A Phase II Trial of Obecabtagene Autoleucel Consolidation in Adult Patients With Acute Lymphoblastic Leukemia in First Complete Remission Without Measurable Residual Disease

The researchers are doing this study to find out whether obecabtagene autoleucel (obe-cel) is an effective treatment for people with B-cell acute lymphoblastic leukemia (ALL) that is in complete remission (CR, meaning all signs of cancer are gone) with no measurable residual disease (MRD-negative, meaning there are no detectable cancer cells). Participants in this study will have received past treatment for their B-cell ALL, and their disease will be in MRD-negative CR for the first time (first MRD-negative CR).

开始日期2026-02-03

申办/合作机构

Memorial Sloan Kettering Cancer Center

[+1]

NCT07053800

/ Not yet recruiting临床2期

A Single-Arm, Open-Label, Phase II Study to Determine the Safety and Efficacy of Obecabtagene Autoleucel (Obe-cel) in Participants With Severe, Refractory Systemic Lupus Erythematosus With Active Lupus Nephritis

This trial aims to find out if obe-cel gets rid of harmful B cells that contribute to systemic lupus erythematosus (SLE)/lupus nephritis (LN) when available treatments have not worked (refractory). The trial includes only 1 group of patients (single arm), including teenage and adult patients aged 12 to 65 years. The objective is to look for benefits of obe-cel in making signs of LN completely disappear (remission) at 6 months after treatment in patients with severe, active LN. The trial will also look for other benefits of obe-cel for up to 24 months after treatment, including the percentage of patients who respond to obe-cel treatment, SLE/LN activity, time to and length of remission, and quality of life. The trial will also assess how long obe-cel stays in the body and the safety of obe-cel.

开始日期2025-09-01

申办/合作机构

Autolus Ltd.

[+1]

NCT07053059

/ Recruiting临床2期IIT

Phase 2 Study Assessing the Clinical Activity and Safety of Obecabtagene Autoleucel as a Consolidation in Patients With Newly Diagnosed High-risk B-cell Acute Lymphocytic Leukemia (ALL)

The goal of this clinical research study is to learn if obecabtagene autoleucel (obe-cel) can help to control newly diagnosed, high-risk B-cell ALL when given as consolidation therapy. Consolidation therapy is given after the first phase of treatment.

开始日期2025-08-26

申办/合作机构

The University of Texas MD Anderson Cancer Center

[+1]

100 项与欧贝妥基奥仑赛相关的临床结果

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100 项与欧贝妥基奥仑赛相关的转化医学

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100 项与欧贝妥基奥仑赛相关的专利（医药）

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项与欧贝妥基奥仑赛相关的文献（医药）

2025-10-05·CHINESE MEDICAL JOURNAL

Novel CD19 Fast-CAR-T cells vs. CD19 conventional CAR-T cells for the treatment of relapsed/refractory CD19-positive B-cell acute lymphoblastic leukemia

Article

作者: Wang, Lu ; Liu, Li ; Liu, Ligen ; Wang, Sanbin ; Zhang, Cheng ; Zhou, Jian ; Wang, Xiaoqi ; Wang, Jishi ; Ge, Jian ; Tu, Sanfang ; Tan, Xu ; Chen, Shangjun ; Li, Yuhua ; Yao, Han ; Zhang, Xi ; Yi, Hai ; Hu, Yongxian ; Chen, Guo

Abstract:

Background::

Treatment with chimeric antigen receptor-T (CAR-T) cells has shown promising effectiveness in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), although the process of preparing for this therapy usually takes a long time. We have recently created CD19 Fast-CAR-T (F-CAR-T) cells, which can be produced within a single day. The objective of this study was to evaluate and contrast the effectiveness and safety of CD19 F-CAR-T cells with those of CD19 conventional CAR-T cells in the management of R/R B-ALL.

Methods::

A multicenter, retrospective analysis of the clinical data of 44 patients with R/R B-ALL was conducted. Overall, 23 patients were administered with innovative CD19 F-CAR-T cells (F-CAR-T group), whereas 21 patients were given CD19 conventional CAR-T cells (C-CAR-T group). We compared the rates of complete remission (CR), minimal residual disease (MRD)-negative CR, leukemia-free survival (LFS), overall survival (OS), and the incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) between the two groups.

