A Phase IB/IIA Study of Remestemcel-L, an ex Vivo Culture-expanded Adult Allogeneic Bone Marrow Derived Mesenchymal Stem Cell Product, for the Treatment of Medically Refractory Ulcerative Colitis

The purpose of this study is to determine the safety and efficacy of using remestemcel-L, an ex vivo culture-expanded adult allogeneic bone marrow derived mesenchymal stem cell product (MSCs) delivered by targeted endoscopic delivery to treat people for medically refractory ulcerative colitis.
This study will enroll adult patients with medically refractory ulcerative colitis who are planning to switch biologic therapy or undergo colectomy as the next stage in their treatment plan.

开始日期2020-11-10

申办/合作机构

The Cleveland Clinic Foundation

[+1]

NCT04548583

/ Unknown status临床1/2期IIT

A Phase IB/IIA Study of Remestemcel-L, an Ex-vivo Culture-expanded Adult Allogeneic Bone Marrow Derived Mesenchymal Stem Cell Product for the Treatment of Medically Refractory Crohn's Colitis

Crohn's disease has several phenotypes (inflammatory, stricturing, fistulizing) and location (small bowel, ileocecal, colon, and perianal). Approximately one third of patients have inflammation limited to the colon. Up to two thirds will become medically refractory and require a total abdominal colectomy for symptom control. The purpose of this study is to determine the safety and efficacy of using allogeneic bone marrow derived mesenchymal stem cells (MSCs) delivered by targeted endoscopic delivery to treat people for medically refractory Crohn's colitis.

开始日期2020-11-04

申办/合作机构

The Cleveland Clinic Foundation

[+1]

NCT04371393

/ Terminated临床3期IIT

Mesenchymal Stromal Cells for the Treatment of Moderate to Severe COVID-19 Acute Respiratory Distress Syndrome

The mortality rate in SARS-CoV-2-related severe ARDS is high despite treatment with antivirals, glucocorticoids, immunoglobulins, and ventilation. Preclinical and clinical evidence indicate that MSCs migrate to the lung and respond to the pro-inflammatory lung environment by releasing anti-inflammatory factors reducing the proliferation of pro-inflammatory cytokines while modulating regulatory T cells and macrophages to promote resolution of inflammation. Therefore, MSCs may have the potential to increase survival in management of COVID-19 induced ARDS.
The primary objective of this phase 3 trial is to evaluate the efficacy and safety of the addition of the mesenchymal stromal cell (MSC) remestemcel-L plus standard of care compared to placebo plus standard of care in patients with acute respiratory distress syndrome (ARDS) due to SARS-CoV-2. The secondary objective is to assess the impact of MSCs on inflammatory biomarkers.

开始日期2020-04-30

申办/合作机构

Icahn School of Medicine at Mount Sinai

[+2]

100 项与 Remestemcel-L-rknd 相关的临床结果

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100 项与 Remestemcel-L-rknd 相关的转化医学

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100 项与 Remestemcel-L-rknd 相关的专利（医药）

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项与 Remestemcel-L-rknd 相关的文献（医药）

2024-01-01·Blood cell therapy

Efficacy of off-the-shelf bone marrow mesenchymal stem cells for pediatric steroid-refractory acute graft-versus-host disease

Article

作者: Mori, Makiko ; Kubota, Hirohito ; Ishikawa, Takahiro ; Oshima, Koichi ; Koh, Katsuyoshi ; Mizushima, Yoshitaka ; Mitani, Yuichi ; Kaneko, Ryota ; Honda, Mamoru ; Watakabe, Mai ; Irikura, Tomoya ; Fukuoka, Kohei ; Arakawa, Yuki

Introduction:

Temcell is a mesenchymal stem cell (MSC) product approved for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in Japan. However, reports regarding Temcell's efficacy in pediatric patients have been scarce, and the appropriate use of MSC therapy against pediatric SR-aGVHD also remains to be determined.

Patients and Methods:

We retrospectively assessed a cohort of pediatric patients treated with Temcell for SR-aGVHD following allogeneic hematopoietic transplantation. MSCs were infused intravenously at a dose of 2 × 10⁶ cells/kg according to the manufacturer's instructions.

