This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study

开始日期2023-12-08

申办/合作机构

Nanoscope Therapeutics, Inc.

NCT06048185

/ Active, not recruitingN/A

Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study

The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.

开始日期2023-10-19

申办/合作机构

Nanoscope Therapeutics, Inc.

NCT05921162

/ Enrolling by invitationN/A

A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection

This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection

开始日期2023-07-14

申办/合作机构

Nanoscope Therapeutics, Inc.

100 项与 Sonpiretigene Isteparvovec 相关的临床结果

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100 项与 Sonpiretigene Isteparvovec 相关的转化医学

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100 项与 Sonpiretigene Isteparvovec 相关的专利（医药）

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项与 Sonpiretigene Isteparvovec 相关的文献（医药）

2025-09-01·EClinicalMedicine

Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial

Article

作者: Ayyagari, Ananta ; Chang, Jean ; Koester, John ; Mahajan, Vinit B ; Ho, Allen C ; Barone, Samuel B ; Chavala, Sai H ; Sharif, Najam A ; Lane, Hayley ; Gregori, Ninel Z ; Carlson, Michael ; Von Tress, Mark ; Lam, Byron L ; Gonzalez, Victor H ; Kim, Sanghoon ; Batabyal, Subrata ; Mohanty, Samarendra K ; Piltz-Seymour, Jody ; Choudry, Nozhat ; Tsang, Stephen H ; Boyer, David S ; Zak, Vitaliy

Background:

Stargardt disease (SD) is an inherited degenerative retinal disease affecting rod and cone photoreceptors and retinal pigment epithelial (RPE) cells, leading to severe and irreversible vision loss. Optogenetics is a promising approach to restoring vision by photosensitizing spared healthy retinal neurons.

Methods:

The STARLIGHT phase 2 open-label study (NCT05417126) was conducted over 48-weeks at two US sites to assess the safety and efficacy of MCO-010 administered via a single intravitreal injection in the worse-seeing eye of SD patients. The study began on July 5, 2022, and was completed on September 28, 2023. MCO-010 targets bipolar cells rather than retinal ganglion cells (RGCs) to utilize more abundant cells that respond to ambient light while preserving natural visual processing pathways after treatment. Six adults (mean age 50 years, range 32-71 years, four males and two females) received 1.2E11 genome copies (gc)/eye of MCO-010. The primary outcome was the incidence, nature, severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), intraocular inflammation, retinal thickness, best-corrected visual acuity (BCVA), and lesion size. Secondary endpoints included change in BCVA, vision-guided mobility, and shape determination accuracy. Visual field perimetry and Michigan Retinal Degeneration Questionnaire (MRDQ) were exploratory endpoints.

Findings:

All six participants had at least one ocular TEAE; non-ocular TEAEs occurred in three participants. The most common TEAEs were conjunctival hemorrhage, ocular hypertension, and vitreous cells (two subjects). There were no deaths, hospitalization, loss of eye, retinal detachment, endophthalmitis, or TEAEs leading to study discontinuation. The BCVA (mean ± SD) of the six treated eyes at baseline and 48-week follow-up was 22.8 ± 9.87 (range 9-35), and 28.3 ± 13.28 (range 4-42) ETDRS letters, respectively. The BCVA change from baseline was 7.2 ± 11.74, 4.2 ± 14.81, and 5.5 ± 12.29 at 12, 24, and 48 weeks, respectively. With a wearable magnifier (low-vision glasses), the BCVA change was 17.8 ± 13.35, 15.7 ± 17.37, 13.3 ± 21.37 at 12, 24, and 48 weeks, respectively. The improvement in mean defect in visual field perimetry was 1.02 ± 3.54, 2.47 ± 5.00, and 2.63 ± 5.26 dB at 12, 24, and 48 weeks, respectively. Specific improvements were noted in reading, and color, and contrast domains of the MRDQ.

Interpretation:

MCO-010 optogenetic phase 2 results support further investigation for treatment SD. To our knowledge, this is the first report of improving on(eye)-chart vision of SD participants utilizing optogenetics.

Funding:

Nanoscope Therapeutics Inc.

