Bemdaneprocel

CAMBRIDGE, Mass.--(BUSINESS WIRE)--GC Therapeutics (GCTx), a pioneering biotechnology company advancing the next generation of cell-based medicines through its TFome™ (Transcription-Factor-ome) induced pluripotent stem cell (iPSC) programming platform that enables expansion into in vivo rejuvenation and reprogramming, today announced the appointment of Stefan Irion, MD, as chief scientific officer. Dr. Irion brings more than two decades of experience translating stem cell science into clinical stage therapeutics. In his role at GCTx, Dr. Irion will lead and direct the company’s research strategy as it advances its differentiated cell therapy pipeline toward the clinic. His appointment follows the recent addition of Ms. Kate Haviland as Board Chair, further strengthening GCTx’s leadership team and positioning the company for its next phase of growth. Concurrently, co-founder Alex Ng, PhD, will assume the role of chief innovation officer, where he will support the continued evolution and innovation of the company’s TFome™ platform, which powers the company’s Cell State Cookbook™ and virtual cell models. Dr. Irion joins GCTx from BlueRock Therapeutics (a wholly-owned, independently operated subsidiary of Bayer AG ), where he served as chief scientific officer and a member of the Executive Committee. At BlueRock, he built and led a research organization of approximately 60 scientists developing cell & gene therapies across multiple therapeutic areas. Earlier, at Memorial Sloan Kettering Cancer Center, Dr. Irion played a central role in developing MSK-DA01 (bemdaneprocel), a first human pluripotent stem cell-derived dopaminergic neuron therapy for Parkinson’s Disease. He served as lead author on the IND package and was instrumental in the program’s out-licensing to BlueRock, where he subsequently guided the program through IND clearance. MSK-DA01 has since completed a Phase 1 clinical trial, received both RMAT and Fast Track designations from the FDA, and has started enrollment for its registrational Phase III study. Prior to BlueRock, Dr. Irion led discovery efforts at iPierian that produced the anti-tau antibody IPN0007, contributing to Bristol-Myers Squibb’s $725 million acquisition of the company in 2014. “We are thrilled to welcome Stefan to our leadership team,” said Parastoo Khoshakhlagh, PhD, CEO and Co-founder of GCTx. “Stefan brings deep expertise in developing novel therapeutics, along with exceptional leadership and an innovative perspective. His strong commitment to patients makes him uniquely suited to help drive GCTx’s next phase of growth. We are confident he will have a meaningful impact on our mission to make cell therapy accessible to patients around the world and to unlock the TFome™ platform capabilities into rejuvenation and in vivo reprogramming.” Dr. Irion