Results:

Twelve patients received eighteen cycles of MSC therapy (median age, 10.3 [1.7-17.8] years), with four receiving additional cycles (one cycle: n = 3, three cycles: n = 1). The severity of aGVHD before MSC therapy was grade I-II in three patients and grade III-IV in nine patients (gut stage 3-4, n= 7; liver stage 3-4; n =2). The median number of immunosuppressive therapy regimens received prior to MSC administration was two (range: 1-5). The first MSC cycle displayed the best overall response rate of 83%, including six patients with a complete response (CR) and with a 49% reduction in the mean daily dose of prednisone after eight weeks. The median time to first response was 3.5 days (range: 2-15 days). Two of the four patients who were re-administered MSCs for recurrent or persistent GVHD achieved a CR. The three-year overall survival rate was 69.4%, while the three-year failure free survival (FFS) rate was 22.2%, with a median FFS of 4.9 months. There were no observable side effects of MSC therapy.

Conclusions:

MSC therapy appears to be an effective and safe treatment for pediatric SR-aGVHD, with a steroid-sparing effect and satisfactory efficacy upon re-administration. Further studies are needed to determine its appropriate combination with additional treatments and the optimal use of re-administration of MSCs.

2023-07-01·Molecular and cellular biochemistry

Pooled evidence from preclinical and clinical studies for stem cell-based therapy in ARDS and COVID-19

Review

作者: Murugan, Dhanashree ; Rangasamy, Loganathan

COVID-19 has severely devastated many lives across the globe. It has been speculated that stem cell-based therapy for COVID-19 treatment could be able to subsidize the effects. In preclinical and clinical studies, stem cell-based therapy has successfully eliminated inflammatory cytokines in ALI, ARDS, and COVID-19. Clinical trials have produced a variety of promising results for validating stem cell therapy in COVID-19 patients. For instance, exosome-based therapy (ExoFlow) showed an 87% survival status, and MSC-based therapy (Mesoblast) achieved an 83% survival rate in moderate to severe COVID-19 patients. This review debates the advantages of cell-free therapy, i.e., stem cell-derived exosome-based therapies, over stem cell-based therapy. This review aims to question whether the immunomodulatory effect of stem cells differs based on their origin and also tries to find possible answers for the best stem cells for treating SARS-CoV-2 infection. The role of stem cells and their extracellular vesicles in the upregulation of regulatory immune cells, growth factors (EGF, FGF, VEGF), and anti-inflammatory cytokines (IL-6, INF-α, galectin-1, notch-1, PDL-1) that promote the tissue regeneration at the injured site. The right side of the image depicts the downregulation of inflammation-inducing immune cells, pro-inflammatory cytokines, and chemokines that could also enhance COVID-19 therapy.

2023-01-01·Frontiers in immunology

Bringing function to the forefront of cell therapy: how do we demonstrate potency?

Article

作者: Weil, Ben ; Lowdell, Mark W

Unlike conventional pharmaceuticals, biologics and Advanced Therapy Medicinal Products (ATMPs) are required to meet a standard of "potency" as part of the final release criteria at completion of manufacture. During early phase clinical trials, most regulatory agencies have been willing to accept very immature potency assays with an expectation that these will be improved, qualified and validated during the clinical development of the drug to Marketing Authorisation Application (MAA) or Biologics License Application (BLA) submission.This model of continuous development of potency assay in parallel with drug development has already led to at least two notable problem cases; namely Iovance and Mesoblast. Both companies completed successful phase III clinical trials but, in both cases, the initial BLA was rejected on the basis that their potency assay for drug product release was inadequate. Fortunately these issues appear to have been overcome in March of this year, with Mesoblast receiving acceptance of their BLA for Remestemcel and Iovance obtaining a rolling BLA approval for Lifileucel.

