Darvadstrocel(TiGenix)

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

DUBLIN--( BUSINESS WIRE )--The "Mesenchymal Stem Cells / Medicinal Signaling Cells (MSCs) - Advances & Applications, 2025" report has been added to ResearchAndMarkets.com's offering. MSCs are multipotent cells that have been referred to as mesenchymal stem cells, medicinal signaling cells, and mesenchymal stromal cells, reflecting their versatility and diverse applications. MSCs can differentiate into a variety of cell types, such as osteoblasts, chondrocytes, myocytes, and adipocytes. Their unique ability to secrete factors that promote tissue repair and modulate their microenvironment with anti-inflammatory and anti-fibrotic effects makes them essential in regenerative medicine. Furthermore, their immuno-evasive properties make MSCs particularly suitable for allogenic transplantation. MSCs serve as vital raw materials across numerous industries, including regenerative medicine, cosmeceuticals, and cultured meat production. Key applications include: Cell-based therapies for musculoskeletal diseases, spinal injuries, and cardiovascular conditions. Engineered tissues and organs, such as skin, bone, blood vessels, and liver. Cell-derived products, such as exosomes and growth factors, utilized in cosmetics and skincare. Cultured meat production, leveraging MSCs' role in muscle development and their ability to differentiate into muscle and fat cells. Globally, 12 MSC-based therapies have been approved for various indications, with the majority concentrated in South Korea, Japan, and the EU. Specifically, the Republic of Korea has approved five products: Queencell from Anterogen, Cellgram AMI from Pharmicell, Cupistem from Anterogen, Cartistem from Medipost, and NeuroNataR from Corestem. Japan has approved two products: Temcell HS from JCR Pharmaceuticals and Stemirac from Nipro Corporation. The EMA in Europe has approved two products: Holoclar from Chiesi Farmaceutici and Alofisel from TiGenix/Takeda. India has approved one product: Stempeucel from Stempeutics. Iran has approved one product: MesestroCell developed by Cell Tech Pharmed. Finally, Australia has approved one product: Remestemcel-L from Mesoblast. Despite this progress, no MSC-based therapeutic have yet received U.S. FDA approval, although the FDA is actively reviewing Mesoblast's Remestemcel-L. The cosmeceutical market is also rapidly adopting MSC-derived products, particularly exosomes, to leverage their anti-aging and regenerative properties, with companies like L'Oreal and Johnson & Johnson investing heavily in this space. Similarly, cultured meat companies are utilizing MSCs for producing muscle and fat cells, enhancing taste and texture. Groundbreaking approvals, such as lab-grown chicken by Upside Foods and Good Meat, have paved the way for further growth in this industry. MSC-based gene editing is expanding, enabling the overexpression of antitumor genes and therapeutic factors. Researchers are also exploring the use of MSCs in 3D printing and engineered organ production, with breakthroughs anticipated in the next decade. Importantly, companies like Cynata Therapeutics are pioneering iPSC-derived MSC production technologies, enabling large-scale therapeutic development. Today there are at least eight companies who are involved with the development of iPSC-derived MSCs therapeutics (iMSCs), including Cynata Therapeutics, Eterna Therapeutics, Implant Therapeutics, Bone Therapeutics, Brooklyn ImmunoTherapeutics, Fujifilm CDI, Citius Pharmaceuticals, and Kiji Therapeutics. There are also over 1,670 clinical trials involving MSCs are registered on ClinicalTrials.gov. Nearly 75% of these clinical studies are using MSCs for the development of regenerative medicine (RM) products. Approximately 14% of the studies are using MSCs for disease modeling. The remaining 11% of the studies are using MSCs for drug discovery and cytotoxicity testing applications. Challenges in these trials include maintaining MSC phenotype and functionality during expansion. However, ongoing advancements suggest a promising