MALVERN, Pa., June 20, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400—a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP)..

宾夕法尼亚州马尔文，2024年6月20日（环球通讯社）--Ocugen，Inc.（“Ocugen”或“公司”）（纳斯达克：OCGN），一家专注于发现、开发和商业化新型基因和细胞疗法及疫苗的生物技术公司，今天宣布，第一名患者已在其OCU400的第三阶段引人注目的临床试验中服用，该试验是针对色素性视网膜炎（RP）开发的修饰基因治疗候选产品。。

“Each clinical milestone achieved by OCU400 brings us closer to providing a one-time treatment for life to patients living with RP,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “Dosing the first patient is especially significant and makes our dedication to serving RP patients—300,000 in the U.S.

Ocugen董事长、首席执行官兼联合创始人Shankar Musunuri博士说：“OCU400实现的每个临床里程碑都使我们更接近于为RP患者提供一次性终身治疗。”。“给第一位患者服用药物特别重要，这使我们致力于为RP患者提供服务，在美国有30万人。

and Europe and 1.6 million worldwide—more tangible.”.

欧洲和全世界160万人更加有形。”。

The Phase 3 liMeliGhT clinical trial was informed by positive Phase 1/2 OCU400 data that suggests positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. 89% (16/18) of RP subjects demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline.

3期聚光灯临床试验由阳性的1/2期OCU400数据提供信息，这些数据表明治疗眼的最佳矫正视力（BCVA）和多亮度活动度测试（MLMT）以及低亮度视力（LLVA）呈阳性趋势。89%（16/18）的RP受试者在BCVA或LLVA或MLMT评分方面表现出治疗眼的保存或改善。

80% (8/10) of RHO mutation subjects experienced either preservation or improvement in MLMT scores from baseline. 78% (14/18) of subjects demonstrated preservation or improvement in treated eyes in MLMT scores from baseline..

80%（8/10）的RHO突变受试者的MLMT评分从基线保持或改善。78%（14/18）的受试者在MLMT评分中表现出治疗后眼睛的保存或改善。。

The Phase 3 study—with the duration of one year—will have a sample size of 150 participants—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

。在每只手臂中，参与者将分别以2:1的比例随机分配到治疗组（OCU400的2.5 x 1010 vg/眼）和未治疗的对照组。

Patients eight years of age and older, with early through late-stage advancement of RP, are being recruited to participate in the liMeliGhT study..

正在招募八岁及以上RP早期至晚期进展的患者参加这项备受关注的研究。。

Luminance Dependent Navigation Assessment (LDNA)—a more sensitive and specific measurement of function than MLMT used in previous Phase 3 clinical trials—is the primary endpoint for the study. The Phase 3 liMeliGhT study will focus on the proportion of responders, in treated and untreated groups, achieving an improvement of at least 2 Lux levels from baseline in the study eyes..

亮度依赖性导航评估（LDNA）-一种比以前的3期临床试验中使用的MLMT更敏感和更具体的功能测量，是该研究的主要终点。第三阶段聚光灯研究将重点关注治疗组和未治疗组的应答者比例，使研究眼中的基线水平至少提高2勒克斯。。

“Patients with RP associated with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit to RP patients,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen.

“与多基因突变相关的RP患者目前没有治疗选择。作为一名视网膜外科医生，我对OCU400的治疗潜力感到鼓舞，可以为RP患者提供长期益处，”杜克大学眼科中心眼科和儿科玻璃体视网膜手术和疾病副教授、杜克大学眼科中心视网膜科学咨询委员会主席Lejla Vajzovic博士说。

“OCU400 is a novel modifier gene therapy approach that could initiate a paradigm shift in the treatment of RP and to field of ophthalmology.”.

“OCU400是一种新型的修饰基因治疗方法，可以启动RP治疗和眼科领域的范式转变。”。

“The current OCU400 Phase 3 study is very exciting and gives hope for thousands of individuals with RP,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants (ARC) and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. “I am encouraged that we may have a potential treatment option to preserve or improve the vision in RP patients regardless of gene mutation, and very pleased that the first patient dosing in the Phase 3 liMeliGhT clinical trial was performed at ARC.”.