161

项与 Remestemcel-L-rknd 相关的新闻（医药）

2025-01-28

·药事纵横

2024年被拒批、延迟、终止的CGT疗法

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。回顾 2024 年，细胞和基因疗法（CGT）领域可谓是波澜壮阔，在取得诸多突破的同时，也面临着部分疗法受挫的情况。来源：细胞基因疗法前沿据不完全统计，2024 年全球有 17 款 CGT 疗法获批上市。当然也有一部分CGT疗法遭遇挫折，或被拒批，或延迟审批或临床试验被迫终止。例如，一些企业的 CGT 产品由于在临床试验中未能达到预期的疗效终点，或者出现了安全性问题，导致审批受阻。还有部分产品因生产工艺复杂、质量控制难度大等问题，无法满足监管部门的要求，从而延迟了上市进程。具体如下：拒批 EB-101 4 月 22 日，Abeona Therapeutics 的细胞疗法prademagene zamikeracel (pz-cel，EB-101)上市申请遭拒，FDA 要求其提供治疗的有效信息及满足额外的 CMC 要求。 EB-101用于治疗隐性营养不良性大疱性表皮松解症患者。它是一款自体的细胞与基因治疗，使用逆转录病毒载体将COL7A1基因导入患者自身的皮肤细胞(角质形成细胞)，并将其移植回患者体内，以使正常的VII型胶原蛋白表达和促进伤口愈合。 Kresladi 6 月 28 日，Rocket Pharmaceuticals 的基因疗法Kresladi™ （marnegragene autotemcel; marne-cel）被拒批，FDA 要求提供有限的额外化学制造和控制 (CMC) 信息以完成审查。 Kresladi（曾用名RP-L201）用于治疗严重白细胞粘附缺陷，是一款用以治疗严重白细胞粘附缺陷-I（LAD-I）的在研基因疗法，该疗法通过慢病毒对患者的自体造血干细胞进行基因工程化改造，以递送ITGB2基因的功能性拷贝。延迟 Ryoncil：Mesoblast 的间充质干细胞疗法 Ryoncil 在审批过程中历经波折，曾收到 2 封 CRL（申请拒绝），直到 2024 年 12 月 18 日才获批上市，用于治疗激素难治性急性移植物抗宿主病。暂停或终止 vx-880 ：1 月 7 日，因两名患者死亡，Vertex 暂停细胞疗法 vx-880 的临床试验，等待独立数据监控委员会和全球监管机构对全部数据的审查。vx-880 是一种干细胞衍生的胰岛细胞疗法，用于治疗 1 型糖尿病。 Posoleucel：1月上旬，AlloVir宣布终止核心产品Posoleucel 的三项3期临床试验。Posoleucel是一种实验性的多病毒特异性T细胞疗法，AlloVir停止的3项临床试验，分别是用于预防多种病毒引起的感染或疾病，治疗病毒相关的出血性膀胱炎（vHC）和治疗腺病毒（AdV）。 fordadistrogene movaparvovec ： 5 月 7 日，一名杜氏肌营养不良患者在使用辉瑞的基因疗法 fordadistrogene movaparvovec的 2 期临床试验中去世，死因是心脏骤停。辉瑞随后暂停了其针对 4 到 8 岁男孩的随机、安慰剂对照 3 期试验 CIFFREO 中的患者交叉试验。两个月后，辉瑞宣布 3 期 CIFFREO 研究未达到主要终点，也未达到关键次要终点，决定终止开发这款产品。 giroctocogene fitelparvovec： 12 月 30 日，辉瑞宣布终止与 Sangamo 在 A 型血友病基因疗法 giroctocogenefitelparvovec上的合作。辉瑞经多方统筹分析，认为目前患者对额外基因治疗选择的兴趣非常有限。 spk-3006 ：是由罗氏子公司 Spark Therapeutics 开发的一种肝靶向的 AAV 试验性基因疗法，用于治疗庞贝病。在罗氏 2024 年上半年的管线梳理中，决定剔除该项目，停止了对其的临床开发。停止试验的决定不是由于安全问题，而是作为 Spark 内部战略组合优先级的一部分做出的。 GLPG5301：Galapagos 公司研发的一款 BCMA CAR-T 疗法 GLPG5301，主要用于治疗成人复发 / 难治性多发性骨髓瘤。因一名患者出现帕金森综合症而在 2024 年 8 月暂停所有临床。不过好在经过审查和采取额外的安全措施后，已于 2024 年第三季度恢复招募。退出 Alofisel（Darvadstrocel）：是一种同种异体间充质干细胞疗法，于2018年在欧盟获批，用于治疗对至少一种常规或生物疗法无效的非活动性/轻度活动性管腔克罗恩病的成人患者的复杂肛周瘘‌。然而，12月13日，武田制药公司宣布自愿撤销Alofisel在欧盟的营销授权。这一决定是基于补充临床试验结果，显示该药物未能达成主要和次要终点，且疗效不如预期‌。回顾 2024 年 FDA 对这些 CGT 疗法的审批动态，无论是获批上市、拒绝还是延迟，都深刻影响着患者的治疗希望、企业的发展命运以及整个行业的未来走向。审批过程中的种种要求与决策，不仅是对疗法安全性和有效性的严格把关，更是推动 CGT 领域朝着更加规范、成熟方向发展的重要力量。参考资料：公开资料

