BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases.

BlueRock Therapeutics LP 是一家临床阶段细胞治疗公司，也是拜耳股份公司全资拥有、独立运营的子公司，今天宣布美国食品药品监督管理局 (FDA) 批准其针对 OpCT-001 的新药临床试验 (IND) 申请，OpCT-001 是一种用于治疗原发性感光细胞疾病的在研诱导多能干细胞 (iPSC) 衍生细胞疗法。

“We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001," said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. "We believe that OpCT-001 has potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our Phase 1/2a clinical study.”

BlueRock Therapeutics 首席开发和医疗官 Amit Rakhit 表示：“我们对迄今为止取得的巨大进展感到高兴，并且很高兴 FDA 批准了我们的IND申请，以启动 OpCT-001 的临床试验。我们相信OpCT-001 有可能恢复原发性光感受器疾病患者的视力，并期待与眼科界合作启动我们的 1/2a 期临床研究。”

OpCT-001 is the first investigational iPSC-derived cell therapy to be clinically evaluated for the treatment of primary photoreceptor diseases. Initiation activities to support a Phase 1/2a study are underway. The Phase 1/2a study is designed as a first-in-human study to evaluate the safety and tolerability of subretinal administration of OpCT-001 in people with primary photoreceptor diseases as well as evaluate the effect of OpCT-001 on retinal structure, visual function and functional vision. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the U.S.

OpCT-001 是首个在临床上用于治疗原发性光感受器疾病的 iPSC 衍生细胞疗法。支持 1/2a 期研究的启动活动正在进行中。1/2a 期研究旨在作为首次人体研究，旨在评估 OpCT-001 在原发性光感受器疾病患者视网膜下注射的安全性和耐受性，以及评估 OpCT-001 对视网膜结构、视觉功能和功能性视力的影响。该研究将评估 OpCT-001 的几个剂量水平，预计将在美国各地招募参与者

Primary photoreceptor diseases are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. Primary photoreceptor diseases affect an estimated 110,0001 people in the U.S. and only limited treatment options exist for this population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated cells in the retina with functional cells.

原发性光感受器疾病是遗传性视网膜疾病的一个亚组，包括视网膜色素变性和视锥-视杆营养不良症。这些疾病会影响视网膜中光感受器细胞的结构和功能，导致儿童和成人出现不可逆的视力丧失。在美国，原发性光感受器疾病影响着约 110,000人，而这一人群的治疗选择有限。OpCT-001 旨在通过用功能性细胞替换视网膜中的退化细胞来恢复由这些疾病引起的视力丧失。

OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of the strategic R&D and clinical manufacturing collaboration between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001 at their cGMP2 facility in Madison, Wisconsin.

作为 BlueRock Therapeutics、FUJIFILM Cellular Dynamics 和 Opsis Therapeutics 于 2021 年建立的战略研发和临床制造合作的一部分，OpCT-001 于 2024 年 1 月从 FUJIFILM Cellular Dynamics 和 Opsis Therapeutics 获得独家许可。作为合作的一部分，FUJIFILM Cellular Dynamics 通过研究、开发和执行关键 IND 支持活动为 BlueRock Therapeutics 提供支持，包括在威斯康星州麦迪逊的 cGMP 2工厂进行 OpCT-001 的临床制造。

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，其功效和安全性尚未确定或得到充分评估。