Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel gene-agnostic therapies for patients suffering severe vision loss from retinal degenerative diseases, today announced the initiation of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its lead investigational therapy, MCO-010, for the treatment of severe vision loss due to retinitis pigmentosa (RP)..

Nanoscope Therapeutics Inc.,一家致力于为视网膜退行性疾病导致严重视力丧失的患者开发和商业化新型基因无关疗法的生物技术公司，今天宣布已开始为其主要研究性疗法 MCO-010 滚动提交生物制品许可申请（BLA）至美国食品药品监督管理局（FDA），该疗法用于治疗因色素性视网膜炎（RP）导致的严重视力丧失。

This marks a significant regulatory milestone as the

这标志着一个重要的监管里程碑，因为

first BLA submitted for a gene-agnostic

首个提交的基因不可知论的BLA

gene

基因

therapy

治疗

for retinal disease

用于视网膜疾病

. Being gene-agnostic means that MCO-010 is designed to address the broad genetic diversity of RP—a condition linked to over 100 known genes and more than 1,000 different mutations—

。MCO-010 基因不可知意味着它旨在应对 RP 的广泛遗传多样性——这种疾病与超过 100 种已知基因和 1,000 多种不同突变相关——

regardless of the underlying mutation

无论潜在的突变如何

The FDA has granted Nanoscope rolling review of its BLA, with the first modules already submitted and completion of the full BLA submission anticipated in early 2026. The application is eligible for priority review under the program's fast-track designation.

美国食品药品监督管理局（FDA）已授予Nanoscope公司对其生物制品许可申请（BLA）进行滚动审查的资格，首批模块已经提交，预计完整的BLA提交将在2026年初完成。该申请符合快速通道指定下的优先审查条件。

If approved, MCO-010 would be the first gene-agnostic therapy to restore vision in legally blind RP patients

如果获得批准，MCO-010 将成为首个用于恢复法定盲 RP 患者视力的基因无关疗法。

, potentially providing a significant market opportunity and, more importantly, establishing the standard of care for patients suffering from vision impairment due to RP.

，可能会提供一个重要的市场机会，更重要的是，为因视网膜色素变性（RP）导致视力受损的患者确立护理标准。

'For the first time, patients who are considered to be on a path to permanent blindness may have a chance to regain sight,' said

“一些被认为将永久失明的患者首次有机会重见光明，”

Sulagna Bhattacharya

苏拉格纳·巴塔查里亚

, CEO and Co-Founder of Nanoscope

，Nanoscope的首席执行官兼联合创始人

. 'We are deeply thankful to the FDA for their guidance as we remain steadfast in our mission to restore vision and bring light back into the lives of those living in darkness.'

“我们深深感谢FDA的指导，因为我们仍然坚定地致力于恢复视力，为生活在黑暗中的人们带回光明。”

MCO-010, Nanoscope's proprietary multi-characteristic opsin (MCO), is delivered via a one-time, in-office intravitreal injection. After injection, MCO-010 activates highly dense bipolar retinal cells to become light sensitive, utilizing remaining visual circuitry following photoreceptor death. MCO-010 is designed to offer a distinct approach in that it does not require genetic testing, surgical intervention, or repeat dosing, making it applicable to a broad population of RP patients, while also fitting into existing retina office workflows..

MCO-010是Nanoscope公司专有的多特性视蛋白（MCO），通过一次性办公室内玻璃体内注射给药。注射后，MCO-010激活高密度的双极视网膜细胞，使其对光敏感，利用感光细胞死亡后残留的视觉回路。MCO-010的独特之处在于它不需要基因测试、手术干预或重复给药，因此适用于广泛的RP患者群体，同时还能融入现有的视网膜诊疗工作流程。

'We've been working on the MCO platform for more than a decade,' added

“我们已经在这个MCO平台工作了十多年，”他补充道。

Samarendra Mohanty

萨曼德拉·莫汉蒂

, PhD, President, Chief Scientific Officer, and Co-Founder of Nanoscope

博士，纳米镜公司总裁、首席科学官兼联合创始人

. 'We've seen this investigational therapy surpass our expectations in the lab and in patients in clinical trials, and we believe we're now one step closer to potentially bringing this pioneering therapy to all RP patients.'

“我们已经看到这种研究性疗法在实验室和临床试验的患者中超越了我们的预期，我们相信我们现在距离将这种开创性疗法带给所有RP患者又近了一步。”

RP is one of the leading causes of blindness in the working-age population in the U.S., affecting more than 100,000 people and leaving over 25,000 legally blind. Vision loss due to RP is highly dependent on the underlying mutation present, and, on average, RP patients lose 1 line of vision on an eye chart approximately every three years, and a majority are legally blind (worse than 20/200 vision) by the age of 60..