基因疗法细胞疗法上市批准ASH会议临床2期

2025-01-16

·PRNewswire

First FDA-Approved Mesenchymal Stromal Cell Therapy Ushers in a New Era of Regenerative Medicine

Transformative Scientific Breakthrough Pioneered by Spencer Trask & Co. Venture Osiris Therapeutics NEW YORK, Jan. 16, 2025 /PRNewswire/ -- The Food and Drug Administration (FDA) approved the first mesenchymal stromal cell (MSC) therapy, RYONCIL®, indicated for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in children two months and older. This milestone in regenerative medicine was developed and patented by Osiris Therapeutics (Osris), a venture founded by Kevin Kimberlin and Dr. Arnold Caplan, and first backed by Spencer Trask & Co. Continue Reading From left: Dr. Arnold Caplan (the "father of MSCs") and Kevin Kimberlin, co-founders of Osiris Therapeutics, Inc. Kimberlin, who previously collaborated with Dr. Jonas Salk to develop the first FDA-approved cell-based immunotherapy, partnered with Dr. Caplan to conduct the first human clinical trials with mesenchymal stromal cells. This work was instrumental in establishing the safety and feasibility of MSC therapies, directly contributing to the development and eventual FDA approval of treatments like RYONCIL. To complete the necessary Phase 3 trials, Osiris licensed the intellectual property in exchange for milestone payments and royalties, and sold the product, originally called Prochymal, to Mesoblast Limited (Mesoblast), creating a powerhouse at the forefront of regenerative medicine. "When we started with Dr. Caplan, we envisioned a future where this living cell would be the medicine," said Kimberlin, Spencer Trask & Co. Chairman. "This FDA action validates our pioneering work and sets the stage for MSCs to treat or cure some of the most challenging unmet needs." Originally thought to be a stem cell, the MSC was characterized by Osiris scientists in a seminal paper published in Science that has been cited 29,925 times since 1999. There are over 1,700 filings at ClinicalTrials.gov to test mesenchymal stromal or mesenchymal stem cell treatments for more than 1300 different medical conditions. Today, the scientific consensus is that the MSC acts primarily by orchestrating the body's response to injury and many infections. "MSCs are to healing what antibiotics are to bacterial infection," Kimberlin stated. "They augment our natural mechanisms to treat and heal once unhealable, untreatable conditions." RYONCIL is the latest in a series of landmark MSC innovations by Osiris which include Osteocel®, the first product marketed in the U.S. containing adult MSCs, and Grafix®, the standard-of-care today for treating chronic diabetic foot ulcers, the leading cause of amputation. RYONCIL will be available to children in the immediate future at transplant centers and hospitals around the United States. Mesoblast is advancing late-stage clinical trials with MSCs for healing cardiovascular conditions and treating lower back pain. About Spencer Trask & Co. Spencer Trask & Co. is an advanced technology development firm that discovers big ideas that drive meaningful change. The firm and its principals have co-founded or first-funded world leaders in genomics, the Internet, mobile communications, healthcare, and artificial intelligence. By supporting smart entrepreneurs, engineers, scientists, and obsessive missionaries, the firm helps create one-of-a-kind opportunities to make the world a better place. . About Kevin Kimberlin Kimberlin is a tech and biotech entrepreneur and, since 1991, Chairman of Spencer Trask & Co. He co-founded or first funded several ventures that have changed the healthcare landscape and have saved tens of thousands of lives. In addition to Osiris Therapeutics, he co-founded the first genomics venture, Myriad Genetics, which discovered the breast cancer gene, BRCA1, and Ciena Corporation, the world leader in the optical networking systems that power the Internet and the infrastructure connecting AI platforms to their data and users. References Lazarus HM, Haynesworth SE, Gerson SL, Rosenthal NS, Caplan AI. Ex vivo expansion and subsequent infusion of human bone marrow-derived stromal progenitor cells (mesenchymal progenitor cells): implications for therapeutic use. Bone Marrow Transplant. 1995 Oct;16(4): 557–64. Maxson S, Lopez EA, Yoo D, Danilkovitch-Miagkova A, LeRoux MA. Concise Review: Role of Mesenchymal Stem Cells in Wound Repair. Stem Cells Transl Med. 2012 Feb;1(2):142–9. Pittenger MF, Mackay AM, Beck SC, Jaiswal RK, Douglas R, Mosca JD, et al. Multilineage Potential of Adult Human Mesenchymal Stem Cells. Science. 1999 Apr 2;284(5411):143–7. Watson JD, Crick FHC. Molecular Structure of Nucleic Acids: A Structure for Deoxyribose Nucleic Acid. Nature. 1953 Apr;171(4356):737–8. Pittenger et al. (1999) has been cited 29926 times. Watson and Crick (1953) has been cited 19694 times. Metrics retrieved from Google Scholar, December 19th, 2024. Pittenger, M.F., Discher, D.E., Péault, B.M. et al. Mesenchymal stem cell perspective: cell biology to clinical progress. npj Regen Med 4, 22 (2019). Stem cell use part of therapy. The Baltimore Sun. 2005 Sep 23 [cited 2024 Dec 19]; Available from: DaVanzo J, Hartzman A, Surfield C, Dobson A. Cryopreserved Placental Membrane Allograft Reduces the Likelihood of Developing a New or Recurring Foot Ulcer and All-Cause Mortality in Diabetic Patients When Compared with Other Cellular- and Tissue-Based Products. Advances in Wound Care. 2023 Apr 1;12(4):169–76. Fan XL, Zhang Y, Li X, Fu QL. Mechanisms underlying the protective effects of mesenchymal stem cell-based therapy. Cell Mol Life Sci. 2020 Jul;77(14):2771-2794. doi: 10.1007/s00018-020-03454-6. Epub 2020 Jan 21. PMID: 31965214; PMCID: PMC7223321. SOURCE Spencer Trask & Co. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

