纳米镜治疗公司宣布发布STARLIGHT第二阶段研究，评估MCO-010基因疗法在斯塔加特病患者中的应用-动脉网

Nanoscope Therapeutics Announces Publication of STARLIGHT Phase 2 Study Evaluating MCO-010 Gene-Agnostic Therapy in Patients with Stargardt Disease

CISION 等信源发布 2025-08-14 20:36

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The

STARLIGHT study is the first and only clinical trial to demonstrate vision improvement in patients with Stargardt disease.

STARLIGHT研究是第一个也是唯一一个证明Stargardt病患者视力改善的临床试验。

MCO-010

demonstrated mean visual acuity improvement of 12 ETDRS letters over 48 weeks in

在48周内，平均视力提高了12个ETDRS字母

patients with atrophy confined to the macula -with no reports of serious adverse events.

黄斑萎缩的患者——无严重不良事件报告。

Nanoscope

纳米镜

has completed an end of phase 2 meeting with the FDA and

已经完成了与FDA的第二阶段结束会议，并

plans to initiate a registration-enabling Phase 3 trial in Stargardt disease patients by the end of 2025

计划在2025年底之前启动针对斯塔加特病患者的注册性三期临床试验

。

DALLAS

达拉斯

，

Aug. 14, 2025

2025年8月14日

/PRNewswire/ --

Nanoscope Therapeutics Inc.,

纳米镜治疗公司，

a biotechnology company committed to developing and commercializing novel gene-agnostic therapies for patients suffering vision loss from retinal degenerative diseases, today announced the publication of positive results from its STARLIGHT clinical trial in

一家致力于为视网膜退行性疾病导致视力丧失的患者开发和商业化新型基因不可知疗法的生物技术公司，今天宣布了其STARLIGHT临床试验的积极结果发表在

eClinical Medicine, part of The Lancet Discovery Science

《临床医学》，《柳叶刀发现科学》的一部分

。

STARLIGHT is the first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease (SD).

STARLIGHT 是首个且唯一一个证明 Stargardt 病（SD）患者视力改善的临床试验。

STARLIGHT is a Phase 2, open-label study designed to assess the safety and efficacy of a single intravitreal injection of gene-agnostic MCO-010 therapy in six participants with severe vision loss due to SD.

STARLIGHT 是一项二期、开放标签研究，旨在评估六名因SD导致严重视力丧失的参与者单次玻璃体内注射基因无关的MCO-010疗法的安全性和有效性。

Key findings include:

主要发现包括：

Mean improvement in best corrected visual acuity (BCVA) from baseline to week 48 was:

从基线到第48周，最佳矫正视力（BCVA）的平均改善为：

For all patients, +5.5 and +9.0 ETDRS letters without and with a wearable low-vision aid, respectively.

所有患者在不使用和使用可穿戴低视力辅助设备的情况下，分别获得了+5.5和+9.0 ETDRS字母。

In individuals with atrophy confined to the macula, +12.0 and +32.0 ETDRS letters, without and with a wearable low-vision aid, respectively.

在黄斑萎缩的个体中，分别使用和不使用可穿戴低视力辅助设备时，ETDRS字母得分分别为+12.0和+32.0。

Some patients observed vision improvement as early as 4 weeks post-treatment that was maintained throughout the duration of the study.

一些患者在治疗后4周就观察到视力改善，并且在整个研究期间都保持了这种改善。

MCO-010 was well tolerated with no reported serious adverse events over 48 weeks.

MCO-010在48周内耐受性良好，无严重不良事件报告。

Stargardt disease, also known as juvenile macular dystrophy, is a major cause of vision loss in children, affecting more than 40,000 people in the US and leaving over 20,000 legally blind. In patients with SD, the light-sensing photoreceptors in the macula degrade, leading to loss of central vision.

Stargardt病，也被称为青少年黄斑营养不良，是儿童视力丧失的主要原因之一，美国有超过40,000人受到影响，其中超过20,000人被认定为法定盲人。在SD患者中，黄斑中的感光光感受器退化，导致中央视力丧失。

Currently, there are no approved treatments for SD..

目前，尚无获批的SD治疗方法。

'While this was an open-label study, this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes.,' added

“虽然这是一项开放标签研究，但这是首批证明Stargardt病患者视力改善的研究之一。这一发现非常显著，因为这些患者通常会在一生中经历不可逆的中央视力丧失。”

Byron Lam

拜伦·林

, MD, Professor of Ophthalmology at Bascom Palmer Eye Institute

医学博士，巴斯科姆·帕尔默眼科研究所眼科学教授

, lead author and principal investigator of the STARLIGHT study. 'I am thrilled to be part of the upcoming Phase 3 trial of MCO-010 in Stargardt disease, helping advance this promising therapy towards potential approval.'

