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 项与瑞昂西尔相关的临床结果

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100 项与瑞昂西尔相关的转化医学

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100 项与瑞昂西尔相关的专利（医药）

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项与瑞昂西尔相关的文献（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 ; Irikura, Tomoya ; Watakabe, Mai ; 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.

2023-01-01·Advances in experimental medicine and biology

Illustrative Potency Assay Examples from Approved Therapies.

Article

作者: Torrents, Sílvia ; Grau-Vorster, Marta ; Vives, Joaquim

Advanced therapy medicinal products (ATMP) encompass a new type of drugs resulting from the manipulation of genes, cells, and tissues to generate innovative medicinal entities with tailored pharmaceutical activity. Definition of suitable potency tests for product release are challenging in this context, in which the active ingredient is composed of living cells and the mechanism of action often is poorly understood. In this chapter, we present and discuss actual potency assays used for the release of representative commercial ATMP from each category of products (namely, KYMRIAH^® (tisagenlecleucel), Holoclar^® (limbal epithelial stem cells), and PROCHYMAL^®/RYONCIL™ (remestemcel-L)). We also examine concerns related to the biological relevance of selected potency assays and challenges ahead for harmonization and broader implementation in compliance with current quality standards and regulatory guidelines.

2022-11-01·Colorectal disease : the official journal of the Association of Coloproctology of Great Britain and Ireland

A phase IB/IIA study of remestemcel‐L, an allogeneic bone marrow‐derived mesenchymal stem cell product, for the treatment of medically refractory ulcerative colitis: an interim analysis

Article

作者: Ream, Justin ; Dadgar, Neda ; Khaitan, Neha ; Fulmer, Clifton ; Nachand, Douglas ; Steele, Scott R. ; Elliott, Kavita ; Lightner, Amy L. ; Matyas, Caroline

Abstract:

Aim:

There have been no studies into the direct injection of mesenchymal stem cells (MSCs) for luminal ulcerative colitis (UC). Our aim was to investigate the efficacy of MSCs delivered locally via endoscopic delivery, as is done in the setting of perianal disease, to treat the local site of inflammation directly.

Method:

A phase IB/IIA randomized control clinical trial of remestemcel‐L, an ex vivo expanded allogeneic bone marrow‐derived MSC product, at a dose of 150 million MSCs versus placebo (2:1 fashion) delivered via direct injection using a 23‐gauge sclerotherapy needle at the time of colonoscopy was designed to assess the safety and efficacy of endoscopic delivery of MSCs for UC. The main outcome measures were adverse events, Mayo score and Mayo endoscopic severity score at 2 weeks, 6 weeks and 3 months post‐MSC delivery.

Results:

Six patients were enrolled and treated; four received MSCs and two placebo. All had been on prior anti‐tumour necrosis factor or anti‐integrin therapy. There were no adverse events related to MSCs. In the treatment group (n = 4), the Mayo endoscopic severity score decreased in all patients by 2 weeks after MSC delivery. At 3 months, all patients were extremely satisfied or satisfied with their MSC treatment based on the inflammatory bowel disease patient‐reported treatment impact (IBD‐PRTI), and treatment response was described as excellent or good in all patients. In the control group (n = 2), the Mayo endoscopic severity score did not increase as a result of being off alternative therapy. At 3 months, patients were dissatisfied according to the IBD‐PRTI, and treatment response was poor or unchanged.

Conclusion:

MSCs may offer a safe therapeutic option for the treatment of medically refractory UC. Early data suggest improved clinical and endoscopic scores by 2 weeks after MSC delivery.