细胞疗法免疫疗法上市批准临床3期

2025-01-11

·药智网

干细胞疗法开启新纪元

近日，NMPA通过优先审评审批程序附条件批准我国首款间充质干细胞疗法艾米迈托赛注射液上市。巧合的是，这也是继FDA在2024年12月18日批准首款间充质干细胞疗法Ryoncil上市之后，两款药品获批仅仅相差15天，并且适应症均为激素难治性急性移植物抗宿主病。 FDA、NMPA接连批准首款干细胞药物，预示着干细胞创新药风口已至。 1 曲折之路回顾Ryoncil细胞药物的研发和申报历史，可以发现其在美国的上市之路走得异常艰辛。从2019年开始首次申请到最终获批用时5年半的时间，其间曾收到2封CRL（申请拒绝），险被搁浅。直到今年3月，Mesoblast终于收到了来自FDA的好消息，FDA表示，经过进一步考虑，其认为提交的Ⅲ期临床数据可以支持其提交Ryoncil BLA申请。根据先前披露的临床数据，接受Ryoncil治疗第28天后的客观缓解率为70.4%，与45%的历史控制率相比有统计学意义的显著增加。接受至少一次输注治疗且随访100天的患者中，患者的死亡率为22％，而类固醇治疗无效的患者第100天死亡率高达70%。最终，FDA在2024年12月18日批准了Mesoblast的第三次上市授权申请。值得一提的是，该产品成分remestemcel-L早在2012年及2016年已分别在加拿大、新西兰（以商品名Prochymal销售）以及日本（以商品名Temcell销售）成功获得上市批准。反观艾米迈托赛的上市之路也并非一帆风顺。早在2013年3月，该药物就在国内提交了IND，然而，由于干细胞疗法的复杂性和监管要求的严格性，其研发进度相对缓慢。直到2020年6月，其临床试验才首次公示，并于2024年6月12日被CDE纳入优先审评审批，同月25日正式递交NDA，最终在2025年1月2日获批上市。尽管在探索性II期临床试验中，MSC组在第28天时相较于安慰剂组并未显示出更高的总缓解率（ORR），但MSCs在平均2周后逐渐显示出治疗效果，特别是完成8次输注且肠道受累的患者，从中获益显著。 Ryoncil与艾米迈托赛的艰难上市之路，一方面，体现出中美两国对于干细胞疗法都采取了极为严格的监管审评制度，另一方面，确实有产品已在其他国家获批，但这些产品未满足FDA与NMPA的标准，因而不能获批，这又体现了不同国家审批监管上的复杂性。 2 蓬勃之势据Research and Markets报告，2023年全球干细胞市场规模达150.7亿美元，预计将从2024年的170.2亿美元增长至2032年的561.5亿美元，预测期间的复合年增长率为16.1%。此外，南方财富网数据显示，到2025年，中国干细胞医疗市场规模预计将达到126亿美元，年复合增长率为19.1%。中美这两款获批的干细胞药物均聚焦于间充质干细胞，是一群具有自我更新和多系分化能力的细胞，广泛分布于人体各组织器官，如骨髓、脐带、脐带血或脂肪等。这类细胞具有向多种间充质系列细胞（如成骨、成软骨及成脂肪细胞等）或非间充质系列细胞分化的潜能，并具有细胞因子分泌功能，在再生医学与细胞治疗的前沿阵地展现出了惊人的应用潜力。在全球范围内，间充质干细胞作为干细胞疗法中的热门分支，其研究与应用正蓬勃发展。截至目前，全球已有655个间充质干细胞项目进入了临床研发阶段，遥遥领先于其他类型的干细胞疗法。目前全球获批上市的间充质干细胞治疗药品约10个，其细胞来源、适应症不尽相同。其中用于治疗急性移植物抗宿主病（aGVHD）的间充质干细胞治疗药品有4款，除我国批准的艾米迈托赛注射液外，还有美国、加拿大、日本批准的骨髓间充质干细胞。图片来源：国信生物转化医学平台间充质干细胞（MSCs）治疗药物选择急性移植物抗宿主病（aGVHD）作为首个适应症，主要是因为激素治疗无效的aGVHD患者受到严重的生命威胁，存在巨大的未满足的临床价值和医疗需求，而MSCs具有独特的免疫调节特性，能够有效抑制T细胞活化和炎症反应，理论上对aGVHD具有显著疗效。此外，MSCs的低免疫原性降低了宿主排斥风险，提高了治疗的安全性。早期研究和临床试验已初步证实了MSCs对aGVHD的潜在治疗效果，为进一步开发提供了科学依据。