STARLIGHT研究的首席作者和主要研究者表示：“我非常高兴能够参与即将进行的MCO-010针对斯塔加特病的第三阶段试验，帮助推动这一有前景的疗法走向潜在的获批。”

'We are grateful to the patients and investigators who participated in the trial,' added

“我们感谢参与试验的患者和研究人员，”他补充道。

Samarendra Mohanty

萨曼德拉·莫汉蒂

, PhD, President / Chief Scientific Officer

，博士，总裁/首席科学官

. 'The results we have seen in STARLIGHT for SD patients reinforce the therapeutic potential of our gene-agnostic MCO-010 platform, building on positive outcomes in both Stargardt and retinitis pigmentosa populations.'

“我们在STARLIGHT试验中看到的SD患者结果进一步证实了我们与基因无关的MCO-010平台的治疗潜力，继Stargardt病和视网膜色素变性人群中的积极成果之后。”

The published manuscript, titled

发表的手稿，标题为

Safety and Efficacy of MCO-010 Optogenetic Therapy in Patients with Stargardt Disease in USA (STARLIGHT): an Open-label Multi-Center Ph2 Trial'

MCO-010光遗传学疗法在美国内斯塔加特病患者中的安全性和有效性（STARLIGHT）：一项开放标签、多中心的二期临床试验

, is now available online and will appear in an upcoming print issue of

现已在线提供，并将出现在即将出版的印刷版中

The Lancet.

柳叶刀。

About MCO-010

关于MCO-010

and the

和

upcoming

即将到来的

STARGAZE Phase 3 Study

STARGAZE第三阶段研究

Nanoscope's proprietary MCO-010 (

纳米镜的专有MCO-010（

sonpiretigene isteparvovec

) is a one-time, in-office intravitreal gene-agnostic therapy designed to restore vision in patients with advanced retinal degeneration. MCO-010 activates highly dense bipolar retinal cells to become light sensitive, utilizing remaining visual circuitry following photoreceptor death. MCO-010 therapy does not require genetic testing, surgical intervention, or repeat dosing, enabling broad patient applicability within existing retina office workflows..

）是一种一次性、在办公室内进行的玻璃体内基因无关疗法，旨在恢复晚期视网膜退化患者的视力。MCO-010 激活高密度的双极视网膜细胞以使其对光敏感，在光感受器死亡后利用残留的视觉回路。MCO-010 疗法不需要基因测试、手术干预或重复给药，能够在现有的视网膜办公室工作流程中实现广泛的患者适用性。

STARGAZE is a Phase 3, randomized, double-masked sham-controlled study designed to evaluate the efficacy and safety of MCO-010 in patients with Stargardt Disease. Following a successful End-of-Phae 2 meeting, and in alignment with FDA guidance, this registration-enabling study will enroll approximately 60 patients across multiple centers.

STARGAZE 是一项 III 期、随机、双盲、假对照研究，旨在评估 MCO-010 在 Stargardt 病患者中的有效性和安全性。在成功的 II 期结束会议后，并根据 FDA 指导，这项注册支持研究将在多个中心招募约 60 名患者。

The primary endpoint is the change in BCVA using a standard vision chart vs sham control. A key secondary endpoint will assess improvement in multi-luminance shape recognition. Top-line results are expected in 2027..

主要终点是使用标准视力表对比假手术对照组的BCVA变化。一个关键的次要终点将评估多亮度形状识别的改善情况。预计将在2027年公布初步结果。

For more information on MCO-010 and Nanoscope Therapeutics visit:

有关MCO-010和Nanoscope Therapeutics的更多信息，请访问：

www.nanostherapeutics.com

www.纳米治疗学.com

。

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第三阶段取得积极结果后

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

), 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有望成为RP患者的标准治疗方案，作为一次性办公室内注射使用，无需进行基因测试。该公司还在MCO-010针对斯塔加特病（SD）的STARLIGHT二期临床试验中展示了令人鼓舞的结果。（

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 快速通道和孤儿药资格，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 IND 的地理萎缩（GA）资产。

Contact:

联系：

Nanoscope Therapeutics

纳米镜治疗学

(817) 857-1186

PR@nanostherapeutics.com

SOURCE Nanoscope Therapeutics

源纳米镜治疗公司

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