future, with projections for at least 50 globally approved MSC-based products by 2040, averaging 3-4 new approvals annually. Key Players in MSC Research Products The demand for MSC-based research products has surged in recent years, with major suppliers expanding their portfolios. Companies like RoosterBio have increased the access to quality MSCs through supply chain industrialization, standardized cell bank product forms, and fit-for-purpose cGMP-compatible cells and media systems. Other leading MSC research product suppliers include PromoCell, Lonza, Thermo Fisher Scientific, STEMCELL Technologies, Miltenyi Biotec, Bio-Techne (R&D Systems), ScienCell Research Laboratories, and the ATCC. Challenges and Opportunities While MSC commercialization faces hurdles-such as scalability and regulatory approval-opportunities abound. The market for MSC-based products is poised for notable growth across the research, therapeutic, cosmetic, and food industries. Within this rapidly changing landscape, having a thorough understanding of the competition is essential. This global strategic report presents rates of MSC clinical trials, scientific publications, patents, funding events, and collaborations. It presents the pipeline for MSC-based therapeutics, identifies market opportunities and threats, and explores future directions and emerging applications. It reveals innovative MSC products, services, and technologies offered by 125 leading competitors. Finally, it presents detailed market size figures for the global MSC market, segmented by geography and business segment with future forecasts through 2031. Key Topics Covered: MESENCHYMAL STEM CELLS (MSCs): AN OVERVIEW Stabilization of hMSCs as a Technology The Impact of MSCs on Regenerative Medicine Timeline of MSC Nomenclature Sources of MSCs Cell Surface Markers in MSCs In vitro Differentiation Potentials of MSCs Soluble Factors Secreted by MSCs Stemness Genes Present in MSCs Types of MSCs and their Biomedical Applications Genes that are Pivotal for MSC's Properties MSC-BASED CLINICAL TRIAL LANDSCAPE MSC-Based Clinical Studies by Geography Sources of MSCs used in Current Clinical Trials Share of Autologous & Allogeneic MSCs Clinical Trials MSC-Based Clinical Trials by Disease Indication MSC-Based Clinical Trials by Phase of Study Select MSC-Based Clinical Trials MESENCHYMAL STEM CELLS (MSCS): PATENT LANDSCAPE MSC Patent Publications by Jurisdiction MSC Patent Applicants MSC Patent Inventors MSC Patent Owners Legal Status of MSC Patent Applications MSC-Derived Exosome Patents Patents for iPSC-Derived MSCs MSC Patents by Therapy Type PUBLISHED SCIENTIFIC PAPERS ON MSCS MSC MANUFACTURING PLATFORMS: AN OVERVIEW Methods of Isolations of MSCs Cell Expansion: Conventional Cultures to Bioreactors Downstream Processing of MSCs Autologous & Allogeneic MSC Manufacturing: A Comparison MSC Manufacturing Economics in Small Scale Large Scale Manufacturing of MSCs CELL & GENE THERAPY CONTRACT MANUFACTURING Staff Shortage Need for Automation Capacity Constraints CDMO Platforms with Greater Potentials CMOs/CDMOs Geographic Distribution of Manufacturing Facilities Continued Rise of Outsourcing Major CDMOs in the U.S Major Cell & Gene Therapy CDMOs in Europe U.K.'s Domination in European Contract Manufacturing BIOENGINEERING SOLUTIONS TO BOOST MSC'S FUNCTIONS Small Molecule Priming of MSCs Particle Engineering of MSCs Genetic Engineering of MSCs Examples of Bioengineered MSCs to Produce Anticancer Therapeutics Engineered MSCs for Neurological Conditions Engineered MSCs for Cardiovascular Diseases Engineered MSCs for Lung Injury Engineered MSCs for Diabetes Bioengineering Approaches to Improve MSC Administration MAJOR DISEASES ADDRESSED BY MSCS Studies Using MSCs for Autoimmune Diseases Studies using MSCs for Cardiovascular Diseases Studies using MSCs for Neurodegenerative Diseases Studies using MSCs for Bone & Cartilage Diseases Studies using MSCs in GvHD Studies using MSCs in Crohn's Disease Studies using MSCs in Type 1 Diabetes Studies using MSCs in Systemic Lupus Erythematosus (SLE) Studies using MSCs in Parkinson's