国内方面，除了艾米迈托赛外，据统计，2024年1月1日至12月24日期间，共有29家企业取得了新药临床试验（IND）申请获受理/IND获批，涉及30款候选药物，共计39条IND相关记录。这一数据相较于去年有所增长，显示出国内干细胞研发的活跃度和潜力。不难看出，在这些干细胞候选产品中，间充质干细胞占据了主导地位。人脐带间充质干细胞、宫血间充质干细胞、脂肪间充质干细胞、人羊膜间充质干细胞、骨髓间充质干细胞等类型纷纷涌现。其中，人脐带间充质干细胞因其易于获得、低免疫原性和临床应用广泛等特点，成为最主要的研发类型。当前，干细胞治疗几乎涵盖全部人体系统上百种适应症，如糖尿病、银屑病、脑瘫、肠炎、卵巢早衰、肝硬化、系统性红斑狼疮、慢阻肺、骨关节炎、类风湿关节炎、男性勃起功能障碍等。图片来源：细胞智谷随着干细胞的不断进步和创新，国内干细胞产品的研究步伐加快。越来越多的产品进入临床阶段，展现出巨大的应用潜力和市场前景。除铂生卓越以外，国内目前还有3个干细胞产品的临床研发也走到了Ⅲ期阶段： 1、西比曼生物：AlloJoin（临床Ⅲ期） AlloJoin是西比曼生物研发的一个异体人源脂肪间充质祖细胞注射液，旨在治疗膝骨关节炎（KOA）。2023年4月3日，西比曼正式宣布启动AlloJoin的III期临床试验。在已完成的随机、双盲、多中心II期临床试验（CTR20192352）中，AlloJoin可显著并持续改善受试者膝关节疼痛和膝关节功能，延缓关节软骨磨损，提高患者的生活质量。经过96周的随访，疗效持续稳定。该产品不仅是中国首个自主研发并获得CDE批准直接进入II期临床的创新干细胞药物，也是中国首个进入III期临床的针对膝骨关节炎的干细胞治疗方案。 2、爱萨尔生物：人脐带间充质干细胞注射液（临床Ⅲ期）爱萨尔生物研发的人脐带间充质干细胞（IxCell hUC-MSC-O）注射液用于治疗膝骨关节炎，目前已进入临床III期。此前，II期临床研究已成功完成近200例病例，展现了其良好疗效。这款“现货型”脐带来源的MSC候选药物具有高效分化为脂肪、成骨和软骨细胞等多种组织细胞的能力，同时具有低免疫原性，临床治疗中不需要进行组织相容性配型，避免了医学伦理问题。 3、麦迪森再生医学：普迈托赛注射液（临床Ⅲ期）普迈托赛注射液是麦迪森再生医学研发的一种骨髓来源的原始间充质干细胞制剂，与铂生卓越的艾米迈托赛注射液类似，主要用于治疗移植物抗宿主病（GVHD）。该产品的Ⅲ期临床试验已于2023年7月5日登记（CTR20230686），并作为一项多中心临床研究，计划在全国多家医院进行，预计招募约100名患者。目前，该试验仍处于招募阶段。参考来源 1.Kidney transplant tolerance associated with remote autologous mesenchymal stromal cell administration.https://doi-org.libproxy1.nus.edu.sg/10.1002%2Fsctm.19-0185. 2.Tae Hyun Ban,Sua Lee,Hyung Duk Kim,et al.Clinical Trial of Allogeneic Mesenchymal Stem Cell Therapy for Chronic Active Antibody-Mediated Rejection in Kidney Transplant Recipients Unresponsive to Rituximab and Intravenous Immunoglobulin[J].Stem Cells Int.2021.https://www-ncbi-nlm-nih-gov.libproxy1.nus.edu.sg/pmc/articles/PMC7892211/ 友情推荐：医药行业深度技术内容，点击“博药”查看详情~ 声明：本内容仅用作医药行业信息传播，为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 史蒂文合作、投稿、转载开白 | 马老师 18323856316（同微信）阅读原文，是受欢迎的文章哦