is a named inventor on multiple patents in stem cell engineering and cell therapy, and his research has been published in leading journals including Nature , Nature Biotechnology , Cell Stem Cell , and Nature Communications . He received his medical degree summa cum laude from the University of Tübingen, Germany, and completed postdoctoral training at the Mount Sinai School of Medicine in New York, and the University Health Network in Toronto. “Having spent my career working to bring cell therapies from bench to bedside, I've seen how much is still possible; and how many patients are still waiting,” said Dr. Irion. “TFome™ opens a new chapter in how we control cell fate and design therapies, giving us a level of precision, scale and flexibility that simply wasn't available before. The Cell State Cookbook™ and virtual cell models not only accelerate what we already know how to do; they expand what's imaginable, from next-generation cell therapies to in vivo reprogramming and rejuvenation approaches that could address diseases we've barely been able to touch. I'm thrilled to join a team that is turning that potential into treatments, and to help make sure more patients benefit sooner.” Dr. Irion’s appointment underscores GCTx’s commitment to advancing innovative cell therapies and making them more accessible to patients in need. About TFome™, the world’s first “plug-and-play” cellular programming platform Induced pluripotent stem cell (iPSC) differentiation into distinct cell types depends on various signaling pathways that ultimately converge onto transcription factors (TFs), which control the sets of genes that are active in a cell. Cell fate is determined by specific TFs acting in concert. GCTx has built the world’s first complete collection of human TFs. Through sophisticated multidimensional screening modalities that integrate genome-scale experimental testing and powerful next-generation predictive AI foundation models informed by GCTx’s proprietary data, TFome™ has been proven to identify fully optimized TF combinations to differentiate iPSCs into virtually any functional cell type. This allows for a single-step, four-day stem cell differentiation process with >90% efficiency with no requirement for cell type-specific optimization for microenvironmental factors. This has not been achieved previously via conventional small-scale testing. TFome™ also enabled the construction of the Cell State Cookbook™ (CSC™) to build the epigenomic transition matrix which is positioned to precisely engineer any cell state for in vivo reprogramming. Through TF-based cell fate programming, TFome is designed to yield first-in-class and best-in-class off-the-shelf cell therapy products up to 100 times more rapidly than conventional methods of cell differentiation with improved potency, efficiency, quality