Opus Genetics Doses First Pediatric Patient in Phase 1/2 Trial for OPGx-LCA5 Gene Therapy

OPGx-LCA5基因治疗的1/2期试验中，Opus Genetics为首位儿科患者给药

February 21, 2025

2025年2月21日

Opus Genetics has reached a significant milestone in its ongoing Phase 1/2 clinical trial for OPGx-LCA5, an investigational gene therapy for Leber congenital amaurosis (LCA). The company announced that the first pediatric patient has been dosed, with initial data from this cohort expected by Q3 2025..

Opus Genetics在其针对Leber先天性黑蒙（LCA）的在研基因疗法OPGx-LCA5的持续1/2期临床试验中达到了一个重要里程碑。公司宣布，第一名儿童患者已经接受给药，预计将在2025年第三季度获得该队列的初步数据。

Promising Early Results in Adult Patients

成人患者早期结果令人鼓舞

The trial has already demonstrated early clinical proof of concept, with meaningful visual improvement observed in adult patients as early as one month post-treatment.

该试验已经展示了早期的临床概念验证，早在治疗后一个月，成年患者就观察到了有意义的视力改善。

Key findings from the trial include:

审判的主要发现包括：

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Six-month data, released in October 2024, confirmed significant visual improvements.

2024年10月发布的六个月数据显示，视力显著改善。

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Twelve-month data on adult LCA5 patients will be presented at a major medical conference in Q2 2025.

成年LCA5患者为期十二个月的数据将在2025年第二季度的一个重要医学会议上展示。

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An FDA Type D meeting is scheduled for March 2025 to discuss the pivotal trial design and registrational endpoints.

计划于2025年3月举行FDA D类会议，讨论关键试验设计和注册终点。

'We are proud to expand the OPGx-LCA5 clinical trial to pediatric patients. Early intervention is crucial to preserving or restoring vision before disease progression,” said George Magrath, MD, CEO of Opus Genetics

“我们很自豪能够将OPGx-LCA5临床试验扩展到儿科患者。早期干预对于在疾病进展之前保护或恢复视力至关重要，” Opus Genetics首席执行官George Magrath博士说道。

Dr. Magrath also emphasized that the durability of positive responses observed at six and twelve months will be a key focus in upcoming FDA discussions regarding a potential Phase 3 trial.

麦格拉斯博士还强调，将在接下来的FDA讨论中重点关注六个月和十二个月时观察到的阳性反应的持久性，以探讨潜在的第三阶段试验。

Phase 1/2 Trial Design and Key Efficacy Endpoints

1/2期试验设计和关键疗效终点

The open-label Phase 1/2 trial evaluates the safety and preliminary efficacy of subretinal gene therapy with OPGx-LCA5 in patients with biallelic LCA5 gene mutations.

开放标签的1/2期试验评估了OPGx-LCA5视网膜下基因治疗在双等位LCA5基因突变患者中的安全性和初步疗效。

Efficacy endpoints include:

疗效终点包括：

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Multi-Luminance Orientation and Mobility Test (MLoMT) to measure functional vision.

多亮度定向与移动测试（MLoMT）用于测量功能性视力。

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Full-Field Stimulus Testing (FST) to assess retinal sensitivity to light.

全视野刺激测试 (FST) 以评估视网膜对光的敏感性。

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Microperimetry to evaluate point-wise sensitivity to light.

微视野检查以评估对光的点敏感性。

Advancing Toward a Potential Treatment for LCA5

迈向LCA5潜在治疗的进展

Opus Genetics continues to work closely with the University of Pennsylvania, regulatory agencies, the medical community, and patient advocates to advance this promising therapy.

Opus Genetics 继续与宾夕法尼亚大学、监管机构、医学界和患者倡导者密切合作，以推进这一有前景的疗法。

With encouraging early results and the expansion of the trial to pediatric patients, OPGx-LCA5 has the potential to become a transformative treatment for individuals with LCA5-related vision loss.

随着令人鼓舞的早期结果以及试验扩展到儿科患者，OPGx-LCA5 有可能成为治疗 LCA5 相关视力丧失患者的变革性疗法。