192

项与瑞昂西尔相关的新闻（医药）

2025-05-28

·Cytiva学堂

Ficoll-Paque应用解析：间充质干细胞的分离

Cytiva智荟专线400-810-9118，2号键#智荟锦囊上一期，智荟专线为大家介绍了Cytiva Ficoll-Paque PLUS（1.077 g/mL）密度梯度介质的组成和分离外周血单个核细胞（PBMCs）的机理。并提示了病人样本特殊性，主要表现在淋巴细胞减少以及低密度的未成熟粒细胞可能会污染白膜层1、2，后者可以通过anti-CD15磁珠去除3、4。相关阅读：Ficoll-Paque应用解析：PBMCs分离中病人样本的特殊性本期，我们将继续为大家介绍更低密度的Ficoll-Paque PREMIUM 1.073（g/mL）用于人间充质干细胞分离的优势。人间充质干细胞治疗市场分析根据Dimension Market Research【5】在2025年3月份发布的报告（Report Code : RC-1217），全球干细胞治疗市场规模预计将由2024年的189亿美元激增至2033年的547亿美元，即复合年均增长率CAGR为12.6%，如图1。其中，占比重最大的为成体干细胞（蓝色部分），主要包括造血干细胞以及各种组织来源的间充质干细胞等，其易于获取、疗效佳、不易癌变，且无伦理风险。图1：全球干细胞治疗市场预测（Dimension Market Research）近期，间充质干细胞的上市申请也捷报连连。2024年12月18日，Mesoblast Limited公司的Ryoncil（remestemcel-L）成为全球首个被FDA批准的间充质干细胞疗法6，用于治疗2个月及以上儿童患者的类固醇难治性急性移植物抗宿主病（SR-aGVHD），作为全球首个“现货型”、同种异体、骨髓间充质基质细胞疗法，也为其它hMSCs细胞治疗产品树立了范例。15天后，2025年 1月2日，国家药品监督管理局（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治疗药品艾米迈托赛注射液（商品名：睿铂生）上市7，其用于治疗14岁以上消化道受累为主的激素治疗失败的急性移植物抗宿主病（aGVHD），作为处方药，是一款人脐带间充质干细胞注射剂，历经12年潜心研发，也是我国首款获批的干细胞药物。前述两个里程碑式的细胞治疗药物，都利用了hMSCs所具有免疫调节和组织修复功能，用于治疗移植物抗宿主病。紧接着，2025年3月22日，根据第二届博鳌乐城干细胞大会报告，三项干细胞相关转化应用项目得到批准，其中两项为间充质干细胞相关，包括脐带间充质干细胞注射液治疗膝骨关节炎技术、改善射血分数减低型心力衰竭的脐带来源间充质干细胞治疗技术。早在1974年，Friedenstein AJ等8通过塑料黏附特性，从骨髓中首次分离出人类间充质干细胞（Human Mesenchymal Stem Cells, hMSCs），后续科学家又从胎盘、脐带、脂肪组织、羊膜、宫血、牙髓等获得hMSCs，其主要优势：hMSCs可在一定代次内进行自我复制并维持未分化状态，具有多分化潜能可用于组织修复9和再生医学。hMSCs通过分泌细胞因子及胞外囊泡发挥抗炎10及免疫调节作用，并被招募到炎症或损伤部位11，可用于免疫性疾病治疗、移植物抗宿主病和促进组织重建（如神经系统）。又因其免疫原性低，具有跨MHC屏障移植12的特性，单一供体可惠及众多患者。此外，hMSCs还可以迁移到肿瘤微环境中，作为药物或基因治疗载体13、14、15，其外泌体16在药物递送方面同样备受瞩目。人间充质干细胞hMSCs分离方法间充质干细胞的分离方法也在不断“进化”：1974年Friedenstein AJ等8使用贴壁法分离骨髓间充质干细胞并沿用至今，后续又发展出密度梯度离心17、组织爬出法18、酶消化法19、红细胞裂解法20、免疫磁珠吸附21、流式细胞术22等，不同组织来源的hMSCs适用的分离方法稍有差异23，也可以组合使用。其中，密度梯度离心法为常用技术之一17，又以Cytiva Ficoll-Paque分离试剂最为经典，通常用于骨髓、脐带、脐带血、胎盘、关节滑膜液等样品23。FICOLL-PAQUE PREMIUM 1.073用于hMSCs分离2008年，Cytiva上市了Ficoll-Paque PREMIUM 1.073 （g/mL），其密度低于常规的Ficoll-Paque PLUS（1.077 g/mL），主要设计用于间充质干细胞或者低密度单核细胞（monocytes）的分离。Ficoll-Paque PREMIUM在严格控制的环境下生产，符合ISO 13485:2003，符合欧盟委员会GMP 指南无菌药品生产要求，依从美国药典章节<1043>的相关规定，因此推荐用作临床研究和细胞治疗24，提供RSF法规支持文件。产品上市同年，2008年9月，Gary Brooke等25即发表文章，将Ficoll-Paque PREMIUM 1.073用于人胎盘间充质干细胞的分离，将胎盘先剪成小块，再使用酶法孵育（100 U/ml I型胶原酶 + 5 μg/ml DNase I），最终获得约30 ml HBSS重悬的单细胞悬液，并在下层铺设12 mL Ficoll-Paque密度梯度介质，540 g离心20 min（20℃，关闭刹车），取白膜层，用HBSS清洗两次，使用完全培养基重悬细胞并接种于8个T175培养瓶，经过连续4次传代到P5，每次接种于90个T175细胞培养瓶（剩余细胞全部冻存），如图2，并最终用于临床试验中。图2：Ficoll-Paque PREMIUM 1.073用于胎盘来源人间充质干细胞的分离和传代扩增紧接着，2010年，GIULIA GRISENDI等26系统地比较了Ficoll-Paque PREMIUM（1.077）及更低密度的Ficoll-Paque PREMIUM 1.073在骨髓间充质干细胞分离中的差异，以期建立符合GMP要求的标准化流程。骨髓来源间充质干细胞（Bone marrow mesenchymal stem cells，BM-MSCs）由于广泛的分化潜能，研究最多，其在再生医学、肿瘤基因治疗、免疫性疾病中得到广泛关注和应用，但是骨髓中hMSCs占比很低（0.001 – 0.01%）26，因此需要分离和扩增后方可进行研究及临床应用。