and cost (SuperCells™). GCTx is positioned to be the first company in the iPSC-based cell therapy space to pursue multiple cell products from one starting point using a similar manufacturing strategy for more accelerated development and timelines. GCTx has built the largest empirical map of TF-driven cell state transitions, the CSC™, based on data from over two million tested TF combinations. The CSC™ powers compounding predictive AI foundation models that capture key features of cell state control and enable rapid identification of optimized TF programs, significantly streamlines discovery for cell therapy development and in vivo reprogramming. TFome™ was invented and developed by a world-class team of scientists in the lab of professor George Church, Ph.D., at Harvard Medical School and the Wyss Institute. About GC Therapeutics GC Therapeutics (GCTx) is a biopharmaceutical company created to scale and unlock the next generation of cell therapy-based medicines. GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™, integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the therapeutic development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx’s initial focus is on developing first-in-class and best-in-class cell therapies for neurological and metabolic diseases with potential for expansion into other therapeutic modalities. Founded in 2019, GCTx is based in Cambridge, MA and has raised $75 million to date from leading life sciences investors. For more information, please visit us at and follow us on LinkedIn and X . Contacts Media Contact: Katie Engleman katie@1abmedia.com

01 张阿姨的膝盖和那18张审批函 张阿姨今年68岁，过去五年她最怕的不是体检报告，而是上下楼梯。膝盖像两把钝刀卡在关节缝里，每迈一步都不由自主地把身体重心甩向扶手。“医生说这叫膝骨关节炎，说全国像我这样的人有1.2亿”——这话并非夸张。据流行病学调查，我国症状性膝骨关节炎患病率达8.1%，60岁以上人群中发病率高达50%，75岁以上则攀升至80%，骨关节炎总患病率达15%，全国患者超过1亿人，被称为“头号致残性疾病”。 更令人揪心的是，这个数字还在以每年新增数百万的速度膨胀。而在这些冰冷统计数字的另一端，是无数个像张阿姨一样被腿疼困住生活的普通人。 2025年1月2日，中国国家药监局批准了首款国产间充质干细胞新药——艾米迈托赛注射液。到了2026年4月22日，数据已经更新至2025年全年共有38款干细胞1类新药获批临床试验，覆盖内分泌系统、呼吸系统、神经系统、消化系统、骨关节及自身免疫性疾病等多个治疗领域。与此同时，上海核发了首张干细胞药品生产许可证，获证企业的人脐带间充质干细胞注射液已完成III期临床试验，正在全力冲刺新药上市申请。这款产品瞄准的，正是膝骨关节炎。 数字不会骗人——政策大门已开，赛道挤满了选手，而这场“细胞革命”的第一站，落在了你我身边最朴素的痛点上。 02 细胞是如何“修理”我们自己的 要理解干细胞为什么能治病，得先弄清一个问题：我们的身体出了毛病，根源在哪里？ 拿帕金森病来说，它的病理核心不难懂——大脑里专门生产一种叫“多巴胺”的神经信号的细胞，正在一批接一批地死亡。多巴胺是大脑控制运动的关键信使，少了它，手就开始抖，身体变得僵硬，走路迈不开步子。市面上大部分药物，本质上是给大脑“补充”缺失的多巴胺，相当于给没油的车硬灌燃料。但随着疾病进展，剩下的健康细胞越来越少，药物的作用越来越弱，副作用却越来越明显——异动症、剂末现象、开关现象层出不穷。 干细胞的逻辑完全不一样。它不是往里“添燃料”，而是直接“换个发动机”。诱导性多能干细胞可以在体外被定向诱导分化为多巴胺能神经元前体细胞，然后通过微创手术移植到患者大脑壳核区域，让新细胞扎根、存活、分泌多巴胺，真正修复受损的神经回路。 同样的原理，到了膝盖这里也完全说得通。膝骨关节炎的本质是关节软骨被磨坏了，而软骨本身几乎没有自我修复能力。传统治疗手段大多是“止痛”——非甾体抗炎药缓解炎症，玻璃酸钠当“润滑剂”，再严重就关节置换。但这些都是治标不治本的路子。人脐带间充质干细胞则能从根源下手：它们可以定向分化为软骨细胞，直接参与软骨修复；还能分泌大量生长因子和抗炎因子，抑制滑膜炎症、调节免疫微环境。说白了，它让身体重新获得了“自己修自己”的能力。 这种“活细胞药物”和传统化学药物的区别，就像把一台坏机器拿去请人修好了，和每周给机器加点润滑油——你选哪个？ 03 从颤抖双手到疼痛膝盖：干细胞在路上 先说帕金森。