Results::

Compared with the C-CAR-T group, the F-CAR-T group had significantly higher CR and MRD-negative rates (95.7% and 91.3%, respectively; 71.4% and 66.7%, respectively; P = 0.036 and P = 0.044). No significant differences were observed in the 1-year or 2-year LFS or OS rates between the two groups: the 1-year and 2-year LFS for the F-CAR-T group vs. C-CAR-T group were 47.8% and 43.5% vs . 38.1% and 23.8% ( P = 0.384 and P = 0.216), while the 1-year and 2-year OS rates were 65.2% and 56.5% vs . 52.4% and 47.6% ( P = 0.395 and P = 0.540). Additionally, among CR patients who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) following CAR-T-cell therapy, there were no significant differences in the 1-year or 2-year LFS or OS rates: 57.1% and 50.0% vs . 47.8% and 34.8% ( P = 0.506 and P = 0.356), 64.3% and 57.1% vs . 65.2% and 56.5% ( P = 0.985 and P = 0.883), respectively. The incidence of CRS was greater in the F-CAR-T group (91.3%) than in the C-CAR-T group (66.7%) ( P = 0.044). The incidence of ICANS was also greater in the F-CAR-T group (30.4%) than in the C-CAR-T group (9.5%) ( P = 0.085), but no treatment-related deaths occurred in the two groups.

Conclusion::

Compared with C-CAR-T-cell therapy, F-CAR-T-cell therapy has a superior remission rate but also leads to a tolerably increased incidence of CRS/ICANS. Further research is needed to explore the function of allo-HSCT as an intermediary therapy after CAR-T-cell therapy.

2025-10-01·ZEITSCHRIFT FUR RHEUMATOLOGIE

Article

作者: Wirsching, Andreas ; Grieshaber-Bouyer, Ricardo ; Hagen, Melanie ; Müller, Fabian ; Mackensen, Andreas ; Böltz, Sebastian ; Kharboutli, Soraya ; Schett, Georg ; Taubmann, Jule ; Garantziotis, Panagiotis ; Völkl, Simon ; Aigner, Michael ; Spörl, Silvia

2025-09-23·Blood Advances

Long-term outcomes of genome-edited “universal” CAR19 T cells for relapsed/refractory B-ALL at a single pediatric center

Article

作者: Rashed, Hebatalla ; Rao, Kanchan ; Gilmour, Kimberly ; Vora, Ajay ; Adams, Stuart ; Mishra, Avijeet Kumar ; Pavasovic, Vesna ; Pinner, Danielle ; Qasim, Waseem ; Veys, Paul ; Sauer, Martin ; Guardo, Daniela