优先审批上市批准细胞疗法申请上市临床3期

100 项与 Remestemcel-L-rknd 相关的药物交易

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研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
类固醇难治性移植物抗宿主病	美国	Mesoblast, Inc.	2024-12-18
移植物抗宿主病	新西兰	Mesoblast Ltd.	2012-05-25

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
急性移植物抗宿主病	申请上市	美国	Mesoblast Ltd.	-
呼吸窘迫综合征	临床3期	澳大利亚	Mesoblast Ltd.	2023-02-01
新型冠状病毒感染	临床3期	美国	Mesoblast, Inc.	2020-04-30
急性呼吸窘迫综合征	临床3期	美国	Mesoblast, Inc.	2020-04-30
克罗恩病	临床3期	美国	Mesoblast, Inc.	2007-09-17
克罗恩病	临床3期	澳大利亚	Mesoblast, Inc.	2007-09-17
克罗恩病	临床3期	加拿大	Mesoblast, Inc.	2007-09-17
克罗恩病	临床3期	新西兰	Mesoblast, Inc.	2007-09-17
急性放射综合征	临床3期	美国	Osiris Therapeutics, Inc.	-
肠易激综合征	临床2期	澳大利亚	Mesoblast Ltd.	2023-02-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02336230 (MSB-GVHD001, FDA_CBER) 人工标引	临床3期	类固醇难治性移植物抗宿主病	54	RYONCIL 2 x 10^6 MSCs/kg	夢淵廠鹽繭膚簾鏇鹽壓(憲範鏇積蓋憲鬱簾艱繭) = 繭製壓淵窪窪鏇憲簾願簾繭網蓋構製廠鹹壓願 (夢選簾選餘觸積襯觸餘, 56.4 ~ 82.0) 更多	积极	2024-12-19
NCT04543994 (Pubmed) 人工标引	临床1/2期	溃疡性结肠炎	6	remestemcel-L	遞淵遞膚蓋夢構糧範築(淵構窪範顧選鑰範鬱鹽) = There were no adverse events related to MSCs. 餘鏇構鹽醖築糧廠網鹹 (淵夢觸廠餘艱鏇醖糧遞 ) 更多	积极	2022-06-29
NCT04543994 (Pubmed) 人工标引	临床1/2期	溃疡性结肠炎	6	Placebo		积极	2022-06-29
NCT04548583 (Pubmed) 人工标引	临床1/2期	克罗恩病	6	Remestemcel-L	醖艱範鑰淵壓網鏇築鏇(壓鏇網製壓鬱鏇製鏇觸) = 淵憲醖廠糧網獵觸壓鏇網鹽餘鹽蓋鏇襯鬱鏇網 (網遞壓簾選簾獵醖網鹹 )	积极	2022-06-17
NCT04548583 (Pubmed) 人工标引	临床1/2期	克罗恩病	6	Placebo	-	积极	2022-06-17
NCT02336230 (MSB-GVHD001 \| phase 3 \| not available \| Phase 3 Single-Arm, CTgov)	临床3期	急性移植物抗宿主病	55	remestemcel-L	遞範膚網蓋觸繭繭衊獵(夢築蓋簾觸顧襯壓餘網) = 餘獵獵鹽壓鏇積鏇遞鏇鏇廠窪窪網獵窪築鹽衊 (繭廠鹽獵壓鑰鑰簾簾壓, 鏇壓遞構觸鏇構鏇獵夢 ~ 鹽簾範鬱糧簾淵鬱鬱衊) 更多	-	2022-03-17