2025-08-01·Journal of Managed Care & Specialty Pharmacy

The effectiveness and value of sonpiretigene isteparvovec for the treatment of advanced retinitis pigmentosa

Article

作者: Rind, David M. ; McKenna, Avery ; Agboola, Foluso ; Richardson, Marina ; Phillips, Marie ; Makam, Anil N. ; Lee, Woojung ; Herce-Hagiwara, Belen

2025-05-01·MOLECULAR THERAPY

Perspectives and open questions in vision restoration with vMCO-010

Letter

作者: Lindner, Moritz

133

项与 Sonpiretigene Isteparvovec 相关的新闻（医药）

2026-01-20

·PRNewswire

Nanoscope Secures Japan MHLW Sakigake and Orphan Drug Designations Across Inherited Retinal Diseases - A First for a Retinal Gene Therapy

Japan's Ministry of Health, Labor and Welfare (MHLW) grants MCO-010 Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations, enabling an accelerated regulatory pathway to treat patients with severe vision loss from inherited retinal diseases Builds on strong global regulatory momentum, including five EMA Orphan designations and multiple FDA expedited programs across retinal indications FDA, EMA and PMDA conditionally approve MOGENRY™ brand name for MCO-010 as Nanoscope's rolling BLA with the FDA for retinitis pigmentosa advances Positions MCO-010 for global pre-commercialization readiness across major markets, reinforcing Nanoscope's leadership in optogenetic therapy DALLAS, Jan. 20, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Sakigake and Orphan Drug designations to MCO-010, Nanoscope's lead asset to treat patients with severe vision loss from inherited retinal diseases (IRDs). The Sakigake designation, Japan's premier fast-track regulatory program for innovative therapies addressing serious unmet medical needs, is designed to accelerate patient access to breakthrough treatments. Sakigake status enables prioritized regulatory consultation and review—often targeting a six-month review timeline—through close collaboration with the Pharmaceuticals and Medical Devices Agency (PMDA) and may support premium pricing following approval. Combined with Orphan Drug designation (for use in less than 50,000 patients with high medical need), the decision establishes a strong regulatory pathway for MCO-010 in Japan. MCO-010 is the first ever retinal gene therapy to be granted both Sakigake and Orphan Drug designations. "These designations from Japan's MHLW mark another pivotal milestone for Nanoscope," said Samarendra Mohanty, PhD, President, and Chief Scientific Officer of Nanoscope Therapeutics. "Sakigake recognition underscores the novelty of MCO-010 as a one-time, in-office intravitreal optogenetic therapy and its potential to address profound vision loss where no approved treatments exist. Together with our progress in the U.S. and Europe, we are advancing one of the most comprehensive global regulatory strategies in optogenetic retinal gene therapy for patients living with inherited retinal diseases." Strong, Major Market Regulatory Momentum The MHLW's decision builds on accelerated regulatory pathways across other major markets: European Union (EMA): Five Orphan designations spanning non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies United States (FDA): Orphan Drug and Fast Track designations for retinitis pigmentosa (RP) Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations for Stargardt disease (SD) In addition, the FDA, EMA and PMDA have conditionally approved the brand name MOGENRY™ for MCO-010, while Nanoscope continues to advance its rolling Biologics License Application (BLA) for marketing approval for RP patients in the United States. With these regulatory pathways now established in Japan, the United States, and Europe, MCO-010 represents the most advanced optogenetic platform therapy in development worldwide. These coordinated designations support accelerated development timelines, enhanced regulatory engagement, and the opportunity to be the first optogenetic therapy approved across major markets. About the MCO Platform MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including Retinitis Pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO treatment does not require genetic testing, invasive surgery, or repeat dosing, enabling broad patient applicability within existing retina office workflows. About Nanoscope Therapeutics Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (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) (NCT05417126) and plans to initiate a Phase 3 registrational trial in early 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start in early 2026. Other IND-ready programs include Leber congenital amaurosis (LCA). Contact: Nanoscope Therapeutics, Inc (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics 21% more press release views with Request a Demo