文章使用13位肿瘤病人的样本，年龄6-62岁（Ave. 48岁），来自9位男性4位女性。每位患者取髂后嵴，0.5-1 mL柠檬酸钠冲出骨髓，使用无钙镁PBS对半稀释，吹散，分别取等份的2.3 mL样品，铺到Ficoll-Paque PREMIUM（1.077或1.073 g/mL）的表面，参考试剂说明书的标准程序进行分离，623 g离心20 min，取白膜层PBS清洗两次，这部分即为单个核细胞MNCs层，其中含BM-MSCs。使用无血清培养基，以800 000 cells/cm2接种密度铺6孔板，每2-3天换液，去除悬浮细胞。待长到80-90%汇聚后，胰酶消化，并以5000 cells/cm2传代，至P4。所有实验设置2组重复。使用Ficoll-Paque PREMIUM 1.077和1.073分离BM-MSCs，细胞收率无显著差异，活率高（≥95%），如图3所示。说明分离有效，且无细胞毒性。图3：使用Ficoll-Paque PREMIUM1.077和1.073分离BM MS后，细胞收率（A）和7-AAD染色后的活力数据（B）根据国际细胞治疗学会（ISCT）于2006年36提出的最低鉴定标准，hMSCs需至少具有三个方面特征： 1 形态学（纺锤形贴壁）； 2 表面标记物（表达如CD90, CD73, CD105, CD146和GD2，且造血标记分子阴性）； 3 多分化潜能（体外特定条件下分化为间质细胞，即成骨、成软骨、成脂分化）。因此，文章中对BM-MSCs进行了流式细胞分选（FACS）分析、体外诱导三系分化，并检测CFU-F（成纤维细胞集落形成单位）的数量。根据流式细胞术分析结果，相对于仅使用裂红试剂去除红细胞的对照样品（Lysis组），Ficoll-Paque PREMIUM 1.077和1.073均显著降低了白细胞共同抗原CD45+细胞（如淋巴细胞和粒细胞）的比例，CD14+单核细胞也有降低但差异不大，同时，CD105+ 目的细胞的比例有显著提高。1.077和1.073二者相比，CD45+杂质细胞的去除效果，后者更佳且具有显著性差异，其它阳性CD分子的比例均有不同程度的提高但差异不显著，如图4。推测由于Ficoll-Paque PREMIUM 1.073（g/mL）的密度比1.077更低，使得比BM-MSCs密度更高的淋巴细胞、粒细胞沉于1.073 g/mL分离液之下，因此白膜层BM-MSCs纯度更高；如果使用1.077 g/mL分离液，则这些细胞均位于白膜层中。使用Cytiva Percoll细胞分离介质自行配制低密度分离液，具有类似效果27。图4：BM-MSCs免疫表型结果（流式细胞术）。Lysis为仅用红细胞裂解液处理（未进行密度分离），作为对照。样品为使用对应密度梯度介质分离后立即检测，未经过培养。贴壁培养15天后，计数CFU-F克隆形成单位（＞50个细胞）。使用两种分离液获得的细胞，形态正常，克隆大小相似，但相比于1.077，1.073分离的BM-MSCs具有显著更高的CFU-F数量（7.9±3.3 X 10^6 cells），达到1.5倍差异。1.073分离后，平均1.2 X 10^5 BM cells形成一个克隆，与健康人类似28。这点对于实验中使用的肿瘤病人样本，由于疾病和治疗造成微环境受损，MSC前体细胞数量本来就较少29，意义更加凸显。此外，传代培养结果再次佐证了CFU-F检测到的增殖潜力差异，一开始两种密度梯度介质分离的BM-MSCs增殖能力相似，P1收获时，每1 mL骨髓样品大约获得10^5 BM-MSCs。但是到了P4，1.073获得的BM-MSCs数量（3.35±0.83 X 10^6）远高于1.077组（1.86±0.49 X 10^6），具有显著性差异。1.073分离的BM-MSCs，从P1到P4细胞总数量增加35.2倍，临床上如果获得至少20 mL骨髓细胞悬液，将在P4收获到29.13 X 10^6个细胞[（3.35 X 10^6 cells/2.3 mL) X 20 mL]。通过计算累积群体倍增（cumulative PD），即细胞在多次传代过程中的倍增次数，结果发现1.073在每个代次的数值均高于1.077，显示了更高的倍增潜力，这对于临床应用需要短时间获得大量细胞具有重要意义。另外，从细胞倍增时间（doubling time）来看，1.073分离的hMSCs在各个代次保持稳定，而1.077则倍增时间逐渐延长提示细胞老化（7-AAD也显示更多死细胞），如图5。图5：累积群体倍增（A）以及倍增时间（B）。对P4代次样品进行FACS分析，1.077和1.073分离培养后的样品，造血（分化）标记物分子表达均低，但是，1.073组有更多比例的细胞表达MSC Markers，如CD90、CD105、CD73和GD2，其中CD90+细胞在两组之间具有显著性差异。从平均荧光强度（Mean fluorescence intensity，MFI）来看，即细胞表面的抗原表达量，1.073组呈现出更高的CD90（增殖潜力强30）、CD146和GD2（干性强31）表达，提示其最初分离的BM-MSCs亚群即具有更高水平的MSC Markers表达量。后续，也成功进行了三系分化。综上，Ficoll-Paque PREMIUM 1.073可以有效分离BM-MSCs，相比于经典的1.077 g/mL密度介质，二者收率相当，但1.073 效果更佳，表现在： 1 收获细胞的增殖潜力更大，有利于满足临床应用需要短时间进行大量培养的需求； 2 因介质密度更低，更好地去除了其它单个核细胞的污染； 3 分离到更多具有更高分化潜能的细胞。2012年，Naser Ahmadbeigi等32进一步研究发现，由于部分BM-MSCs会形成聚集体，采用Ficoll 1.073进行分离时，可以同时回收管底细胞并裂红，继续培养获得BM-MSCs聚集体克隆33。2017年，Baghaei K等34开发出一种简单快速的从骨髓中分离、诱导分化和鉴定hMSCs的方法。通过回收Ficoll-Paque离心后的白膜层并进行贴壁培养，去除非贴壁细胞后，成功进行了三系分化以及FACS表型鉴定。2025年3月，Ester Moñivas等35发表文章再次证明Ficoll-Paque介质分离BM-MSCs的有效性，并且在GMP环境下将手工分离和Cytiva自动化封闭系统Sepax细胞处理仪进行平行比较，证明二者均有效，后者收率稍高。