胞表达系统）等欢迎垂询，欢迎关注公众号，每日一起学习生命科学前沿领域资讯！ 关注我们：

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