视网膜色素变性（RP）是美国工作年龄人口中致盲的主要原因之一，影响超过10万人，其中2.5万人以上被认定为法定盲人。RP导致的视力丧失高度依赖于潜在的基因突变，平均而言，RP患者大约每三年在视力表上失去一行视力，并且大多数人在60岁之前会成为法定盲人（视力低于20/200）。

'One of the most challenging aspects of my entire career has been telling patients with RP there is no restorative treatment available as they experience progressive, irreversible, permanent vision loss,' said

“在我整个职业生涯中，最具挑战性的方面之一就是告诉视网膜色素变性患者，随着他们经历进行性、不可逆且永久的视力丧失，目前尚无恢复性治疗方法。”

Dr.

博士

Allen C. Ho

何艾伦

, MD, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor for Nanoscope

医学博士，威尔斯眼科医院视网膜研究主任，Nanoscope首席医疗顾问

. 'Based on the preclinical science and evidence in clinical trials, MCO-010 represents a potential, important paradigm shift for patients and retina specialists, providing hope for meaningful improvement in the quality of life for the neediest retina patients of all.'

“基于临床前科学和临床试验中的证据，MCO-010 代表了患者和视网膜专家潜在的重要范式转变，为所有最需要帮助的视网膜患者提供了生活质量显著改善的希望。”

In Nanoscope's pivotal RESTORE Phase

在纳米镜的关键恢复阶段

trial, MCO-010 met its Best Corrected Visual Acuity primary endpoints in both dose groups at 52 weeks versus sham-control, with vision gains from baseline of >0.3 LogMAR, a result equivalent to three or more lines on an eye chart. In ongoing long-term follow-up, visual acuity gains were observed through 3 years, with no serious adverse events reported in treated eyes.

在试验中，MCO-010在52周时两个剂量组均达到了与假手术对照组相比的最佳矫正视力主要终点，基线视力提高了>0.3 LogMAR，这一结果相当于视力表上的三行或更多行。在正在进行的长期随访中，观察到视力的提高持续了三年，且未报告治疗眼出现严重不良事件。

Further multi-year follow up is scheduled with these patients to evaluate longer-term efficacy and safety..

计划对这些患者进行进一步的多年随访，以评估更长期的有效性和安全性。

'It's the first time the FDA will be evaluating an application for a gene-agnostic therapy for inherited retinal disease,' concluded

“这是FDA首次评估针对遗传性视网膜疾病的基因无关疗法的申请，”总结道。

Glenn Sblendorio

格伦·斯布莱多里奥

, Chairman of the Board of Directors for Nanoscope

，纳米镜董事会主席

. 'We feel confident in our clinical data, which we believe will mark the dawn of a new era in retinal care. Our goal is to bring back the light of hope for RP patients —and eventually for many other retinal degenerative conditions.'

“我们对我们的临床数据充满信心，我们认为这将标志着视网膜治疗新纪元的开始。我们的目标是为RP患者带回希望之光——并最终为许多其他视网膜退行性疾病带来希望。”

About Nanoscope Therapeutics

关于纳米镜治疗学

Nanoscope Therapeutics is developing gene-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase

纳米镜治疗公司正在为数百万因视网膜退行性疾病而失明的患者开发与基因无关的、恢复视力的光遗传学疗法。在RESTORE阶段取得了积极成果之后

multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (

多中心、随机、双盲、假对照的视网膜色素变性（RP）临床试验 (

NCT04945772

NCT04945772

), a rolling BLA submission to the FDA has been initiated. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (.

)，已向FDA提交了滚动BLA申请。如果获得批准，MCO-010有潜力成为RP患者的护理标准，作为一次性办公室注射治疗，无需基因测试。该公司在MCO-010针对斯塔加特病（SD）的STARLIGHT 2期临床试验中也显示出令人鼓舞的结果。（

NCT05417126

NCT05417126

) and plans to initiate a

）并计划启动一项

Phase 3 registrational trial

第三阶段注册试验

in 2025. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD. Preclinical programs include Leber congenital amaurosis (LCA), in IND-enabling studies, as well as an IND-ready asset for geographic atrophy (GA).

在2025年。MCO-010 已获得 FDA 的快速通道和孤儿药资格认定，适用于 RP 和 SD。临床前项目包括正在进行 IND 支持性研究的莱伯先天性黑蒙（LCA），以及一个已准备好进入 IND 阶段的地理性萎缩（GA）资产。