Cytiva干细胞整体解决方案Ficoll-Paque PREMIUM 1.073联合自动化封闭系统如Cytiva Sepax C-Pro细胞处理仪，将助力干细胞临床应用建立安全可靠、可重复的标准化操作流程。细胞分离后的放大培养，可根据需求选择Cytiva成熟工艺，包括培养基（Hyclone基础培养基、血清）、微载体（如Cytodex 1或Cytodex 3），以及硬件设备如Xuri W25 细胞扩增系统、iCELLis一次性固定床生物反应器系统或Xcellerex XDR 一次性生物反应器。另外，Cytiva中国Fast Trak可助力从头开发工艺。图6：Cytiva干细胞整体解决方案细胞分离液相关产品今日文中提到的相关资料，如Ficoll-Paque PREMIUM 1.073中文操作说明书、中文数据文件，以及Cytiva微载体、Xuri W25 细胞扩增系统、iCELLis一次性固定床生物反应器、Xcellerex XDR 产品数据文件等，欢迎扫描下方二维码即刻下载如果大家对产品和技术感兴趣，请拨打下方「智荟专线」联系我们！智荟专线：400-810-9118（转1号线询价购买或2号线技术支持）参考文献1. van den Akker EL, Baan CC, van den Berg B, et al. Ficoll-separated mononuclear cells from sepsis patients are contaminated with granulocytes. Intensive Care Med 2008;34:912–6.2. Luz E Cabrera, Pirkka T Pekkarinen, Maria Alander, et al. Characterization of low-density granulocytes in COVID-19. PLoS Pathog. 2021 Jul 6;17(7):e1009721.3. Schenz J, Obermaier M, Uhle S, Weigand MA, Uhle F. Low-Density Granulocyte Contamination From Peripheral Blood Mononuclear Cells of Patients With Sepsis and How to Remove It - A Technical Report. Front Immunol. 2021 Aug 18;12:684119. doi: 10.3389/fimmu.2021.684119. PMID: 34484182; PMCID: PMC8416421.4. Preobrazhensky SN, Bahler DW. Immunomagnetic bead separation of mononuclear cells from contaminating granulocytes in cryopreserved blood samples. Cryobiology. 2009 Dec;59(3):366-8. doi: 10.1016/j.cryobiol.2009.09.008. Epub 2009 Sep 18.5. https://dimensionmarketresearch.com/report/stem-cell-therapy-market/6. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-remestemcel-l-rknd-steroid-refractory-acute-graft-versus-host-disease-pediatric7. https://www.nmpa.gov.cn/zhuanti/cxylqx/cxypxx/20250102192741146.html8. Friedenstein AJ, Deriglasova UF, Kulagina NN, Panasuk AF, Rudakowa SF, Luri á EA, Ruadkow. Precursors for fibroblasts in different populations of hematopoietic cells as detected by the in vitro colony assay method. Exp Hematol. 1974;2:83 – 92.9. Pittenger MF, Mosca JD, McIntosh KR. Human mesenchymal stem cells: progenitor cells for cartilage, bone, fat and stroma. Curr Top Microbiol Immunol. 2000;251:3-11. doi: 10.1007/978-3-642-57276-0_1. PMID: 11036752.10. Ferrini E, Stellari FF, Franceschi V, Macchi F, Russo L, Murgia A, Grisendi G, Villetti G, Dominici M, Donofrio G. Persistency of Mesenchymal Stromal/Stem Cells in Lungs. Front Cell Dev Biol. 2021 Jul 16;9:709225. doi: 10.3389/fcell.2021.709225. PMID: 34336863; PMCID: PMC8322774.11. Karp, J. M. & Leng Teo, G. S. Mesenchymal stem cell homing: the devil is in the details. Cell Stem Cell 4, 206–216 (2009).12. Le Blanc, K., Tammik, L., Sundberg, B., Haynesworth, S.E. & Ringden, O. (2003) Mesenchymal stem cells inhibit and stimulate mixed lymphocyte cultures and mitogenic responses independently of the major histocompatibility complex. Scandinavian Journal of Immunology, 57, 11–20.13. Hong X, Miller C, Savant-Bhonsale S, Kalkanis SN. Antitumor treatment using interleukin-12 secreting marrow stromal cells in an invasive glioma model. Neurosurgery. 