Disease Studies using MSCs in Alzheimer's Disease Studies using MSCs in Kidney Failure Studies using MSCs in Spinal Cord Injury Studies using MSCs for Wound Healing RECENT COLLABORATIONS IN MSC SECTOR, 2020-2024 Collaboration between BioSolution Designs & RoosterBio Collaboration between CytoMed Therapeutics, Ltd. & Sengkang General Hospital Collaboration between REPROCELL & Histocell Collaboration between FUJIFILM Dyosynth & RoosterBio RoosterBio's Collaboration with AGC Biologics RoosterBio's Partnership with ShiftBio RoosterBio's Partnership with Univercells Technologies Collaboration between Cynata & Fujifilm Collaboration between American CryoStem Corp. & BioTherapeutic Lab Corp. RoosterBio's Collaboration with Sartorius Collaboration between American CryoStem and CRADA RoosterBio's Collaboration with Sartorius Korea Biotech Collaboration between Catalent & BrainStorm Collaboration between Cipla & Stempeutics Aethlon's Collaboration with University of Pittsburgh RoosterBio's Collaboration with Senti Biosciences MSC-BASED PRODUCTS IN THE MARKET Approved MSC Therapies Queencell Cellgram AMI Cupistem Cartistem NeuroNataR Holoclar Remestemcel-L Temcell HS Stempeucel Alifosel MesestroCell Stemirac Marketed Biomaterial Carrier-Based MSCs & MSC Progenitors Osteocel AlloStem Grafix Cellentra VCBM Trinity ELITE Map3 BIO4 Trinity Evolution Carticel Chondron DeNovo NT Chondrocelect Ossron JACC MACI Ortho-ACI Ossgrow Cartigrow Topical Cosmetic Products Containing MSCs/MSC-Derived Exosomes MARKET ANALYSIS Market Size for MSC Therapies Global Demand for Mesenchymal Stem Cells (MSCs) COMPANY PROFILES Advancells Aegle Therapeutics AGC Biologics AlloSource, Inc. Ambulero American CryoStem Corporation American Type Culture Collection (ATCC) Amniotics Andelyn Biosciences Anemocyte S.r.l Anterogen, Co., Ltd. Avid Bioservices, Inc. Bacthera Baylx, Inc BioCardia BioCentriq BioEden, Inc Bioinova s.r.o BioRestorative Therapies Bioscience Institute S.p.A Blue Horizon International Boehringer Ingelheim BioXellence Bonus Biogroup, Ltd BrainStorm Cell Therapeutics, Inc. Catalent CCRM Cell2Cure ApS Cell Care Therapeutics Cellcolabs CELLeBRAIN Cellipont Bioservices Celprogen, Inc CellProthera CellResearch Corporation, Pte, Ltd. Cell Surgical Network (CSN) Celltex Therapeutics Corporation CellTherapies Cellular Biomedicine Group Charless River Laboratories CHIESI Farmaceutici S.p.A Citius Pharmaceuticals, Inc CorestemChemon, Inc. Creative Bioarray Creative Medical Technology Holdings, Inc Curia Global, Inc Cynata Therapeutics, Ltd. Cytovance Biologics Defined Bioscience, Inc Direct Biologics eQcell, Inc. Excellos Exosla Therapeutics EXOSOMEplus Exothera Fujifilm Diosynth Biotechnologies Future Cell Japan, Co., Ltd. Genezen GenScript Biotech Corporation Hope Biosciences, Implant Therapeutics, Inc INCELL Corporation InGeneron Invitrx Therapeutics Japan Tissue Engineering, Co., Ltd. (J-TEC) JCR Pharmaceuticals, Co., Ltd. Jointechlabs Kangstem Biotech, Co., Ltd. Kimera Labs LifeCell Longeveron Lonza Group, Ltd Lorem Cytori USA, Inc. MEDPOST Mesoblast, Ltd Millipore Sigma NecstGen NextCell NIPRO Corporation Novadip Biosciences Novus Biologicals NuVasive OCT Therapies & Research Pvt., Ltd OmniaBio, Inc Orthocell, Ltd Orthofix Medical, Inc. Ossium Health Personal Cell Sciences Corporation Personalized Stem Cells, Inc. Pfizer CentreOne Pluri Biotech, Ltd. Porton Advanced Prometheus Life Technologies PromoCell PuREC, Co., Ltd Regrow Biosciences Reliance Life Sciences Remedy Cell Rentschler Biopharma REPROCELL USA Resilence RESTEM RHEACELL Richter-HELM RoosterBio RTI Surgical RoslinCT Samsung Biologics San Bio, Co., Ltd. Smith & Nephew, Inc StemBioSys, Inc STEMCELL Technologies, Inc Stemedica Cell Technologies, Inc Stemmatters StemMedical Stempeutics Research Pvt., Ltd StromaBio TaiwanBio Therapeutics, Co., Ltd Takeda Pharmaceutical, Co., Ltd Tempo Bioscience ThermoFisher Scientific Vericel Corporation Wuxi App Tec Xintela For more information about this report visit https://www.researchandmarkets.com/r/upnyhu About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.