孤儿药临床2期快速通道临床结果基因疗法

2026-01-16

·北京佰鹤明智能科技有限公司

眼科近十年的创新发展巨变

三连一下，了解更多内容 ❖❖❖ 2010年至今，临床和手术中的技术不断改进，目标是为患者提供更早期、更精确的诊断以及简化的管理。眼科领域的人工智能进展增强了疾病检测和管理能力，但临床整合和数据质量方面仍存在挑战。基因疗法和CRISPR为遗传性眼部疾病提供了有前景的治疗方法，临床试验取得了重大进展。光遗传学在退行性眼病的视力恢复方面具有潜力，正在进行的临床试验探索各种治疗选择。光学相干断层扫描(OCT)的进展改善了手术效果和诊断，未来发展集中在分辨率、成像深度和人工智能整合方面。新型药物和手术创新不断推进年龄相关性黄斑变性及其他眼部疾病的治疗选择。随着眼科技术飞速发展，人工智能和基因疗法占据了中心舞台。 ➭人工智能过去十年中，人工智能(AI)在检测和管理眼部疾病(如糖尿病性视网膜病变(DR)、青光眼和年龄相关性黄斑变性(AMD)方面的开发和应用有了飞跃式发展。人工智能的应用将通过自动筛查、精准诊断和优化治疗方案彻底改变眼科护理。在糖尿病性视网膜病变中，人工智能算法分析视网膜图像以准确识别病变，这对眼科临床实践中的医生有帮助。像IDx-DR(IDx Technologies公司)这样的系统已获得FDA批准，可自主检测可转诊的糖尿病性视网膜病变。对于青光眼，深度学习模型通过评估眼底照片中的视神经乳头形态来检测损伤。在年龄相关性黄斑变性中，人工智能可以量化玻璃膜疣，并从彩色眼底和光学相干断层扫描(OCT)图像中诊断疾病严重程度。人工智能还被用于早产儿视网膜病变、圆锥角膜和干眼症的筛查。人工智能还可以通过预测疾病进展和对抗血管内皮生长因子(VEGF)疗法的反应来帮助临床医生管理疾病。由于训练数据的质量和多样性、缺乏严格的临床验证以及监管批准和临床医生信任方面的挑战等限制，这些能力仍在不断完善中。目前的其他障碍包括将人工智能整合到现有的临床工作流程中以及确保人工智能决策过程的透明度。不过，将人工智能研究转化为可行的临床工具的工作仍在继续。算法性能在很大程度上取决于训练数据的质量和多样性。缺乏可解释性的“黑匣子”人工智能系统引发了合理的质疑，并且它们尚未无缝整合到现有的临床实践中。然而，研究人员仍相信“人工智能有巨大潜力为临床医生提供数据驱动的客观评估，这可能从根本上改变眼科护理。” ➭基因疗法最近，眼部基因治疗的重点一直是与各种遗传因素相关的疾病。由于眼睛具有独特的特征，如易于接近以及能够针对角膜和视网膜疾病，包括色素性视网膜炎(RP)、莱伯先天性黑蒙(LCA)、年龄相关性黄斑变性(AMD)和斯塔加特病，因此眼睛是基因治疗的理想候选对象。目前，文献记载了该领域的33项临床试验，其中最有希望的结果来自针对LCA的试验。最初的研究促使人们进一步涉足青光眼、AMD、RP和脉络膜视网膜病变。基因治疗的有效性需要将遗传物质高效、持续地递送至特定细胞。为此，研究人员使用了病毒载体，尽管存在免疫反应风险，并且基因突变导致了非病毒载体系统的发展。 2017年FDA批准的首个基因治疗药物是Luxturna（voretigene neparvovec-rzyl,Spark Therapeutics公司），用于治疗一种遗传性视网膜疾病，即LCA。根据美国眼科学会的数据，这种疗法使用AAV2病毒载体来替代导致几种常染色体隐性视网膜营养不良的RPE65基因突变拷贝，包括RP和LCA的亚型。发表在《柳叶刀》杂志上的3期研究表明，“一次性视网膜下注射Luxturna可使患有双等位基因RPE65突变的儿童和成人患者的光感知能力大幅提高。这种改善在长达3年及更长时间内保持稳定。这一进展被视为遗传性视网膜疾病患者治疗模式的转变。” ➭基因编辑诸如成簇规律间隔短回文重复序列/CRISPR相关蛋白9(CRISPR/Cas9)和光遗传学等新兴技术，或许能够通过修改遗传密码来实现视觉恢复。 CRISPR于2020年3月首次在眼科临床试验中用于治疗10型LCA。一名患者在波特兰俄勒冈健康与科学大学凯西眼科研究所接受了基于CRISPR的EDIT-101疗法注射到眼睛中。该手术旨在修复CEP290基因的突变。波士顿麻省眼耳医院的哈佛医学院研究人员进行了一项概念验证基因编辑一期至二期研究，评估了该技术对患有CEP290相关遗传性视网膜变性患者的效果。14名患者在其较差的眼睛接受了EDIT-101视网膜下注射。主要研究结果是安全性。 ➭光遗传学光遗传学的最新进展有望恢复退行性眼病患者的视力。光遗传学被定义为利用光来控制靶向细胞的细胞活性的技术。尽管光遗传学是一项相对较新的技术，但目前已经在临床前和一期/二期临床试验中探索了多种治疗选择，旨在开发针对主要致盲眼病，如青光眼和RP的新型、安全且有效的治疗方法。