2026年4月，一个重磅数据在全球神经科学领域掀起了不小的波澜。在2026年美国神经病学学会年会上，拜耳旗下BlueRock Therapeutics公布了其干细胞疗法bemdaneprocel的两年随访数据：12名入组患者中，28例与治疗相关的不良事件均为轻至中度，且无一与移植细胞本身直接相关，无颅内出血、无死亡、无移植诱导的运动障碍。更关键的是疗效——患者在“开期”状态（无令人困扰的异动症）下平均每日多了2.1小时的有效活动时间，“关期”减少了2.0小时，MDS-UPDRS第三部分运动评分在服药状态下改善了5.7分。 2.1小时意味着什么？意味着一个常年被“关期”困在家里的帕金森患者，每天多出两个小时能和家人好好吃顿饭、出门晒晒太阳、做点自己想做的事。 日本方面也传来了好消息。Nature Biotechnology报道，2026年3月6日，日本厚生劳动省有条件批准了全球首款iPSC衍生细胞疗法用于帕金森病治疗，这项产品在包含7名帕金森患者的1/2期试验中，有4名表现出运动功能改善且未出现严重不良事件。 中国自己的步伐也没落下。北京天坛医院正在开展iPSC衍生多巴胺能神经前体细胞的注册临床试验，入组的中晚期帕金森患者在接受细胞移植后，运动功能和情绪、睡眠、日间疲劳感等非运动症状均出现了显著改善。一位入组患者在感谢信中写道：“重新点燃了我几近熄灭的生命之火。” 再说膝盖。数据同样硬。中南大学湘雅医院团队对6项临床研究、共248例膝骨关节病患者进行的Meta分析表明，接受人脐血间充质干细胞治疗的试验组目测类比疼痛评分显著低于对照组，Lysholm膝关节功能评分也显著优于对照组。而在另一项纳入242名KOA患者的回顾性队列研究中，Kellgren-Lawrence分级为中度（2/3级）的患者在接受脂肪来源干细胞关节腔注射后，KOOS总分、日常生活功能、生活质量分数以及VAS疼痛评分均实现了显著提升，轻中度关节肿胀或不适发生率仅约5%至6%，整体安全性良好。 赛道上的选手越来越多了。2025年至2026年初，仅针对膝骨关节炎，就有源品生物的“人脐带间充质干细胞注射液”、普华赛尔的“人脐带源间充质干细胞注射液”等多款1类新药相继获批IND。韩国MEDIPOST公司的脐带血间充质干细胞疗法Cartistem已在美国获批III期临床，目标正是发展为全球首个疾病修饰性骨关节炎药物。全球范围内，一场以干细胞为核心的慢病治疗范式迁移，已经悄然拉开帷幕。 04 当修复取代抑制，细胞正重新定义“治病”二字 回到张阿姨的故事。如今她的双腿不再需要扶手“护驾”，走路步子轻了大半。她的膝盖没有被替换成金属假体，疼痛也没有被化学药物“压下去”——那些“罢工”的软骨细胞内，外援干细胞正在帮忙重建软骨基质，抑制磨损的加速，尝试让膝关节重获新生。 从1.2亿膝骨关节炎患者，到全球上千万帕金森病患者，细胞治疗正在改写的远不止一份份病历。它以“活细胞”为武器，从根本上触及了制药行业过去几十年难以触达的命题——当疾病的发生源于细胞层面的崩溃时，解决问题的唯一办法，正是动用生命最基础的力量去重建生命。 2026年的中国，18款新获批的干细胞药物只是一个开始。从政策切口看，国家重点研发计划已设“细胞与基因治疗”为颠覆性专项，国务院818号令正式实施“临床研究备案＋转化应用审批”双轨监管。从产业维度看，2025年中国细胞治疗产业规模已达120亿元，预计2027年突破500亿元。而最终，真正改变一切的，不是这些数字，而是一个个曾经被慢性病耗尽生活热情的人，重新站直了、走出来了、笑了。 这场细胞治疗的大幕，才刚刚拉开。 参考资料： BlueRock Therapeutics. Positive safety data for bemdaneprocel in Parkinson’s disease. AAN 2026 Annual Meeting. April 2026. 日本住友制药、Racthera公司. Amchepry iPS细胞疗法获日本厚生劳动省批准.《Nature Biotechnology》. 2026;44:498. iRegene Therapeutics. FDA grants RMAT designation to NouvNeu001 for Parkinson disease. PR Newswire. January 2026. 首都医科大学附属北京天坛医院. iPSC衍生多巴胺能神经前体细胞治疗原发性帕金森病注册临床试验进展.《药时代》. 2026年4月. 刘彦哲, 刘华, 杨土保, 刘育鹏. 人脐血间充质干细胞对膝骨关节病患者疼痛和功能影响的Meta分析.《中国组织工程研究》. 2026;30(19):5066-5071. MEDIPOST. FDA Clears Phase 3 Trial for Cartistem in Knee Osteoarthritis. Korea Biomedical Review. February 2026. 国家药品监督管理局药品审评中心（CDE）. 干细胞新药临床试验获批与受理数据统计（2025.1—2026.4）. 上海爱萨尔生物医药股份有限公司. 获上海首张干细胞《药品生产许可证》. 源品细胞生物科技集团有限公司. 人脐带间充质干细胞注射液获批IND治疗膝骨关节炎. 普华赛尔生物医疗科技有限公司. 人脐带源间充质干细胞注射液IND获批. 2026年1月. 国务院.《生物医学新技术临床研究和临床转化应用管理条例》（818号令）. 2025年9月. 《“十四五”生物经济发展规划》. 国家发展改革委. 2022年. 中国健康管理协会生物治疗分会. 新规元年：干细胞技术迎国家级规范. 2025年10月. 国家卫生健康委. 对十四届全国人大三次会议第7870号建议的答复. 2025年9月. 华经产业研究院. 2025年中国细胞治疗产业规模数据. 流行病学调查数据（膝骨关节炎患病率8.1%，骨关节炎总患病率15%，患者超1亿人）. 2026年. 你怎么看？欢迎在留言区和我们说说： 你是否了解过细胞治疗这类抗癌方式？ 抗癌路上，你最担心的问题是什么？ 你最想知道细胞治疗的哪些真相？ 你在抗癌中遇到了哪些难题？ 留言告诉我，下期为你解答最关心的抗癌疑问。 #818号令 #828号令 #细胞行业 #细胞治疗 #干细胞 #细胞转型 #IIT临床研究 #IND申报 #孤儿药 #医疗合规 #细胞出路 #细胞工厂 #CAR-T #医疗政策 #细胞价格  特别声明  本公众号所有文章旨在宣传生命科学领域的最新技术和研究，探索最前沿的资讯和进展，不代表本公众号立场。本文所有内容均不构成任何医疗指导。如有需求请通过正规就医渠道及时就医。 如果您有任何需求，可以在留言区留言，也可以扫码咨询