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Bemdaneprocel is created to replace the dopamine-producing neurons lost due to Parkinson’s disease. Credit: Annick Vanblaere from Pixabay. The US Food and Drug Administration (FDA) has granted a regenerative medicine advanced therapy (RMAT) designation for BlueRock Therapeutics’ investigational cell therapy bemdaneprocel (BRT-DA01) for Parkinson’s disease. Recommended Buyer's Guides Buyer's Guide Leading Guide to Packaging Materials, Containers and Containment Services for the Pharmaceutical Industry Buyer's Guide Top Guide for Drug Delivery Systems BlueRock is a wholly owned independently operated Bayer subsidiary. Bemdaneprocel is claimed to be the most clinically advanced cell therapy for Parkinson’s disease treatment in the US. The therapy is created to replace the dopamine-producing neurons lost in the course of the disease. It was assessed in the multi-site, multicentre, non-randomised, open-label, non-controlled study. See Also: Dupixent gets closer to EU approval as the FDA delays its decision on COPD Bristol Myers Squibb’s Breyanzi gains FDA approval for lymphoma The trial involved 12 subjects diagnosed with Parkinson’s disease who underwent surgical transplantation of bemdaneprocel cells into the post-commissural putamen bilaterally, followed by a one-year immunosuppression regimen. Data from the trial, announced in March 2024, indicated that bemdaneprocel was well tolerated and did not present major safety issues up to 18 months post-treatment. Furthermore, an increase in the F-DOPA PET imaging signal was observed after the cessation of immune suppression therapy at 12 months, suggesting that the transplanted cells survive and engraft in the brain. The RMAT designation is managed by the FDA’s Center for Biologics, Evaluation and Research (CBER). It is specifically for investigational regenerative therapies, including cell therapies, aimed at treating, modifying, reversing or curing serious ailments. Therapies granted RMAT status can benefit from expedited development review and development planning guidance from senior CBER managers. This status also facilitates early discussions about possible surrogate endpoints and strategies to support accelerated approval and satisfy post-approval needs. BlueRock Therapeutics president and CEO Seth Ettenberg stated: “We are excited about the positive data from the bemdaneprocel Phase I clinical trial and believe it has great potential to help patients living with Parkinson’s disease regain functions they have lost to the disease. “Now with this RMAT designation in hand, we look forward to closely collaborating with the FDA to ready this programme for Phase II clinical studies.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

在获得再生医学先进疗法（RMAT）认定之前，bemdaneprocel I期临床试验结果表明其耐受性良好，且在18个月内没有出现重大安全问题Bemdaneprocel是美国临床最先进的研究性细胞疗法，用于治疗帕金森病患者近日，拜耳和BlueRock Therapeutics LP（一家临床阶段细胞治疗公司，拜耳公司全资独立运营的子公司）宣布，BlueRock用于治疗帕金森病的研究性细胞疗法bemdaneprocel已获得美国食品药品监督管理局（FDA）的再生医学先进疗法（RMAT）认定。Seth EttenbergBlueRock Therapeutics总裁兼首席执行官“我们对bemdaneprocel I期临床试验的积极数据感到兴奋，并相信它具有帮助帕金森病患者恢复因疾病而丧失的功能的巨大潜力。基于RMAT认定，我们期待与FDA密切合作，为该项目的II期临床研究做好准备。”Bemdaneprocel是美国治疗帕金森病的临床最先进的研究性细胞疗法。3月公布的I期临床试验数据表明，bemdaneprocel在18个月时耐受性良好，没有出现重大安全问题。此外，根据研究方案中的定义，在停止免疫抑制治疗12个月后，观察到F-DOPA PET成像信号有所增加，这表明移植的细胞能够存活并植入大脑。RMAT项目由FDA生物制品评估和研究中心（CBER）管理，致力于细胞疗法等研究性再生医学，旨在治疗、改变、逆转或治愈严重疾病。获得RMAT认定的研究性疗法有资格获得CBER高级管理人员的快速开发审查和开发规划指导。RMAT还为讨论潜在替代终点，以及加速批准和满足批准后要求敞开了大门。Christian Rommel拜耳处方药事业部执行委员会成员兼全球研发负责人“Bemdaneprocel获得RMAT认定证明，该候选药物有可能从根本上改变我们对帕金森病治疗的看法。我们致力于为患者提供突破性创新，并为bemdaneprocel进一步扫清开发障碍感到自豪和兴奋。”关于拜耳拜耳作为一家跨国企业，在生命科学领域的健康与农业方面具有核心竞争力。公司致力于通过产品和服务，帮助人们克服全球人口不断增长和老龄化带来的重大挑战，造福人类和地球繁荣发展。拜耳致力于推动可持续发展并对业务产生积极影响。同时，集团还通过科技创新和业务增长来提升盈利能力并创造价值。在全球，拜耳品牌代表着可信、可靠及优质。在2023财年，拜耳的员工人数约为100,000名，销售额为476亿欧元。不计特殊项目的研究开发投入为58亿欧元。更多信息请见www.bayer.com。前瞻性声明本新闻稿包括拜耳集团管理层基于当前设想和预测所作的前瞻性声明。各种已知和未知的风险、不确定性和其它因素均可能导致公司未来的实际运营结果、财务状况、发展或业绩与上述前瞻性表述中所作出的估计产生重大差异。这些因素包括在拜耳官方网站http://www.bayer.com/上公开的拜耳各项报告。本公司没有责任更新这些前瞻性声明或使其符合未来发生的事件或发展。