光遗传学恢复视力的方法主要旨在通过将光敏感蛋白插入没有内在光敏感性的下游视网膜神经元来替代丢失或功能失调的光感受器。这种方法很有吸引力，因为它与视网膜变性的遗传原因无关，这让人们希望所有形式的视网膜营养不良和退行性疾病都能得到治疗。《自然方法》将光遗传学评为2010年科学与工程领域的“度年方法”。《科学》报道光遗传学是“十年突破”之一。2021年，这项技术首次应用于医学，一名因视网膜色素变性而失明的患者恢复了部分视力。一种编码ChrimsonR的腺相关病毒载体被注射到这位患有40年视网膜色素变性的58岁患者的眼睛中。结合通过特制护目镜进行光刺激以激活经光遗传学转导的视网膜神经节细胞，患者报告称，在佩戴护目镜时，仅用接受载体治疗的眼睛就能感知、定位、计数和触摸不同物体。一种很有前景的光遗传学疗法是MCO-010(纳米镜疗法公司)，该疗法目前正在作为视网膜色素变性和斯特格氏病的治疗方法进行研究。MCO-010已获得FDA针对这些病症的快速通道和孤儿药认定。该分子通过单次玻璃体内注射引入眼睛的腺相关病毒进行递送。除了MCO-010，其他用于视网膜色素变性的光遗传学疗法还包括GS030(基因视力生物制药公司)；RST- 001(RetroSense/艾尔建公司)；BS01(仿生视觉公司)和RTx - 015(瑞伊疗法公司)。这些疗法在细胞靶点、动力学以及光谱和光敏感性方面存在差异。 ➭研究DNA损伤的新方法纽约洛克菲勒大学的博士后研究员艾米丽·C·贝克维特博士及其团队正在深入研究DNA损伤在分子层面的原因和机制。这一研究方向正在探究酵母如何在细胞周期检查点(即人类的共济失调毛细血管扩张症和Rad3相关通路)中感知DNA损伤。当细胞中积累了过多损伤时，细胞必须在继续分裂之前优先进行修复。如果细胞带着过多DNA损伤进行分裂，就会导致致癌突变。人类进化出了一种叫做‘复制应激和DNA损伤检查点’的机制，这是一个非常复杂的通路，基本上能感知是否存在过多损伤，并告诉细胞停止一切活动，直到修复完成。这是正常细胞功能的一个基本/内在部分，因为DNA损伤是不可避免的。例如，在正常的细胞代谢和阳光照射过程中，DNA不断受到损伤并得到修复。这个检查点就像是细胞的一个内置安全装置。所有真核生物，即DNA存在于细胞核中的细胞的生物体，都有这个通路。有缺陷的DNA损伤检查点蛋白，它们无法感知损伤；在这种情况下，细胞会继续分裂并传播损伤，使生物体更容易发生突变/患癌症。第二个方面涉及正常的检查点蛋白，但癌症是由其他原因发展而来的；在这种情况下，一种方法是用药物抑制检查点，使癌细胞更加失控从而杀死它们。 ➭光学相干断层扫描技术的进展 21世纪初，光学相干断层扫描(OCT)成为眼内手术中一项非常重要的技术，当时外科医生认识到了其可视化实时组织和手术界面的潜力。术中OCT改变了视网膜专科医生观察和处理视网膜疾病的方式。它在术中的应用允许在手术过程中进行实时反馈，能够精确评估微观结构变化并验证手术效果。自1991年推出以来，OCT的主要进展包括谱域OCT的发展、扫频源OCT(SS-OCT)以实现更宽视野和更快扫描、用于无创三维血流成像的OCT血管造影、用于实时手术指导的术中OCT，以及技术扩展到家庭监测设备和手持版本。这些创新带来了视网膜和脉络膜疾病检测的改善、手术效果的提高以及新的诊断可能性。多年来，这项技术发展迅速。 2010年，引入了傅里叶域锁模(FDML)OCT，使用FDML激光进行组织扫描，提供了非常高的速度和图像清晰度。 2015年，谱域OCT与扫频源OCT相结合，提高了图像准确性和深度。 2020年，先进的OCT血管造影和分子OCT被引入，能够对血流和特定分子进行成像。 2024年，光学相干断层扫描(OCT)被应用于神经外科、职业治疗和皮肤科。预测OCT研发在几个关键领域即将取得重大进展：提高分辨率和成像深度已成为主要关注点，科学家们致力于开发更优质的光源和扫描技术，以提高图像质量并实现更深的组织穿透。此外，高速成像是一个重要的探索领域，旨在改进成像硬件和软件，同时减少运动伪影，从而提升实时成像能力。眼科领域越来越关注将OCT与其他成像方式(如荧光和超声)结合使用，并开发更准确、可靠的早期疾病检测和评估治疗效果的方法。OCT分子成像的一个重要趋势是人工智能和机器学习的整合，特别是在预测分析方面。随着用于分割、分类和量化的人工智能网络的发展，对人工分析的需求将减少。还正在创建预测模型，以根据现有的OCT数据估计疾病进展和治疗结果，从而辅助治疗规划。这涉及整合物理学家、工程师、临床医生和计算机科学家的专业知识，以加强技术进步向临床实践的转化。这种合作还应包括法律和伦理方面的考虑，以保护新兴的OCT技术不被滥用，并确保在人工智能应用中尊重隐私和道德标准。简化OCT并开发具有成本效益的OCT系统对于在资源有限的环境中使用很重要，尤其注重小型化和成本降低。