2009;64:1139 – 47.14. Loebinger, M. R., Eddaoudi, A., Davies, D. & Janes, S. M. Mesenchymal stem cell delivery of TRAIL can eliminate metastatic cancer. Cancer Res. 69, 4134–4142 (2009).15. Pereboeva L, Komarova S, Mikheeva G, Krasnykh V, Curiel DT. Approaches to utilize mesenchymal progenitor cells as cellular vehicles. Stem Cells. 2003;21:389 – 4016. Dong L, Pu Y, Zhang L, Qi Q, Xu L, Li W, Wei C, Wang X, Zhou S, Zhu J, Wang X, Liu F, Chen X, Su C. Human umbilical cord mesenchymal stem cell-derived extracellular vesicles promote lung adenocarcinoma growth by transferring miR-410. Cell Death Dis. 2018 Feb 13;9(2):218. doi: 10.1038/s41419-018-0323-5. PMID: 29440630; PMCID: PMC5833395.17. J.J. Minguell, A. Erices, P. Conget. Mesenchymal stem cells. Exp Biol Med (Maywood), 226 (2001), pp. 507-52018. Hendijani F. Explant culture: An advantageous method for isolation of mesenchymal stem cells from human tissues. Cell Prolif 2017; 50: e12334.19. Siclari VA, Zhu J, Akiyama K, Liu F, Zhang X, Chandra A, et al. Mesenchymal progenitors residing close to the bone surface are functionally distinct from those in the central bone marrow. Bone. 2013;53:575–86. doi: 10.1016/j.bone.2012.12.013.20. Horn P, Bork S, Diehlmann A Walenda T, Eckstein V, Ho AD, Wagner W. Isolation of human mesenchymal stromal cells is more efficient by red blood cell lysis. Cytotherapy. 2008;10: 676 – 85.21. Jiang Y, Jahagirdar BN, Reinhardt RL, Schwartz RE, Keene CD, Ortiz-Gonzalez XR, et al. Pluripotency of mesenchymal stem cells derived from adult marrow. Nature. 2002; 418:41 – 9.22. Van Vlasselaer P, Falla N, Snoeck H, Mathieu E. Characterization and purification of osteogenic cells from murine bone marrow by two-color cell sorting using anti-Sca-1 monoclonal antibody and wheat germ agglutinin. Blood. 1994 Aug 1;84(3):753-63. PMID: 7519072.23. Mushahary D, Spittler A, Kasper C, Weber V, Charwat V. Isolation, cultivation, and characterization of human mesenchymal stem cells. Cytometry A. 2018 Jan;93(1):19-31. doi: 10.1002/cyto.a.23242. Epub 2017 Oct 26. PMID: 29072818.24. Isolation of mononuclear cells Methodology and applications. Handbook. CY13988-13Nov20-HB.25. Brooke G, Rossetti T, Pelekanos R, Ilic N, Murray P, Hancock S, Antonenas V, Huang G, Gottlieb D, Bradstock K, Atkinson K. Manufacturing of human placenta-derived mesenchymal stem cells for clinical trials. Br J Haematol. 