Opus Genetics Reports 1-Month Pediatric Results for OPGx-LCA5 Gene Therapy in Inherited Retinal Disease

Opus遗传学公司报告了OPGx-LCA5基因疗法在遗传性视网膜疾病中的1个月儿科结果

April 09, 2025

2025年4月9日

Opus Genetics has reported new 1-month clinical data from the first pediatric patients treated with its investigational gene therapy candidate, OPGx-LCA5, for Leber congenital amaurosis (LCA5)-related inherited retinal disease (IRD). This rare, early-onset retinal condition currently has no approved treatments and is associated with severe vision loss from a young age..

Opus Genetics 报告了其在研基因疗法候选药物 OPGx-LCA5 治疗与 Leber 先天性黑蒙 (LCA5) 相关的遗传性视网膜疾病 (IRD) 的首例儿科患者的新 1 个月临床数据。这种罕见的早发性视网膜疾病目前尚无获批的治疗方法，并且与从小严重视力丧失相关。

Early Signs of Efficacy in Pediatric Cohort

儿科队列中疗效的早期迹象

These initial pediatric findings build on previously reported 6- and 12-month results in adult participants, which demonstrated durable improvements in visual function. The first pediatric patient, a 16-year-old, received a single subretinal injection of OPGx-LCA5. Within just one month, the patient reported brighter vision and showed new levels of independence in reading and mobility—indicating a clinically meaningful improvement in visual function.

这些初步的儿科研究结果建立在之前报告的成年参与者6个月和12个月的研究结果之上，这些结果显示了视觉功能的持久改善。第一名儿科患者是一名16岁的青少年，接受了单次视网膜下注射OPGx-LCA5。仅一个月内，该患者报告视力变得更明亮，并在阅读和移动方面表现出新的独立性，这表明视觉功能有了临床意义上的改善。

Importantly, no drug-related adverse events were observed..

重要的是，未观察到与药物相关的不良事件。

A second pediatric patient has recently been treated, and full enrollment of the pediatric cohort is expected to be completed by the second quarter of 2025. Initial data from all three pediatric participants are anticipated by the third quarter of 2025.

近日，第二位儿科患者已接受治疗，预计将在2025年第二季度完成儿科队列的全部入组。预计在2025年第三季度获得所有三名儿科参与者初始数据。

Advancing Pediatric Gene Therapy for Inherited Retinal Disease

推进儿科遗传性视网膜疾病的基因治疗

Opus Genetics emphasized the potential of gene therapy to restore vision before irreversible retinal damage occurs, particularly when applied in pediatric patients. The early data signals not only the therapeutic potential of OPGx-LCA5 but also the value of intervening during the early stages of retinal degeneration..

Opus Genetics 强调了基因疗法在不可逆的视网膜损伤发生之前恢复视力的潜力，特别是在儿科患者中的应用。早期数据不仅表明了 OPGx-LCA5 的治疗潜力，还显示了在视网膜退化的早期阶段进行干预的价值。

Study investigator Dr. Tomas Aleman of the Scheie Eye Institute noted that the observed visual improvements are consistent with earlier adult data. He described how the treated patient was able to distinguish letters and navigate with a new level of independence just one-month post-treatment, reinforcing the importance of early intervention in gene therapy trials..

斯凯伊眼科研究所的研究员托马斯·阿莱曼博士指出，观察到的视力改善与早期的成人数据一致。他描述了接受治疗的患者在治疗后仅一个月内就能够分辨字母并以新的独立性进行导航，这进一步强调了在基因治疗试验中早期干预的重要性。

Building Toward a Registrational Trial

建立注册试验的方向

The company recently conducted a Type D meeting with the U.S. Food and Drug Administration (FDA) to discuss the regulatory pathway for OPGx-LCA5. During this meeting, Opus proposed the design of a single-arm, adaptive pivotal trial that could enroll as few as 19 patients. The proposed primary endpoint for this study is the multi-luminance orientation and mobility test (MLoMT), a virtual-reality-based functional vision test that has previously supported FDA approvals in similar therapeutic contexts..

该公司最近与美国食品药品监督管理局 (FDA) 举行了一次D类会议，讨论OPGx-LCA5的监管路径。在此次会议上，Opus提出了一个单臂、适应性关键试验的设计方案，该试验可能仅需招募19名患者。这项研究的主要终点设定为多亮度定向移动测试 (MLoMT)，这是一种基于虚拟现实的功能性视力测试，此前已在类似的治疗背景下支持了FDA的审批。

The FDA provided constructive feedback on Opus’s proposed statistical analysis plan and its approach to chemistry, manufacturing, and controls (CMC). While the agency requested additional data on certain components, Opus stated that it will submit further materials and continue ongoing dialogue with the FDA.

美国食品药品监督管理局（FDA）对Opus提出的统计分析计划及其在化学、制造和控制（CMC）方面的做法提供了建设性的反馈。尽管该机构要求提供某些组件的更多数据，但Opus表示将提交更多材料，并继续与FDA保持持续对话。

The company anticipates that the pivotal trial could begin in the first quarter of 2026, pending regulatory alignment..

公司预计，关键试验可能于 2026 年第一季度开始，具体取决于监管部门的批准。

Upcoming Scientific Presentation

即将举行的科学报告

Looking ahead, Opus Genetics will present the 1-year data from its ongoing study at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting on May 4, 2025. These long-term results are expected to provide further insight into the efficacy, safety, and durability of OPGx-LCA5 across both pediatric and adult populations..

展望未来，Opus Genetics 将于2025年5月4日在视觉与眼科研究协会（ARVO）年会上展示其正在进行的研究的1年数据。这些长期结果预计将为进一步了解OPGx-LCA5在儿童和成人人群中的有效性、安全性和持久性提供更多信息。

“Early intervention may be particularly beneficial in pediatric patients, given the progressive nature of this disease,” said George Magrath, MD, CEO of Opus Genetics. “We believe these findings provide further evidence supporting the potential of OPGx-LCA5 to restore meaningful vision in patients affected by LCA5.”.

“鉴于这种疾病的进展性，早期干预可能对儿科患者特别有益，” Opus Genetics 首席执行官乔治·马格拉思 (George Magrath) 医学博士说道。“我们认为这些发现为进一步证明 OPGx-LCA5 在恢复 LCA5 患者有意义的视力方面的潜力提供了更多证据。”