此外，OCT越来越多地应用于个性化医疗，其中利用成像数据来确定适合个体患者的最佳治疗方案。算法分析数据库中的相关信息以提供个性化的治疗建议，从而改善患者护理。 ➭治疗方法和药物 21世纪初开发的首批用于治疗湿性年龄相关性黄斑变性(AMD)的玻璃体内抗血管内皮生长因子(VEGF)药物是贝伐单抗(阿瓦斯汀，基因泰克公司)、雷珠单抗( Lucentis，基因泰克公司)。阿柏西普(艾力雅, 再生元制药公司)于2011年推出，随后2019年有布罗利珠单抗(Beovu，诺华制药公司)，2022年有法西单抗(Vabysmo，基因泰克公司)，2023年有高剂量8毫克制剂的阿柏西普。在此期间的其他进展包括生物类似药和药物递送系统（如眼内植入物、如Susvimo<雷珠单抗注射液，基因泰克公司>）的推出。这些创新旨在提高疗效并延长注射间隔时间。一种用于治疗地图样萎缩GA的新药Izervay（阿伐西普，安斯泰来制药公司）于2023年获批。虽然该药物不能阻止AMD终末期的进展，但可以减缓其进程。展望未来，正在研发中的新治疗策略旨在减轻湿性AMD患者的治疗负担。此外，基因疗法有望通过一次性治疗实现对湿性AMD的长期控制。干性AMD治疗的产品线正在扩大，包括针对地图样萎缩GA和Stargardt病的新候选药物。 ➭手术创新 FDA批准了RxSight公司的光可调人工晶状体和光输送装置，这是首个可在白内障手术后对人工晶状体的屈光度进行微小调整，以使患者在不戴眼镜时拥有更好视力的医疗装备系统。RxSight人工晶状体由一种对紫外线有反应的材料制成，术后17-21天由光输送装置输送紫外线。根据FDA新闻稿，患者在1至2周内接受三到四次光治疗，每次持续40至150秒，具体时间取决于调整程度。来源：瞳神书院Phoenix&眼界参考文献（向下滑动） 1.Hashemian H, Peto, T, Ambrosio Jr. R, 等人. 人工智能在眼科的应用：最新综合综述. 眼科与视觉研究杂志. 2024;；19:354–367. DOI: 10.18502/jovr.v19i3.15893. 2.李哲，王丽，吴旭，等. 眼科领域的人工智能：通往现实世界临床应用的道路. Cell Rep Med. 2023;；4:101095. DOI: 10.1016/j.xcrm.2023.101095. 3.刘X，Faes L，Kale AU，等. 深度学习与医疗专业人员在医学影像疾病检测中的表现比较：系统综述与荟萃分析. 《柳叶刀·数字健康》。 2019;1:e271–97。DOI：10.1016/S2589-7500(19)30123-2. 4.Maurya R, Vikal A, Narang RK, Patel P, Kurmi BD. 眼科基因治疗策略的最新进展与应用：眼科治疗学的突破。《实验眼科研究》2024;245:109983. https://doi.org/10.1016/j.exer.2024.109983. 5.科夫纳A. 美国食品药品监督管理局宣布批准基因疗法治疗遗传性视网膜营养不良，具有里程碑意义。美国眼科学会。 2017; 12月19日。https://www.aao.org/education/headline/fda-announces-landmark-approval-of-gene-therapy-in. 6.Russell S, Bennett J, Wellman JA, 等人。沃瑞替尼（AAV2-hRPE65v2）治疗RPE65介导的遗传性视网膜营养不良患者的疗效和安全性：一项随机、对照、开放标签、III期试验。《柳叶刀》。 2017;390:849–860. 7.White F. 俄勒冈健康与科学大学（OHSU）在人体内首次进行CRISPR基因编辑。俄勒冈健康与科学大学新闻。 2020; 发表于3月4日。https://news.ohsu.edu/2020/03/04/ohsu-performs-first-ever-crispr-gene-editing-within-human-body#:~:text=BRILLIANCE%20trial-,Andreas%20Lauer%2C%20M.D.%2C%20right%2C%20performs%20the%20first%2Dever,for%20the%20BRILLIANCE%20clinical%20trial. 8.皮尔斯·EA，阿莱曼·TS，贾亚桑德拉·KT，等. CEP290相关视网膜变性的基因编辑. 新英格兰医学杂志. 2024;；390:1972-1984. 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基因疗法临床研究