2009 Feb;144(4):571-9. doi: 10.1111/j.1365-2141.2008.07492.x. Epub 2008 Dec 5. PMID: 19077161.26. Grisendi G, Annerén C, Cafarelli L, Sternieri R, Veronesi E, Cervo GL, Luminari S, Maur M, Frassoldati A, Palazzi G, Otsuru S, Bambi F, Paolucci P, Pierfranco C, Horwitz E, Dominici M. GMP-manufactured density gradient media for optimized mesenchymal stromal/stem cell isolation and expansion. Cytotherapy. 2010 Jul;12(4):466-77. doi: 10.3109/14653241003649510. PMID: 20353309.27. Lange C, Schroeder J, Stute N, Lioznov MV, Zander AR et al. High-potential human mesenchymal stem cells. Stem Cells Dev. 2005;14:70 – 80.28. Castro-Malaspina H, Gay RE, Resnick G, Kapoor, Kapoor N, Meyers P, et al. Characterization of human bone marrow fibroblast colony-forming cells (CFU-F) and their progeny. Blood. 1980;56:289 – 301.29. Campioni D, Lanza F, Moretti S, Dominici M, Punturieri M, Pauli S, et al. Functional and immunophenotypic characteristics of isolated CD105+ and fibroblast+ stromal cells from AML: implications for their plasticity along endothelial lineage. Cytotherapy. 2003;5:66 – 79.30. Baum CM, Weissman IL, Tsukamoto AS, Buckle AM, Peault B. Isolation of a candidate human hematopoietic stem-cell population. Proc Natl Acad Sci USA. 1992;89:2804 – 8.31. Xu J, Liao WB, Gu DS, Liang L, Liu M, Du W, et al. Neural ganglioside GD2 identifi es a subpopulation of mesenchymal stem cells in umbilical cord. Cell Phis Bioch. 2009; 23:415 – 24.32. Naser Ahmadbeigi, Masoud Soleimani, Farshad Babaeijandaghi, et al. The aggregate nature of human mesenchymal stromal cells in native bone marrow. Cytotherapy. 2012 Sep;14(8):917-24.33. Reinhold J Medina, Ken Kataoka, Masahiro Miyazaki, et al. Efficient differentiation into skin cells of bone marrow cells recovered in a pellet after density gradient fractionation. Int J Mol Med. 2006 May;17(5):721-7.34. Baghaei K, Hashemi SM, Tokhanbigli S, et al. Isolation, differentiation, and characterization of mesenchymal stem cells from human bone marrow. Gastroenterol Hepatol Bed Bench. 2017 Summer;10(3):208-213. PMID: 29118937; PMCID: PMC5660271.35. Moñivas E, Aguayo C, de la Calle S, Martínez P, Repollés N, Zurita M. Comparison between manual and automated methods for the isolation of mononuclear cells and mesenchymal stem cells using ficoll: efficacy and reproducibility. Front Cell Dev Biol. 2025 Mar 21;13:1556697. doi: 10.3389/fcell.2025.1556697. PMID: 40191228; PMCID: PMC11969043.36. Dominici M, Le Blanc K, Mueller I, Slaper-Cortenbach I, Marini F, Krause D, et al. Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement. Cytotherapy.2006;8:315 – 7.本期关键词：#FicollPaque #Xuri #iCELLis #Xcellerex

