– BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced the publication of the 18-month data from its Phase 1 exPDite clinical trial for bemdaneprocel in the journal

– BlueRock Therapeutics LP是一家处于临床阶段的细胞治疗公司，也是拜耳股份公司（Bayer AG）的全资子公司，今天宣布其在期刊上发表了关于bemdaneprocel的1期exPDite临床试验的18个月数据。

Nature.

自然。

“The concept of “rebuilding” brain networks that have been lost to disease is compelling,” said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine’s Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators, “The results of this early phase clinical trial demonstrate the promise of regenerative medicine and should provide hope for Parkinson’s disease patients and their families.” .

“‘重建’因疾病而丧失的脑网络这一概念非常引人注目，”加州大学欧文分校医学院神经病学系主任、加州大学欧文分校的克莱尔·亨奇克利夫医学博士说道，她也是这项研究的主要研究者之一，“这项早期临床试验的结果展示了再生医学的潜力，应为帕金森病患者及其家属带来希望。”

The publication, which is available

该出版物现已可供使用

online

在线

, confirms that 18 months following surgery, treatment with bemdaneprocel did not cause any serious adverse events related to cell therapy. In addition, positron emission tomography (PET) imaging techniques demonstrated sustained neuron cell engraftment in the trial’s high and low dose cohorts following discontinuation of immunosuppression after one year post-treatment.

，证实了在手术后18个月，使用bemdaneprocel治疗没有引发任何与细胞疗法相关的严重不良事件。此外，正电子发射断层扫描（PET）成像技术显示，在治疗一年后停止免疫抑制后，试验的高剂量和低剂量组中神经元细胞植入仍然持续存在。

The publication also reports encouraging trends in secondary and exploratory subjective and objective endpoints related to motor function. These data were first presented in March 2024 at the Alzheimer’s and Parkinson’s Diseases Conference in Lisbon, Portugal.  .

该出版物还报告了与运动功能相关的次要和探索性主观及客观终点的鼓舞人心的趋势。这些数据于 2024 年 3 月在葡萄牙里斯本举行的阿尔茨海默病和帕金森病会议上首次公布。

“Cell therapy is a potential new treatment option for individuals with Parkinson's disease and our team is immensely proud of having bemdaneprocel’s 18-month Phase 1 data published in such a prestigious journal as

“细胞疗法是帕金森病患者潜在的新治疗选择，我们的团队为 bemdaneprocel 的 18 个月第一阶段数据能够发表在如此权威的期刊上感到无比自豪。

Nature

自然

,” said Amit Rakhit, MD, MBA Head of Development and Chief Medical Officer at BlueRock Therapeutics. “Our efforts are now focused on maintaining our forward momentum to initiate and enroll the Phase 3 trial as we advance bemdaneprocel’s development to this exciting next stage.”

”BlueRock Therapeutics的开发主管兼首席医疗官Amit Rakhit医学博士、工商管理硕士说道，“我们的努力现在集中在保持前进的动力，以启动并招募第3阶段试验，因为我们正在将bemdaneprocel的开发推进到这个令人兴奋的下一阶段。”

The Phase 3 trial, called exPDite-2, is expected to be initiated in the first half of 2025.

名为exPDite-2的III期试验预计将于2025年上半年启动。

About bemdaneprocel (BRT-DA01)

关于bemdaneprocel（BRT-DA01）

Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from human embryonic stem cells, developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease.

Bemdaneprocel（BRT-DA01）是一种研究性细胞疗法，旨在替代帕金森病中丧失的多巴胺生成神经元。这些多巴胺能神经前体细胞来源于人类胚胎干细胞，在植入后会发育为成熟的多巴胺神经元。在手术过程中，这些神经前体细胞被植入帕金森病患者的大脑中。

When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson’s disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track designation followed in 2024 by a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.

当移植后，它们有可能重新形成受到帕金森病严重影响的神经网络，并有可能恢复患者的运动和非运动功能。2021年，bemdaneprocel获得快速通道资格，随后在2024年获得美国食品药品监督管理局（FDA）的再生医学先进疗法（RMAT）资格。

Data from the Phase 1 trial’s 12 participants presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery.

2024年国际帕金森病与运动障碍大会（MDS）上公布的1期试验12名参与者的数据表明，术后24个月药物产品具有良好的耐受性，无严重不良事件发生。此外，在术后24个月与运动功能障碍相关的次要终点观察到了令人鼓舞的趋势。

These participants remain in a long-term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority. .

这些参与者仍处于长期的持续评估研究中。Bemdaneprocel 尚未被任何卫生当局批准用于治疗任何疾病或医疗状况。

About Parkinson’s disease

关于帕金森病

Parkinson’s disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person’s daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement.

帕金森病（PD）是一种进展性的神经退行性疾病。它对患者的日常生活有显著影响。在帕金森病中，大脑中产生多巴胺的神经细胞死亡导致运动功能的持续丧失。症状包括震颤、肌肉僵硬和运动迟缓。

Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD.

此外，帕金森病患者还会经历非运动症状，包括疲劳、缺乏精力、认知问题和抑郁。症状通常会随着时间的推移而加重，使日常任务变得困难。在过去的25年中，帕金森病的患病率翻了一番。如今，全球估计有超过1000万人患有帕金森病。

This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms so new therapies are needed. .

这使得它成为世界上第二常见的神经退行性疾病，也是最常见的运动障碍。目前尚无治愈方法，现有的治疗方案缺乏对症状的整体管理，因此需要新的治疗方法。

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, ophthalmic, cardiovascular and immunological diseases.

BlueRock Therapeutics LP 是一家临床阶段的细胞治疗公司，专注于创造细胞药物来治疗毁灭性疾病。我们正在利用细胞治疗的力量，为遭受神经、眼科、心血管和免疫疾病困扰的患者开发一系列新药。

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 disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is expected to begin a Phase 3 clinical trial in H1 2025.

我们两款新型研究性细胞疗法，用于治疗帕金森病的bemdaneprocel（BRT-DA01）和用于治疗原发性视杆细胞疾病的OpCT-001，已进入临床阶段。Bemdaneprocel获得了美国FDA（食品药品监督管理局）的RMAT（再生医学先进疗法）和快速通道资格，预计将于2025年上半年开始进行III期临床试验。

OpCT-001 is expected to begin Phase 1 clinical testing in H1 2025. 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 as a cornerstone of its newly formed Cell & Gene Therapy platform.

OpCT-001预计将于2025年上半年开始一期临床试验。BlueRock成立于2016年，是Versant Ventures和拜耳旗下影响力投资部门Leaps共同创立的合资企业，该部门专注于投资具有颠覆性创新的突破性技术。2019年底，BlueRock成为拜耳集团新成立的细胞与基因治疗平台的基石，并作为其全资拥有且独立运营的子公司。

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. For more information, visit .

我们的文化定义为：无论面对何种挑战，都有坚持的勇气；有变革医学、传递希望的紧迫感；有使命指引的诚信；以及心怀社区的意识，理解到我们都是比自身更宏大事业的一部分。欲了解更多信息，请访问。

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，无人饥饿”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着信任、可靠性和质量。在2024财年，集团拥有约93,000名员工，销售额达466亿欧元。