BlueRock Therapeutics的研究性细胞疗法 bemdaneprocel用于治疗帕金森病，获得日本先锋再生医学产品称号-动脉网

BlueRock Therapeutics investigative cell therapy bemdaneprocel for treating Parkinson’s disease receives Pioneering Regenerative Medical Product designation in Japan

拜耳等信源发布 2025-12-17 15:35

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December

十二月

2025

08:30 AM

上午 08:30

Europe/Amsterdam

欧洲/阿姆斯特丹

BlueRock Therapeutics investigative cell therapy bemdaneprocel for treating Parkinson’s disease receives Pioneering Regenerative Medical Product designation in Japan

BlueRock Therapeutics 的研究性细胞疗法 bemdaneprocel 用于治疗帕金森病，获得日本先锋再生医学产品称号。

Not intended for UK Media

不适用于英国媒体

Summary

摘要

Designation enables preferred procedural treatment and review consultation by Japan’s Ministry of Health, Labor and Welfare / Parkinson’s disease affects approximately 10 million people worldwide and 250,000 in Japan

指定使得日本厚生劳动省能够优先进行程序处理和审查咨询/帕金森病在全球影响约1000万人，其中在日本有25万人受影响。

Berlin, Germany and Cambridge, MA, USA, December 17, 2025

德国柏林，美国马萨诸塞州剑桥，2025年12月17日

– Bayer AG and its wholly owned, independently operated subsidiary BlueRock Therapeutics LP, a clinical stage cell therapy company, today announced that the investigative cell therapy bemdaneprocel, has received Pioneering Regenerative Medical Product designation (SAKIGAKE) from Japan’s Ministry of Health, Labor and Welfare (MHLW.) A drug candidate that receives Sakigake designation may be eligible for more frequent interaction with the MHLW to discuss the drug candidate’s development plan as well as eligibility for accelerated approval and priority review, if relevant criteria are met. .

– 拜耳公司及其全资独立运营的子公司、处于临床阶段的细胞治疗公司BlueRock Therapeutics LP今天宣布，其研究性细胞疗法bemdaneprocel获得了日本厚生劳动省（MHLW）授予的先锋再生医疗产品（SAKIGAKE）资格。获得SAKIGAKE资格的候选药物将有资格与厚生劳动省进行更频繁的互动，以讨论该候选药物的开发计划，并在符合条件的情况下获得加速批准和优先审查。

“We are honored that bemdaneprocel has received Sakigake designation in Japan, recognizing the urgent need for new and innovative treatment options for people living with this debilitating condition,” said Gabi Belfort, MD PhD and Bemdaneprocel Product Lead at BlueRock Therapeutics. “This designation will allow us to work closely with Japan’s regulators to advance our therapy as efficiently as possible and bring new hope to people living with Parkinson’s disease and their families.”.

“我们感到非常荣幸，bemdaneprocel在日本获得了Sakigake指定，这体现了对这一衰竭性疾病患者急需新的创新治疗方案的认可。” BlueRock Therapeutics的医学博士兼Bemdaneprocel产品负责人Gabi Belfort说道，“这一指定将使我们能够与日本的监管机构紧密合作，尽可能高效地推进我们的疗法，为帕金森病患者及其家庭带来新的希望。”

Parkinson’s disease is the world’s second leading neurodegenerative disease, affecting approximately 10 million people worldwide and 250,000 in Japan.

帕金森病是全球第二常见的神经退行性疾病，影响全世界约1000万人，日本约有25万人受影响。

“Receiving the Sakigake designation for bemdaneprocel highlights its potential to change how we approach Parkinson’s disease treatment,” said Christian Rommel, PhD and Global Head of Research and Development for Bayer's Pharmaceuticals Division. “Our collaboration with the Japanese regulatory authority reflects our dedication to partnering with healthcare systems and regulators worldwide.

“Bemdaneprocel 获得 Sakigake 指定突显了其改变我们治疗帕金森病方式的潜力，”拜耳制药部门全球研发主管 Christian Rommel 博士表示。“我们与日本监管机构的合作反映了我们致力于与全球医疗系统和监管机构合作的决心。”

Together, we aim to improve patient access to innovative therapies and make strides in enhancing patient outcomes.” .

共同致力于改善患者获得创新疗法的机会，并在提高患者治疗效果方面取得进展。

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 pluripotent stem cells that continue developing into mature dopamine neurons after implantation.

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。这些多巴胺能神经元前体来源于人类胚胎多能干细胞，在植入后会继续发育为成熟的多巴胺神经元。

In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. 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.

在手术过程中，这些神经前体细胞会被植入帕金森病患者的大脑中。移植后，它们有潜力重新形成受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。

In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. A pivotal Phase III clinical trial (exPDite-2) to assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control is currently enrolling participants.