细胞疗法优先审批申请上市临床结果ASH会议

2025-05-15

·GlobeNewswire-Health Care

FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®

NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. This period of statutory exclusivity means that the FDA will not approve another mesenchymal stromal or stem cell (MSC) products for this indication during the 7-year period from the approval of Ryoncil®. Separately, Mesoblast has biologic exclusivity preventing another sponsor from referencing the Ryoncil® biologic license application (BLA) until December 2036, twelve years from its first approval which would prevent market entry by a biosimilar. These statutory exclusivities are in addition to Mesoblast’s strong U.S. intellectual property position on MSC composition of matter, manufacturing and indications, including SR-aGvHD, that provide a commercial barrier to entry against competitors through 2044. About Mesoblast Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com. Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China. About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications are expected to provide commercial protection extending through to at least 2044 in major markets. About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast. Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise. Release authorized by the Chief Executive. For more information, please contact: Corporate Communications / Investors Paul Hughes T: +61 3 9639 6036 Media – Global Allison Worldwide Emma Neal T: +1 603 545 4843 E: emma.neal@allisonworldwide.com Media – Australia BlueDot Media Steve Dabkowski T: +61 419 880 486 E: steve@bluedot.net.au

细胞疗法孤儿药上市批准免疫疗法临床研究

2025-04-30

·GlobeNewswire-Health Care

Appendix 4c Quarterly Activity Report for Quarter Ended March 31, 2025

NEW YORK, April 29, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided highlights of its recent activities for the third quarter ended March 31, 2025. “We were very pleased to have made Ryoncil® (remestemcel-L) commercially available to treat children with acute GVHD within one quarter of receiving FDA approval as the first mesenchymal stromal cell (MSC) therapy approved in the US for any indication,” said Dr. Silviu Itescu, CEO of Mesoblast. “With our strong cash position we are well placed to expand Ryoncil® indications to other serious and life-threatening pediatric inflammatory diseases, and to adults with acute GvHD.” FINANCIAL HIGHLIGHTS Net operating cash spend for the quarter was US$12.7 million.Cash on hand at the end of the quarter was US$182 million (A$290 million)1. OPERATIONAL HIGHLIGHTS Ryoncil® (remestemcel-L) U.S. Launch for Steroid-Refractory Acute Graft Versus Host Disease Ryoncil® became commercially available for purchase in the United States on March 28, 2025, with Federal Medicaid coverage, and to date 15 infusion kits have been purchased for patients to start or continue their treatment course.Ryoncil® infusion kits are purchased and distributed by Cencora to enable the efficient and secure delivery of cryopreserved product to U.S. treatment centers, either directly or via a specialty pharmacy option.To date, ten priority transplant centers have been fully onboarded, five of whom have enrolled patients through the MyMesoblast™ hub.Mesoblast anticipates onboarding an additional ten priority transplant centers in the current quarter.The full team of nine key account managers (KAMs) commenced activities in the last week of April. The KAMs will accelerate onboarding of the remaining 35 priority transplant centers, accounting for 80% of U.S. pediatric transplants, and will drive the business to provide on the ground engagement with healthcare providers and administrators.Mesoblast has continued to expand coverage for Ryoncil® to over 104 million US lives insured by commercial and government payers.To date, 37 of the 51 States provide fee-for-service Medicaid coverage for Ryoncil® through Orphan Drug Lists or medical exception / prior authorization (PA) process. The remainder will come online July 1, 2025, with mandatory coverage for all 44 million lives.To assist patients and institutions with insurance coverage, financial assistance, and access programs, ensuring that no patient is left behind in receiving this potentially life-saving therapy, Mesoblast has established a patient access hub termed MyMesoblast™, where Ryoncil® is now available for ordering. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found. Revascor® (rexlemestrocel-L) for Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation Mesoblast has a Type B meeting with FDA scheduled for this quarter to discuss the accelerated approval pathway for Revascor® (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation. The meeting will be held under Mesoblast’s Regenerative Medicines Advanced Therapy (RMAT) designation for REVASCOR.In a Type B meeting last year, FDA provided guidance to Mesoblast that the company was eligible to file for accelerated approval of REVASCOR in patients with end-stage HFrEF based on the totality of data across two randomized controlled trials. FDA also guided that a single confirmatory trial in class II/III patients with ischemic HFrEF and inflammation will need to be completed after any accelerated approval is obtained.The key objectives of the meeting are to obtain FDA feedback on relevant chemistry, manufacturing & controls (CMC), alignment on potency assays for commercial product release, and Mesoblast’s proposed design and primary endpoint for the confirmatory trial.In November 2024 a publication in the prestigious peer-reviewed European Journal of Heart Failure (EJHF) reported that a single intramyocardial injection of REVASCOR results in improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation.2 This identifies the HFrEF population that is responsive to REVASCOR and will be the target of a confirmatory trial after accelerated approval, if received. Rexlemestrocel-L for Chronic Low Back Pain associated with Degenerative Disc Disease – Phase 3 Program The confirmatory Phase 3 trial of Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration is actively enrolling and treating patients at multiple sites across the U.S.FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication.This endpoint was successfully met in Mesoblast’s first Phase 3 trial. Key secondary measures include improvement in quality of life and function.A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately 50% of prescription opioid usage in the US. Significant pain reduction and opioid cessation were observed in Mesoblast’s first Phase 3 trial.FDA has designated rexlemestrocel-L a RMAT for the treatment of chronic low back pain. Corporate During the period, Mesoblast successfully completed a global private placement primarily to existing major US, UK, and Australian shareholders raising A$260 million (US$161 million).Strengthened Board of Directors with appointment of Dr. Gregory George and Ms Lyn Cobley.Mesoblast was added to the S&P Dow Jones Indices’ S&P/ASX 200 Index effective March 6, 2025, on the Australian Stock Exchange (ASX). Other Fees to Non-Executive Directors were US$50,306, consulting payments to Non-Executive Directors were Nil and salary payments to full-time Executive Directors were US$223,092, detailed in Item 6 of the Appendix 4C cash flow report for the quarter.3 From August 2023 to July 2025, our Non-Executive Directors have voluntarily reduced cash payment of their fees by 50% and Executive Directors (our Chief Executive and Chief Medical Officers) reduced their base salaries by 30%, in lieu of accepting equity-based incentives. A copy of the Appendix 4C – Quarterly Cash Flow Report for the third quarter FY2025 is available on the investor page of the company’s website www.mesoblast.com. About Mesoblast Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com. Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China. About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2041 in all major markets. About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast References / Footnotes Translated at 1A$:0.6413US$ being the March 31, 2025 rate as reported by the Reserve Bank of Australia.Perin EC. Et al. Mesenchymal precursor cells reduce mortality and major morbidity in ischaemic heart failure with inflammation: DREAM-HF. Eur J Heart Fail 2024. https://doi.org/10.1002/ejhf.3522As required by ASX listing rule 4.7 and reported in Item 6 of the Appendix 4C, reported are the aggregated total payments to related parties being Executive Directors and Non-Executive Directors. Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise. Release authorized by the Chief Executive. For more information, please contact: Corporate Communications / InvestorsPaul HughesT: +61 3 9639 6036 Media – Global Allison WorldwideEmma NealT: +1 603 545 4843E: emma.neal@allisonworldwide.com Media – AustraliaBlueDot MediaSteve DabkowskiT: +61 419 880 486E: steve@bluedot.net.au