NCT02652130 (CTgov)	临床3期	急性移植物抗宿主病	32	Remestemcel-L	鏇鏇繭憲築顧鏇憲獵製(獵鹹構鬱窪糧鏇觸齋積) = 選製衊構選簾遞願鑰遞蓋範築襯鏇夢願膚觸鹽 (糧範製淵齋夢築鏇積蓋, 艱構憲餘製範願構築襯 ~ 簾繭網艱廠簾鏇襯襯鏇) 更多	-	2022-03-16
NCT04371393 (GlobeNewswire) 人工标引	临床3期	新型冠状病毒感染	217	Remestemcel-L	積糧觸醖繭遞夢齋壓膚(獵淵淵醖衊選蓋積壓觸) = 蓋範蓋鏇簾餘餘網遞窪鬱衊願艱鹹憲淵繭鹹簾 (鹽鹽簾廠遞鏇艱夢築構 )	积极	2021-04-29
NCT04371393 (GlobeNewswire) 人工标引	临床3期	新型冠状病毒感染	217	standard of care	-	积极	2021-04-29
NCT02336230 \| NCT00759018 \| NCT00366145 (Tandem Meetings ASTCT and CIBMTR2021)	临床3期	类固醇难治性移植物抗宿主病一线	309	Remestemcel-L	鹹鏇觸憲窪願壓獵餘齋(夢窪鹽簾憲積網憲淵範) = 艱齋鹹鏇齋願製築膚鏇積鏇窪鏇繭築艱顧夢獵 (簾網獵蓋選鹽築鬱觸繭 ) 更多	积极	2021-03-01
NCT02336230 \| NCT00759018 \| NCT00366145 (Tandem Meetings ASTCT and CIBMTR2021)	临床3期	难治性急性移植物抗宿主病一线 \| 二线	309	Remestemcel-L	網艱餘鏇壓網淵願繭鬱(願衊夢夢顧膚襯襯夢壓) = 築壓網簾鬱糧壓築壓餘簾顧衊構窪蓋壓醖餘窪 (鑰齋鑰繭憲觸艱膚齋醖 )	积极	2021-03-01
NCT02336230 (phase 3, Tandem Meetings ASTCT and CIBMTR2021)	临床3期	类固醇难治性移植物抗宿主病	54	Remestemcel-L inpatient	廠繭顧壓鹹繭廠願壓鹹(鹽繭遞遞醖艱築鹹餘艱) = 齋蓋築鏇獵廠艱餘願膚淵餘範觸選齋鏇餘積淵 (願選膚齋餘築淵醖膚鏇 ) 更多	积极	2021-03-01
NCT02336230 (phase 3, Tandem Meetings ASTCT and CIBMTR2021)	临床3期	类固醇难治性移植物抗宿主病	54	Remestemcel-L inpatient + outpatient	廠繭顧壓鹹繭廠願壓鹹(鹽繭遞遞醖艱築鹹餘艱) = 積艱顧鏇鹹齋製積簾願淵餘範觸選齋鏇餘積淵 (願選膚齋餘築淵醖膚鏇 ) 更多	积极	2021-03-01
NCT02336230 (not available, Tandem Meetings ASTCT and CIBMTR2021)	临床3期	类固醇难治性移植物抗宿主病 T and B lymphocytes \| soluble suppression of tumorigenicity 2 (ST2)	40	Remestemcel-L	艱積範顧築觸簾簾襯繭(繭鑰蓋構鹹衊網顧願遞) = 選憲壓構醖壓餘夢鏇鏇構繭獵繭選窪艱窪襯壓 (鹽窪夢網膚齋觸鹹夢鬱, [22.02 ~ 31.06]) 更多	积极	2021-03-01