2021年，bemdaneprocel获得快速通道资格，2024年获得FDA的再生医学先进疗法（RMAT）资格。目前，一项关键的III期临床试验（exPDite-2）正在招募参与者，以评估bemdaneprocel与假手术对照相比的疗效、安全性和整体影响。

Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority..

贝马单抗尚未被任何卫生机构批准用于治疗任何疾病或医疗状况。

About exPDite-2

关于exPDite-2

exPDite-2 is the first Phase III pivotal clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease. In a Phase I study with 12 participants, bemdaneprocel was well tolerated, with no serious adverse events related to drug product at 36 months post-surgery.

exPDite-2 是首个针对研究性同种异体多能干细胞衍生疗法治疗帕金森病的 III 期关键临床试验。在一项包含 12 名参与者的 I 期研究中，bemdaneprocel 表现良好，在术后 36 个月未出现与药物相关的严重不良事件。

In addition, encouraging trends were observed in secondary endpoints related to motor impairments at 36 months post-surgery. Building on these results, exPDite-2 is a multicenter, double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control.

此外，在手术后 36 个月时观察到与运动障碍相关的次要终点有令人鼓舞的趋势。基于这些结果，exPDite-2 是一项多中心、双盲试验，将评估 bemdaneprocel 与假手术对照相比的疗效、安全性和总体影响。

The trial is designed to enroll approximately 102 participants with Parkinson’s disease in US, Canada, and Australia. The primary endpoint of the Phase III study is change from baseline to week 78 in PD motor diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day.

该试验旨在招募大约 102 名来自美国、加拿大和澳大利亚的帕金森病患者。III 期研究的主要终点是从基线到第 78 周，以 16 小时清醒日调整后，无明显异动症的 ON 时间（PD 运动日记测量）的变化。

In addition, the trial will incorporate secondary endpoints designed to assess objective measures of movement, non-motor symptoms, safety and tolerability, and instruments that capture activities of daily living and quality of life..

此外，试验还将纳入次要终点，以评估运动的客观指标、非运动症状、安全性和耐受性，以及捕捉日常生活活动和生活质量的工具。

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）是一种进展性神经退行性疾病，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、精力不足、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 are inadequate and lack holistic management of symptoms so there is an urgent need for new therapies..

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，现有的治疗选择不充分，缺乏对症状的整体管理，因此迫切需要新的治疗方法。

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 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。我们正在利用细胞治疗的力量，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分别是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 III clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase I clinical trial, CLARICO 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.

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，目前正在进行III期临床试验exPDite-2。OpCT-001获得了FDA的快速通道资格，目前正在进行I期临床试验CLARICO。BlueRock于2016年作为Versant Ventures与拜耳旗下影响力投资部门Leaps by Bayer的合资企业成立，该部门致力于投资具有颠覆性突破创新的技术。

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.

2019 年底，BlueRock 成为了拜耳集团的全资独立运营子公司。我们的文化以无畏任何挑战而坚持下去的勇气、变革医学和传递希望的紧迫感、以使命为导向的诚信以及以社区为重的精神为核心理念，因为我们深知，我们都是比自身更宏大的事业的一部分。

For more information, visit .

有关更多信息，请访问。

www.bluerocktx.com

。

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.

拜耳是一家在全球范围内拥有医疗保健和营养等生命科学领域核心竞争力的企业。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信任、可靠性和质量。2024财年，该集团拥有约93,000名员工，销售额达466亿欧元。

R&D expenses amounted to 6.2 billion euros. For more information, go to .

研发费用共计62亿欧元。欲了解更多信息，请访问。

www.bayer.com

。

Find more information at

更多信息请访问

https://pharma.bayer.com

在Facebook上关注我们：

http://www.facebook.com/bayer

Find information at

查找信息于

www.bluerocktx.com

在LinkedIn上关注我们：

www.linkedin.com/company/bluerocktx

Forward-Looking Statements

前瞻性声明

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here.

本发布可能包含基于拜耳管理层当前假设和预测的前瞻性声明。各种已知和未知的风险、不确定性和其他因素可能导致公司的实际未来结果、财务状况、发展或业绩与这里提供的估计存在重大差异。

These factors include those discussed in Bayer’s public reports which are available on the Bayer website at .

这些因素包括拜耳公开报告中讨论的因素，这些报告可在拜耳网站上查阅。

www.bayer.com

. The company assumes no liability whatsoever to update these forward-looking statements or to conform

公司不承担更新这些前瞻性声明或使其符合的任何责任。

them to future events or developments.

让他们参与未来的事件或发展。

Bayer contact for media inquiries:

拜耳媒体咨询联系人：

Dr. Imke Meyer, phone +49 214 60001275

伊姆克·迈尔博士，电话 +49 214 60001275