临床3期细胞疗法加速审批财报高管变更

100 项与瑞昂西尔相关的药物交易

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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	加拿大	Mesoblast Ltd.	2014-05-02

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	餘糧憲衊鏇襯觸顧繭鏇(繭衊顧構顧膚顧艱窪願) = 艱衊衊壓齋廠壓夢繭築鹽襯顧淵壓鏇觸網製觸 (膚鬱蓋餘糧膚選遞壓獵, 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	鬱淵願襯鏇膚繭鹹壓廠(鹹鹽蓋壓窪遞鬱膚簾壓) = There were no adverse events related to MSCs. 餘獵繭鹽觸製廠廠衊顧 (壓齋壓糧蓋膚鹹餘網鹹 ) 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	餘鑰憲鏇網憲膚夢鬱窪(築構衊壓憲窪鹽繭願壓) = 襯顧觸鏇壓簾鏇鑰製鬱簾繭夢範鹹夢築廠廠鑰 (艱窪夢夢餘襯淵襯鬱願 )	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	膚鹹壓簾鏇獵獵襯遞鹹 = 選廠衊網鹹願夢範積鏇繭鏇鏇鬱糧繭範鬱獵憲 (鑰構鹹網鏇艱襯夢糧構, 鏇遞鏇鏇遞鏇網繭廠齋 ~ 齋鬱餘簾蓋鑰夢選顧繭) 更多	-	2022-03-17
NCT02652130 (CTgov)	临床3期	急性移植物抗宿主病	32	Remestemcel-L	築壓鹽糧衊鑰壓鏇網製 = 淵餘鏇遞繭製廠鬱獵選糧壓憲願鑰範壓遞衊壓 (窪積網艱選襯積鏇餘廠, 鹹遞齋範膚築淵網構憲 ~ 鹹衊選艱繭獵獵淵衊淵) 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	衊鑰艱願鏇蓋衊壓範獵(選壓廠廠遞顧壓願蓋壓) = 製鹽願鹹衊襯積襯顧夢遞淵積遞鏇網淵鏇膚餘 (餘蓋鏇獵顧淵鬱鏇壓範 )	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 (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	廠獵鹽築網襯膚憲齋範(淵積遞艱鹽醖鹽積繭顧) = 簾簾願廠鏇遞築蓋壓積願鹽餘製膚醖齋願鬱糧 (餘齋醖衊顧糧廠齋範衊 ) 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	廠獵鹽築網襯膚憲齋範(淵積遞艱鹽醖鹽積繭顧) = 製壓艱鹹艱築鹹築蓋鬱願鹽餘製膚醖齋願鬱糧 (餘齋醖衊顧糧廠齋範衊 ) 更多	积极	2021-03-01
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	餘蓋獵繭艱構選淵壓膚(壓壓衊網襯艱網襯襯膚) = 繭製鹽糧獵範窪鏇鬱鏇衊窪範鬱淵蓋夢醖製願 (觸糧鹹齋構蓋範鹹鹽襯 ) 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	醖鹽襯蓋構願鹹願衊膚(鏇齋獵艱獵壓積夢鑰蓋) = 膚醖餘網獵遞繭蓋餘簾鹽鑰鹽獵鬱廠鏇窪構顧 (選鑰製艱憲鑰衊壓夢築 )	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	餘願鏇醖艱製顧憲醖簾(鬱網淵憲製鑰範遞廠築) = 鬱簾襯餘鑰遞鹽夢糧築鑰鹽觸糧淵觸範衊簾網 (觸積壓鑰淵積憲積醖製, [22.02 ~ 31.06]) 更多	积极	2021-03-01