– Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced that the first patient received the investigational therapy in CLARICO, a Phase 1/2a clinical trial of OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases.

拜耳公司和其全资子公司、临床阶段细胞治疗公司 BlueRock Therapeutics LP 今天宣布，在 CLARICO 一期/2a 期临床试验中，首位患者已接受了 OpCT-001 的研究性治疗。OpCT-001 是一种研究性的诱导多能干细胞（iPSC）衍生细胞疗法，用于治疗原发性光感受器疾病。

OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor (PR) diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy..

OpCT-001是首个用于治疗原发性光感受器（PR）疾病的临床测试的iPSC衍生研究性细胞疗法，这类疾病属于遗传性视网膜疾病的一个亚组，包括视网膜色素变性和锥杆细胞营养不良症。

“The initiation of the CLARICO trial represents a key milestone for the OpCT-001 program,” said Amit Rakhit, MD, MBA, BlueRock’s Chief Development and Medical Officer. “We believe OpCT-001 holds significant promise as a novel therapeutic approach for restoring vision in people living with primary photoreceptor diseases, and we look forward to assessing its safety and tolerability profile as we advance this important program in our pipeline.”.

“CLARICO 试验的启动代表了 OpCT-001 项目的一个关键里程碑，”BlueRock 的首席开发和医学官 Amit Rakhit 医学博士、工商管理硕士说道。“我们相信 OpCT-001 作为一种恢复原发性光感受器疾病患者视力的创新治疗手段具有重大潜力，我们期待在推进这一重要项目的进程中评估其安全性和耐受性。”

CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and includes a dose-escalation design.

CLARICO 是一项 Phase 1/2a 期首次人体、多中心、两部分的干预性研究，旨在评估 OpCT-001 在约 54 名原发性光感受器疾病成人患者中的安全性、耐受性及其对临床结果的影响。Phase 1 将专注于安全性，并包含剂量递增设计。

Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups..

第二阶段旨在收集更多的安全数据，并评估OpCT-001对视觉功能、功能性视力以及不同临床亚组中植入解剖指标的影响。

“We aim to transform treatment options for patients facing irreversible vision loss. OpCT-001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease,” said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer.

“我们的目标是为面临不可逆视力丧失的患者改变治疗选择。OpCT-001是我们正在研究的诱导多能干细胞衍生细胞疗法，有潜力恢复原发性光感受器疾病患者的视力，”拜耳制药部门执行副总裁兼全球研发主管克里斯蒂安·罗梅尔表示。

“We are excited to announce the first patient in the CLARICO trial, the first-ever clinical trial for an iPSC-derived treatment in this field.”.

“我们很高兴地宣布，CLARICO 试验中的首位患者已经入组，这是该领域首个使用 iPSC 衍生疗法的临床试验。”

Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells.

原发性光感受器疾病影响视网膜中光感受器细胞的结构和功能，导致儿童和成人不可逆的视力丧失。OpCT-001旨在通过用功能性细胞替换视网膜中退化的细胞，恢复原发性光感受器疾病患者的视力。

Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000 people in the U.S..

目前，针对影响美国约 11 万人的原发性光感受器疾病的治疗选择有限。

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

OpCT-001 是一种研究性细胞疗法，尚未获得任何监管机构的批准，其有效性和安全性尚未确立或完全评估。

About CLARICO

关于CLARICO

CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and features a dose-escalation design.

CLARICO是一项1/2a期首次人体、多中心、两部分的干预性研究，旨在评估OpCT-001在多达约54名原发性光感受器疾病成人患者中的安全性、耐受性及其对临床结果的影响。第1阶段将专注于安全性，并采用剂量递增设计。

Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4).

第二阶段旨在收集更多的安全数据，并评估OpCT-001对不同临床亚组视觉功能、功能性视力和解剖学植入指标的影响。第一阶段将包括4个计划剂量水平，分4个队列（第1至第4队列）进行给药。

Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants in 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data.

第一阶段的剂量递增将采用标准的3+3方案，总共12至24名法律定义上的盲人参与者（每组约3至6人）将接受OpCT-001。第二阶段计划在两个队列（第5组和第6组）中最多招募15名参与者，以评估根据第一阶段安全性和耐受性数据选出的两种OpCT-001剂量水平。

Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. Further details of the trial can be found at .

第二阶段的参与者将被随机分配到队列5或队列6，比例为1:1。第二阶段的参与者以及外科团队之外的研究者/研究中心人员将对OpCT-001的剂量分配保持盲态。试验的更多细节可以在以下位置找到。

About BlueRock Therapeutics LP

关于 BlueRock Therapeutics LP

BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs.

BlueRock Therapeutics LP是一家临床阶段的细胞治疗公司，专注于开发细胞药物以治疗严重疾病。我们正在利用细胞疗法的力量，为患有神经和眼科疾病的患者创建一系列新药。我们的两种新型研究性细胞疗法，用于治疗帕金森病的bemdaneprocel（BRT-DA01）和用于治疗原发性光感受器疾病的OpCT-001，目前都处于临床阶段。

Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase 3 clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase 1 clinical trial, Clarico.

贝姆达内普罗塞尔获得了美国食品药品监督管理局（FDA）的RMAT（再生医学高级疗法）和快速通道资格认定，正在第三阶段临床试验exPDite-2中进行测试。OpCT-001获得了FDA的快速通道资格认定，正在第一阶段临床试验Clarico中进行测试。

BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves.

BlueRock 成立于2016年，是Versant Ventures和拜耳旗下影响力投资部门Leaps的合资企业，该部门致力于投资能够带来范式转变的突破性创新。2019年底，BlueRock成为拜耳集团全资拥有的独立运营子公司。我们的文化以无畏挑战的勇气、变革医学和传递希望的紧迫感、由使命引导的诚信，以及对社区的责任感为核心，深知我们每个人都是比自身更宏大事业的一部分。

About Bayer

关于拜耳

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.

拜耳是一家在医疗保健和营养等生命科学领域具有核心竞争力的全球性企业。秉承“人人健康，无饥饿”的使命，公司通过支持应对不断增长和老龄化的全球人口所带来的重大挑战，设计其产品和服务以帮助人类和地球繁荣发展。

Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros.

拜耳致力于通过其业务推动可持续发展并产生积极影响。同时，集团旨在通过创新和增长提高盈利能力并创造价值。拜耳品牌在全球范围内代表信任、可靠性和质量。在2024财年，该集团拥有约93,000名员工，销售额达到466亿欧元。