$1M TRAP award to support preclinical safety study for gene therapy vector targeting rhodopsin- RHO-adRP

100万美元TRAP奖，用于支持针对视紫红质-RHO-adRP的基因治疗载体的临床前安全性研究

Additional project and operational funding to support MERTK gene therapy IND-enabling studies

额外的项目和运营资金，以支持MERTK基因治疗IND使能研究

RESEARCH TRIANGLE PARK, N.C., June 13, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs..

研究三角公园，北卡罗来纳州，2024年6月13日（环球通讯社）--Opus Genetics是一家以患者为先的临床阶段基因治疗公司，开发遗传性视网膜疾病的治疗方法，今天宣布它已从抗击失明基金会获得170万美元的项目资助，以帮助推进两个临床前候选项目。。

“Opus is immensely grateful for the generous support from the Foundation Fighting Blindness, which will catalyze our efforts in pioneering treatments for inherited retinal diseases. This $1.7 million funding infusion makes a significant impact to help accelerate the development of these two preclinical candidates,” said Ben Yerxa, Ph.D., chief executive officer of Opus.

Opus首席执行官Ben Yerxa博士说：“Opus非常感谢抗击失明基金会的慷慨支持，这将促进我们在开创性治疗遗传性视网膜疾病方面的努力。这170万美元的资金注入对帮助加速这两个临床前候选人的发展产生了重大影响。”。

“Together, we strive toward a future where therapies to help treat patients with inherited retinal diseases are readily available.”.

“我们共同努力，迈向一个有助于治疗遗传性视网膜疾病患者的疗法随时可用的未来。”。

Opus received a $1M Translational Research Acceleration Program (TRAP) award to conduct a preclinical safety study for a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP). The safety study will be conducted in the established canine animal model at the University of Pennsylvania School of Veterinary Medicine (Penn Vet).

Opus获得了100万美元的转化研究加速计划（TRAP）奖，用于针对视紫红质介导的常染色体显性视网膜色素变性（RHO-adRP）的基因治疗载体进行临床前安全性研究。安全性研究将在宾夕法尼亚大学兽医学院（Penn Vet）建立的犬类动物模型中进行。

The Company anticipates this is the last preclinical study required before the gene therapy will enter clinical trials. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone..

该公司预计这是基因治疗进入临床试验之前所需的最后一项临床前研究。RHO adRP是最常见的IRD之一，估计仅在美国就影响大约51000人中的一人，即6000多人。。

Additionally, Opus received approximately $700,000 in project-based and operational funding to support the preclinical development of a novel viral vector for treating retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene. The Company is collaborating with the Foundation to begin IND-enabling studies for a newly designed adeno-associated virus (AAV) viral vector to replace mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina.

此外，Opus获得了大约70万美元的项目和运营资金，用于支持一种新型病毒载体的临床前开发，该病毒载体用于治疗原癌基因酪氨酸蛋白激酶MER（MERTK）基因突变引起的视网膜色素变性。该公司正在与基金会合作，开始对新设计的腺相关病毒（AAV）病毒载体进行IND研究，以替代视网膜色素上皮（RPE）细胞中突变的MERTK基因。

The initial funding will provide the resources for testing the vector in an established animal model of the disease and to conduct early safety assessments in larger animals. MERTK mutations cause a rod-cone dystrophy with early macular atrophy, and retinitis pigmentosa is the most common retinal phenotype..

最初的资金将提供资源，用于在已建立的疾病动物模型中测试该病媒，并在较大的动物中进行早期安全评估。MERTK突变会导致视锥细胞营养不良并伴有早期黄斑萎缩，色素性视网膜炎是最常见的视网膜表型。。

“We're excited to announce our funding commitment to Opus, a trailblazer in inherited retinal disease therapeutics and a company created based on our mission of ultimately curing blindness caused by retinal degenerative diseases. This investment highlights our steadfast dedication to hastening innovative solutions for those combating inherited retinal diseases,” said Jason Menzo, chief executive officer of the Foundation Fighting Blindness.

“我们很高兴宣布我们对Opus的资助承诺，Opus是遗传性视网膜疾病治疗的开拓者，也是基于我们最终治愈视网膜退行性疾病引起的失明的使命而创建的一家公司。这项投资突显了我们坚定的致力于加速为那些抗击遗传性视网膜疾病的人提供创新解决方案，”抗击失明基金会首席执行官杰森·门佐（JasonMenzo）表示。

“In collaboration with Opus, we're propelling forward promising clinical candidate programs with the potential to revolutionize the lives of those affected by these challenging conditions.”.

“与Opus合作，我们正在推进有前途的临床候选项目，有可能彻底改变受这些挑战条件影响的人的生活。”。

About Foundation Fighting Blindness

关于基金会抗击失明

Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $816 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome.

抗击失明基金会成立于1971年，是世界上视网膜退行性疾病研究的主要私人资金来源。该基金会已经筹集了8.16亿美元用于加速研究，以预防，治疗和治疗由各种致盲性视网膜疾病引起的失明，包括色素性视网膜炎，黄斑变性和Usher综合征。

Visit FightingBlindness.org for more information..

有关更多信息，请访问FightingBlindness.org。。

About Opus Genetics

关于Opus Genetics

Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases.

Opus Genetics是一家针对遗传性视网膜疾病的临床阶段基因治疗公司，具有独特的模型和目的。在抗击失明基金会风险部门RD基金的支持下，Opus将无与伦比的洞察力和对患者需求的承诺与众多孤儿视网膜疾病的全资项目相结合。

Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients.

其基于AAV的基因治疗组合，包括目前正在进行1/2期临床试验的备受嘲笑的LCA5领先项目，解决了一些最被忽视的遗传性失明形式，同时创造了新的孤儿制造规模和效率。该公司位于北卡罗来纳州三角研究园，利用最佳科学知识和眼科基因治疗先驱的专业知识，透明地推动患者的变革性治疗。

For more information, visit www.opusgenetics.com..

For more information, visit www.opusgenetics.com..

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