2026-01-07

·PRNewswire

Nanoscope Therapeutics Granted New U.S. Patent Covering Its Proprietary Multi-Characteristic Opsin Platform for Vision Restoration

DALLAS, Jan. 7, 2026 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, announced that the U.S. Patent and Trademark Office (USPTO) has granted U.S. Patent No. 12459977, recognizing the company's Multi-Characteristic Opsin (MCO) technology platform as a novel and innovative synthetic opsin for optogenetic modulation. The newly issued patent provides broad protection for methods of using the MCO platform to restore vision in patients who have lost photoreceptors due to retinal degenerative diseases such as retinitis pigmentosa , Stargardt disease, and geographic atrophy. In granting the patent, the USPTO acknowledged the distinctive attributes of Nanoscope's MCO platform, which differentiates it from other optogenetic approaches through its broad spectral sensitivity, fast kinetics, and ability to function via ambient light activation—eliminating the need for external light amplification devices. The patent extends intellectual property protection in the United States through 2039, with the potential for additional patent term extension following FDA approval of therapeutic products based on the technology. Nanoscope has also secured corresponding patents in Australia and Japan with patents in China and the EU pending, reinforcing its industry-leading, global intellectual property position in optogenetics and supporting long-term commercial exclusivity in major markets. "This patent represents more than an incremental IP milestone—it reinforces the strategic value of the MCO platform as a durable, first-in-class technology with broad clinical and commercial potential," said Sulagna Bhattacharya, CEO of Nanoscope Therapeutics. "This strengthens the foundation of our pipeline by extending market exclusivity, enhancing competitive barriers, and supporting multiple future indications built on a single, scalable optogenetic platform." The granting of this continuation-in-part patent further underscores the MCO platform's potential to deliver meaningful therapeutic benefit to patients suffering from severe vision loss, supporting Nanoscope's mission to restore sight to millions affected by retinal degenerative diseases for which no approved treatments currently exist. About the MCO Platform MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including Retinitis Pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO treatment does not require genetic testing, invasive surgery, or repeat dosing, enabling broad patient applicability within existing retina office workflows. About Nanoscope Therapeutics Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for retinitis pigmentosa (RP) (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) (NCT05417126) and plans to initiate a Phase 3 registrational trial in early 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. A Phase 2 program for MCO in geographic atrophy (GA) is expected to start in early 2026. Other IND-ready programs include Leber congenital amaurosis (LCA). Contact: Nanoscope Therapeutics (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics 21% more press release views with Request a Demo