157

项与欧贝妥基奥仑赛相关的新闻（医药）

2026-04-16

·瑞孚迪生命科学

精准细胞计数为CGT研发与质控保驾护航

细胞与基因治疗（CGT）作为当下最具潜力的热门赛道，全球研发管线已达数千项，为肿瘤个体化治疗、罕见病、神经退行性疾病等领域带来全新治疗可能。细胞计数是几乎所有细胞相关实验与生产的基础环节，如何实现精准、稳定、可追溯的细胞计数，已成为CGT领域的核心关注点。美国NIST与FDA等监管机构以及国内细胞计数标准已陆续出台相关指导文件，助力企业规范细胞计数操作。其中，细胞数目和细胞活率更是CAR-T等细胞治疗产品生产、放行与质量控制的关键指标。如图1所示，在细胞治疗全流程中，每一步都离不开精准的细胞计数支撑[1]。图1：自体细胞治疗的流程图[1] 随着科学和技术的进步，CGT领域的发展越来越成熟，国内外大大小小的公司埋下的研究种子开始逐渐开花结果。在多家公司的CGT产品不断获得FDA审批后，临床获益病人的数量也在不断增加，给处于绝望期的病人带来新的希望。截至到2026年4月，FDA批准通过的CGT领域的产品总共有48款，具体名单如下表1所示。表1：FDA批准通过的CGT产品列表 Product & Trade Name Manufacturer ABECMA (idecabtagene vicleucel) Celgene Corporation, a Bristol-Myers Squibb Company ADSTILADRIN (nadofaragene firadenovec-vcng) Ferring Pharmaceuticals A/S ALLOCORD (HPC, Cord Blood) SSM Cardinal Glennon Children's Medical Center AMTAGVI (lifileucel) Iovance Biotherapeutics, Inc. AUCATZYL (obecabtagene autoleucel) Autolus Limited BEQVEZ (fidanacogene elaparvovec-dzkt) Pfizer, Inc. BREYANZI (lisocabtagene maraleucel) Juno Therapeutics, Inc., a Bristol-Myers Squibb Company CARVYKTI (ciltacabtagene autoleucel) Janssen Biotech, Inc. CASGEVY (exagamglogene autotemcel [exa-cel]) Vertex Pharmaceuticals Incorporated CLEVECORD (HPC Cord Blood) Cleveland Cord Blood Center Ducord, HPC Cord Blood Duke University School of Medicine ELEVIDYS (delandistrogene moxeparvovec-rokl) Sarapeta Therapeutics, Inc. ENCELTO (revakinagene taroretcel-lwey) Neurotech Pharmaceuticals, Inc. GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen) Organogenesis Incorporated HEMACORD (HPC, cord blood) New York Blood Center HEMGENIX (etranacogene dezaparvovec-drlb) CSL Behring LLC HPC, Cord Blood Clinimmune Labs, University of Colorado Cord Blood Bank HPC, Cord Blood - MD Anderson Cord Blood Bank MD Anderson Cord Blood Bank HPC, Cord Blood - LifeSouth LifeSouth Community Blood Centers, Inc. HPC, Cord Blood - Bloodworks Bloodworks IMLYGIC (talimogene laherparepvec) BioVex, Inc., a subsidiary of Amgen Inc. ITVISMA (onasemnogene abeparvovec-brve) Novartis Gene Therapies, Inc. KEBILIDI (eladocagene exuparvovec-tneq) PTC Therapeutics KYMRIAH (tisagenlecleucel) Novartis Pharmaceuticals Corporation LANTIDRA (donislecel) CellTrans Inc. LAVIV (Azficel-T) Fibrocell Technologies LENMELDY (atidarsagene autotemcel) Orchard Therapeutics (Europe) Limited LUXTURNA (voretigene neparvovec-rzyl) Spark Therapeutics, Inc. LYFGENIA (lovotibeglogene autotemcel [lovo-cel]) bluebird bio, Inc. MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane) Vericel Corp. OMISIRGE (omidubicel-onlv) Gamida Cell Ltd. PAPZIMEOS (zopapogene imadenovec-drba) Precigen, Inc. PROVENGE (sipuleucel-T) Dendreon Corp. REGENECYTE (HPC, Cord Blood) StemCyte, Inc. RETHYMIC (allogeneic processed thymus tissue – agdc) Enzyvant Therapeutics GmbH ROCTAVIAN (valoctocogene roxaparvovec-rvox) BioMarin Pharmaceutical Inc RYONCIL (remestemcel-L-rknd) Mesoblast, Inc. SYMVESS (acellular tissue engineered vessel-tyod) Humacyte Global, Inc. SKYSONA (elivaldogene autotemcel) bluebird bio, Inc. STRATAGRAFT (allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsat) Stratatech Corporation TECARTUS (brexucabtagene autoleucel) Kite Pharma, Inc. TECELRA (afamitresgene autoleucel) Adaptimmune LLC VYJUVEK (beremagene geperpavec-svdt) Krystal Biotech, Inc. WASKYRA (etuvetidigene autotemcel) Fondazione Telethon ETS YESCARTA (axicabtagene ciloleucel) Kite Pharma, Incorporated ZEVASKYN (prademagene zamikeracel) Abeona Therapeutics, Inc. ZYNTEGLO (betibeglogene autotemcel) bluebird bio, Inc. 上下滑动查看更多详情见网址：https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products 除了国外CGT市场的繁华，国内CGT市场也处于非常兴盛的时期，数千家公司布局CGT赛道，我们从国家药品监督管理局药品审评中心（Center For Drug Evaluation，NMPA）统计发现，截至到2026年4月，通过搜索细胞注射液的关键词就已经查到有320款受理品种，其中干细胞注射液为203款，T细胞注射液为168款由CDE批准获得临床试验许可。图2：CDE官网受理品种的部分截图（左右滑动查看更多）详情见网址：https://www.cde.org.cn 随着818&828号令推出，国内大力推广CGT的发展。目前，国内多款CAR-T疗法以及干细胞疗法获批上市。上市国家药物名称生产厂商靶点上市时间中国奕凯达复星凯特 CD19 2021 中国倍诺达药明巨诺 CD19 2021 中国福可苏驯鹿/信达 BCMA 2023 中国源瑞达合源生物 CD19 2023 中国赛恺泽科济药业 BCMA 2024 中国卡卫狄传奇生物 BCMA 2024 中国恒凯莱恒润达生 CD19 2025 中国普利得凯重庆精准生物 CD19 2025 中国睿铂生铂生卓越干细胞 2025 图3：国内CDE获批上市的CAR-T疗法&干细胞疗法 CGT领域离不开细胞精准计数，瑞孚迪（Revvity）公司旗下的Cellometer系列的细胞计数仪为多家公司的细胞治疗产品提供助力。Cellometer系列细胞计数仪不仅支持台盼蓝染色方法，同时支持AO/PI双荧光染色方法，通过AO/PI染色对原代或者复杂样本（如全血、PBMCs、肝细胞和小鼠脾细胞等）中的有核细胞进行精准的识别计数，其中无核样本（如红细胞、血小板或碎片等）被排除在外。MD Anderson在2019年由FDA批准的首个针对实体瘤的非病毒TCR-T疗法获批临床，以及国内工业巨头药明巨诺等在文献中推荐Cellometer系列的计数产品[2,3]。图4：Cellometer细胞计数仪助力国内外细胞治疗公司细胞计数仪是CGT领域核心质控设备。瑞孚迪作为行业开拓者，深度参与国标GB/T 46490-2025制定，其细胞计数仪系列已应用于国内首款干细胞及多款CAR-T产品质控。作为受邀参与国际标准研讨的设备厂商，瑞孚迪精准把握行业挑战与市场需求。将推出专题，持续分享细胞计数标准发展及质控关键指标。品牌将持续以技术创新为核心，深耕用户需求，提供更适配、更可靠的产品，助力细胞治疗产业高质量发展。点击了解相关产品 Cellometer Ascend 细胞计数仪 Cellometer K2 双荧光细胞计数仪相关阅读央视点赞！首张“干细胞药物”开出，点亮生命之光！ 818/828号令指明行业新方向，瑞孚迪细胞计数系列助力标准再升级！迈向“细胞溯源”：国际标准引领精准医疗新时代参考文献 [1] Pierce L T , Sarkar S , Chan L ,et al.Outcomes from a cell viability workshop: fit-for-purpose considerations for cell viability measurements for cellular therapeutic products[J]. 2021.DOI:10.18609/CGTI.2021.076. [2] Singh H , Moyes JSE, and Cooper LIN ,et al.Manufacture of T cells using the Sleeping Beauty system to enforce expression of a CD19-specific chimeric antigen receptor[J]. Cancer Gene Therapy,2015 , 22:95–100. [3] Wang W , Hao M , Cheng Y ,et al.JWCAR029 Is a CD19-Targeted CAR T Cell Product with Process and Quality Controls Delivered As a Flat Dose of CAR T Cell to Patients with NHL[J].Blood, 2018, 132(Suppl_1):5387-5387.DOI:10.1182/blood-2018-99-114434. 本期精彩内容从农业到宠物：类器官与器官芯片技术如何重塑动物健康研究精准细胞计数为CGT研发与质控保驾护航会议邀请丨瑞孚迪与您相约4月22日-23日第七届BIONNOVA生物医药创新者论坛 .

