Research Triangle Park, N.C. – August 20, 2024 – Opus Genetics has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for its ocular gene therapy, OPGx-LCA5, which aims to treat patients with the inherited retinal disease Leber congenital amaurosis type 5 (LCA5).

北卡罗来纳州三角研究公园（Research Triangle Park，N.C.）–2024年8月20日–Opus Genetics宣布，美国食品和药物管理局（FDA）已将罕见儿科疾病（RPD）指定为其眼部基因疗法OPGx-LCA5，旨在治疗遗传性视网膜疾病Leber先天性黑蒙5型（LCA5）患者。

OPGx-LCA5 utilizes an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina in patients with LCA5, a condition caused by biallelic mutations in the LCA5 gene..

OPGx-LCA5利用腺相关病毒8（AAV8）载体将功能性LCA5基因精确传递到LCA5患者的视网膜外，这种情况是由LCA5基因的双等位基因突变引起的。。

'We are thrilled to receive Rare Pediatric Disease designation from the FDA for our OPGx-LCA5 gene therapy,' said Ben Yerxa, Ph.D., Chief Executive Officer of Opus. 'This important milestone brings us closer to delivering a potential treatment for patients with LCA5. At Opus, we’re committed to advancing therapies that help treat patients with inherited retinal diseases, and this designation further validates the potential impact of our innovative ocular gene therapy approach.

Opus首席执行官BenYerxa博士说：“我们很高兴从FDA获得OPGx-LCA5基因治疗罕见儿科疾病的提名。”这一重要里程碑使我们更接近为LCA5患者提供潜在治疗。在Opus，我们致力于推进有助于治疗遗传性视网膜疾病患者的治疗方法，这一指定进一步验证了我们创新的眼部基因治疗方法的潜在影响。

We look forward to providing updates on the Phase 1/2 clinical trial evaluating OPGx-LCA5 soon.'.

我们期待着尽快提供评估OPGx-LCA5的1/2期临床试验的更新。”。

LCA5 is a form of early-onset retinal degeneration affecting approximately one in 1.7 million individuals in the United States. Currently, there are no approved treatments for LCA5-related vision loss. OPGx-LCA5 is being investigated in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania.

LCA5是一种早发性视网膜变性，在美国约有170万人中有一人受到影响。。OPGx-LCA5正在宾夕法尼亚大学进行一项开放标签的剂量递增1/2期临床试验。

This study is designed to assess the safety and preliminary efficacy of OPGx-LCA5 in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene..

这项研究旨在评估OPGx-LCA5在15例因LCA5基因双等位基因突变而遗传性视网膜变性患者中的安全性和初步疗效。。

For more information on the clinical trial, visit clinicaltrials.gov (NCT05616793)

有关临床试验的更多信息，请访问clinicaltrials.gov（NCT05616793）

The FDA's Rare Pediatric Disease designation is awarded to therapeutics aimed at treating serious or life-threatening rare diseases that primarily affect individuals under 18 years old. With this designation, Opus gains access to valuable incentives and support from the FDA throughout its development program.

美国食品和药物管理局（FDA）的罕见儿科疾病称号授予旨在治疗主要影响18岁以下个体的严重或危及生命的罕见疾病的治疗药物。通过这一指定，Opus在其整个发展计划中获得了FDA的宝贵激励和支持。

Additionally, OPGx-LCA5 will be eligible to receive a priority review voucher upon approval of any subsequent marketing application. This voucher can be sold or transferred to other companies..

此外，OPGx-LCA5在任何后续营销申请获得批准后，将有资格获得优先审查凭证。此凭证可以出售或转让给其他公司。。

About OPGx-LCA5

关于OPGx-LCA5

OPGx-LCA5 is a gene therapy designed to address Leber congenital amaurosis type 5 (LCA5), a severe early-onset inherited retinal dystrophy caused by biallelic mutations in the LCA5 gene. The LCA5 gene encodes the protein lebercilin. Research on LCA5 patients has shown a dissociation between retinal architecture and visual function, highlighting a potential for therapeutic intervention through gene augmentation..

OPGx-LCA5是一种基因疗法，旨在解决Leber先天性5型黑蒙（LCA5），这是一种严重的早发性遗传性视网膜营养不良，由LCA5基因的双等位基因突变引起。LCA5基因编码蛋白质lebercilin。对LCA5患者的研究表明，视网膜结构与视觉功能之间存在分离，突出了通过基因增强进行治疗干预的潜力。。

OPGx-LCA5 employs an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the outer retina. Currently, OPGx-LCA5 is under evaluation in a Phase 1/2 clinical trial at the University of Pennsylvania, aimed at determining its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene..

OPGx-LCA5使用腺相关病毒8（AAV8）载体将功能性LCA5基因传递到外部视网膜。目前，OPGx-LCA5正在宾夕法尼亚大学进行1/2期临床试验，旨在确定其对LCA5基因双等位基因突变导致的遗传性视网膜变性患者的安全性和初步疗效。。