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适应症	最高研发状态	国家/地区	公司	日期
视网膜色素变性	申请上市	美国	Nanoscope Therapeutics, Inc.	2025-07-14
斯塔加特病	临床2期	美国	Nanoscope Therapeutics, Inc.	2022-07-05
视网膜炎	临床2期	美国	Nanoscope Therapeutics, Inc.	2021-07-13
视网膜炎	临床2期	波多黎各	Nanoscope Therapeutics, Inc.	2021-07-13
莱伯先天性黑蒙	临床前	美国	Nanoscope Therapeutics, Inc.	2025-04-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


RESTORE (EURETINA2025)	临床2/3期	视网膜色素变性	27	MCO-010 low-dose	簾鹽網窪製網淵醖構淵(鏇淵衊糧願鹹淵淵膚築) = 窪網繭膚艱壓鬱獵網願襯膚鑰觸夢憲鬱窪構餘 (顧構淵襯鬱齋鹹選願窪, 0.14) 更多	积极	2025-09-04
				MCO-010 high-dose	簾鹽網窪製網淵醖構淵(鏇淵衊糧願鹹淵淵膚築) = 壓鹹遞顧鹹鬱艱網選製襯膚鑰觸夢憲鬱窪構餘 (顧構淵襯鬱齋鹹選願窪, 0.112) 更多
NCT04945772 (PRNewswire) 人工标引	临床2期	视网膜色素变性	-	MCO-010 gene therapy	製願醖繭艱繭簾繭蓋願(網鏇觸淵夢選壓構獵網) = 鹹膚製範繭簾獵膚醖鬱網蓋鹹構製選製鹹網築 (膚築壓鹽窪鹽蓋餘鑰廠 ) 更多	积极	2024-10-31
				sham-control	夢選艱膚鏇醖衊鹽壓觸(齋衊顧構糧艱顧蓋網觸) = 築糧製顧願鑰淵廠糧築簾簾鹹齋觸鹹顧願鬱繭 (憲獵製築壓蓋鹽範遞獵 ) 更多
NCT04945772 (RESTORE, Biospace) 人工标引	临床2期	视网膜色素变性	27	MCO-010 high dose	鑰廠糧艱鹹夢齋願願構(鹹夢衊鹹鏇遞鬱淵簾膚) = 窪艱齋衊襯獵齋繭築鑰選糧糧鬱蓋遞蓋襯夢蓋 (遞鹹構顧廠築顧構艱鬱, 0.0829) 更多	积极	2024-03-26
				MCO-010 low dose	鑰廠糧艱鹹夢齋願願構(鹹夢衊鹹鏇遞鬱淵簾膚) = 築鹽顧襯繭顧憲構願觸選糧糧鬱蓋遞蓋襯夢蓋 (遞鹹構顧廠築顧構艱鬱, 0.1244) 更多
NCT05417126 (STARLIGHT, ARVO2023) 人工标引	临床2期	斯塔加特病	6	MCO-010	構糧鏇鹹餘淵鹽憲夢餘(鹹淵鹹淵範壓鏇壓顧餘) = 顧淵衊簾觸構築遞願齋選齋襯積鏇醖遞夢鬱憲 (鹽構衊製獵鏇觸醖夢獵 ) 更多	积极	2023-06-01