“目前的OCU400 3期研究非常令人兴奋，为数千名RP患者带来了希望，”Benjamin Bakall博士说，他是视网膜咨询公司（ARC）临床研究主任，也是亚利桑那大学凤凰城医学院临床助理教授。“我感到鼓舞的是，无论基因突变如何，我们都可能有一种潜在的治疗选择来保持或改善RP患者的视力，并且非常高兴在ARC进行了3期聚光灯临床试验中的第一次患者给药。”。

“We are grateful for our continued collaboration with Dr. Bakall and the team at ARC,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. 'We are excited to expand our enrollment to include more centers and patients representing a diverse array of RP gene mutations, which will be a validation of this novel gene therapy platform.

Ocugen首席医疗官Huma Qamar博士说：“我们非常感谢与Bakall博士和ARC团队的持续合作。”我们很高兴扩大招生范围，以包括更多代表各种RP基因突变的中心和患者，这将是对这种新型基因治疗平台的验证。

We will provide updates as our progress continues.'.

。

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA and that the EMA provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA). With the first dosing of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA and MAA approval targets..

Ocugen先前宣布，OCU400已获得FDA的孤儿药和RMAT指定，并且EMA提供了美国试验提交上市授权申请（MAA）的可接受性。随着3期临床试验的首次给药，OCU400仍在2026年BLA和MAA批准目标的轨道上。。

About OCU400

关于OCU400

OCU400 is the Company’s gene-agnostic modifier gene therapy product based on nuclear hormone receptor (NHR) gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.

OCU400是该公司基于核激素受体（NHR）基因NR2E3的基因不可知修饰基因治疗产品。NR2E3调节视网膜内的多种生理功能，例如感光细胞的发育和维持，代谢，光转导，炎症和细胞存活网络。

Through its drive functionality, OCU400 resets altered/affected cellular gene networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with RP..

通过其驱动功能，OCU400重置了改变/受影响的细胞基因网络，并建立了稳态-一种平衡状态，有可能改善RP患者的视网膜健康和功能。。

About Modifier Gene Therapy

关于修饰基因疗法

Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA and Stargardt disease, as well as multifactorial diseases like dry age-related macular degeneration (dAMD). Our modifier gene therapy platform is based on the use of NHRs, master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina.

。我们的修饰基因治疗平台基于NHR的使用，NHR是主要的基因调节剂，具有恢复稳态的潜力-视网膜的基本生物过程。

Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks.

与仅针对一种基因突变的单基因替代疗法不同，我们认为我们的修饰基因治疗平台通过使用NHR代表了一种新方法，有可能解决由一种产品的多个基因突变引起的多种视网膜疾病，并解决可能由多基因网络失衡引起的复杂疾病。

Currently, Ocugen has three modifier gene therapy programs in the clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400 Phase 3 liMeliGhT clinical trial, the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA) secondary to dAMD and the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease are currently underway.

目前，Ocugen在临床上有三个修饰基因治疗程序：OCU400，OCU410和OCU410ST。除了OCU400 3期聚光灯临床试验外，目前正在进行继发于dAMD的地理萎缩（GA）的OCU410 1/2期ArMaDa临床试验和Stargardt病的OCU410ST 1/2期GARDian临床试验。

GA affects approximately two to three million people in the U.S. and EU combined and Stargardt disease affects nearly 100,000 people in the U.S. and EU combined..

GA在美国和欧盟总共影响了大约200万至300万人，Stargardt病在美国和欧盟总共影响了近10万人。。

About Ocugen, Inc.

关于Ocugen，Inc。

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital.

Ocugen，Inc.是一家生物技术公司，专注于发现、开发和商业化新型基因和细胞疗法以及疫苗，以改善健康并为全球患者带来希望。我们正在通过勇敢的创新创造新的科学途径，利用我们独特的智力和人力资本，对患者的生活产生影响。

Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn..

我们突破性的修饰基因治疗平台有可能用单一产品治疗多种视网膜疾病，我们正在推进传染病研究，以支持公共卫生和骨科疾病，以解决未满足的医疗需求。在www.ocugen.com上了解更多信息，并在X和LinkedIn上关注我们。。