2026-04-14

·BioSpace

Asgard Therapeutics appoints Professor Dr Wolfram Brugger as Chief Medical Officer as it advances transformative ‘personalized off-the-shelf’ immunotherapy toward clinical development

Medical oncologist (ex AstraZeneca, MorphoSys, Autolus Therapeutics) joins to advance Asgard’s lead asset AT-108 into Phase 1 clinical development AT-108 is a transformative immunotherapy that triggers a personalized immune response against a patient’s tumors, despite being an off-the-shelf drug Asgard is currently progressing IND-enabling studies of this first-in-class therapy, which reprograms cancer cells in vivo, forcing them to present their antigens to the immune system LUND, Sweden, April 14, 2026 (GLOBE NEWSWIRE) -- Asgard Therapeutics, a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy, today announces the appointment of Prof Dr Wolfram Brugger as Chief Medical Officer. Prof Dr Brugger, who has been involved in more than 130 Phase I-III clinical oncology trials with numerous modalities across a wide variety of cancer types, including solid tumors, will strengthen Asgard’s leadership as it prepares to transition to a clinical-stage biotech. Cristiana Pires, Co-founder and Chief Executive Officer of Asgard Therapeutics, said : “Strengthening our leadership team with Wolfram, who is such a highly experienced clinical trials expert and practicing medical oncologist, is a privilege and we are excited to welcome him on board. Wolfram’s proven capabilities and track record will ensure that Asgard is perfectly positioned as we advance AT-108 towards the clinic." Prof Dr Wolfram Brugger, incoming Chief Medical Officer of Asgard Therapeutics, said: “I’ve always been driven by bringing new modalities to patients, and I am excited to be developing a ‘personalized off-the-shelf’ immunotherapy which has great potential to help patients with a wide variety of cancers. Asgard’s in vivo cell reprogramming technology has transformative potential, and the pre-clinical proof-of-concept data, both published and upcoming data to be presented at key scientific conferences, is highly convincing. Having treated hundreds of cancer patients in clinical trials throughout my career, I look forward to utilizing my experience to shape Asgard’s clinical strategy, driving AT-108 through clinical development.” Wolfram joins Asgard from NASDAQ-listed Autolus Therapeutics where he led the global clinical development program of AUTO1 (AUCATZYL® / obe-cel), an autologous CD19 CAR T therapy, in hematological and autoimmune indications. AUTO1 received FDA approval in 2024 for relapsed / refractory B-cell acute lymphoblastic leukemia (B-ALL) and conditional approval was granted by the EMA and MHRA in 2025. Prior to this he was at MorphoSys where he headed the global clinical development program for the monoclonal antibody tafasitamab across various blood cancer indications. Tafasitamab (in combination with lenalidomide) was granted accelerated approval by the FDA in relapsed/refractory DLBCL in 2020 and conditional approval by the EMA in the same indication in 2021. At AstraZeneca, he was global medical lead for Phase I / early Phase II trials for several oncology assets in numerous cancer types, including testing them in combination with checkpoint inhibitors. Prior to moving into drug development, he was Chief & Medical Director in the Department of Hematology, Oncology, Immunology & Infectious Diseases at a Teaching Hospital of Freiburg University in Germany. He holds an MD and PhD from Albert-Ludwigs University Freiburg and Eberhard-Karls University Tübingen, Germany, with a specialist registration in Internal Medicine, Medical Oncology and Hematology. Asgard is currently focused on advancing IND-enabling studies and CMC activities to support clinical development of AT-108. Key proof-of-concept data on AT-108 were published in 2024 in the high-impact, peer-reviewed journal Science 1 . For further information, please contact: Asgard Therapeutics Cristiana Pires, Co-founder and CEO    Email: cristiana.pires@asgardthx.com Optimum Strategic Communications Mary Clark, Stephen Adams, Elena Bates Tel: +44 (0) 20 8142 3740 Email: asgard@optimumcomms.com Notes to Editors About Asgard Therapeutics Asgard Therapeutics is a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy. The company builds on ground-breaking and proprietary reprogramming technologies to develop gene therapy products designed to set in motion efficient and personalized immune responses. Asgard Therapeutics aims to establish a pipeline of off-the shelf cancer immunotherapies that trigger personalized anti-cancer immune responses for the benefit of cancer patients in need. Asgard is backed by Novo Holdings, Boehringer Ingelheim Venture Fund, Industrifonden, RV Invest and Johnson & Johnson Innovation – JJDC, Inc. For more information, please visit: About AT-108 AT-108 is a first-in-class, off-the-shelf gene therapy that directly reprograms tumor cells into a rare subset of dendritic cells critical for mounting efficient cytotoxic T cell responses, cDC1 cells (conventional dendritic cells type 1). Reprogramming forces the tumor cells to present their tumor antigens, ultimately leading to a personalized anti-tumor immune response. It is based on a replication-deficient adenoviral vector that delivers three proprietary reprogramming factors into tumor cells, rewiring their gene expression signatures and thus “programming” them to become antigen-presenting cDC1-like cells. _______________________ 1

2026-04-09

·百度百家

120万天价救命药有救了？外资入局搅动中国细胞治疗，能打破“天价药”困局吗

看着病床上日渐消瘦的父亲，张伟的眉头紧锁了整整三天。弥漫性大B细胞淋巴瘤，这个拗口的医学名词如今成了全家人每天必须面对的梦魇。六轮化疗下来，父亲体内的癌细胞依然顽固地生长着，主治医生在病历上写下“复发难治”四个字时，张伟的手心全是汗。“还有一个选择，”医生顿了顿，“CAR-T细胞疗法，但费用要120万。”120万——这个数字在安静的诊室里像一枚炸弹，炸碎了张伟最后一线希望。就在无数个像张伟这样的家庭陷入困境时，一扇政策的大门悄然开启。国务院于2026年4月9日发布《中国（内蒙古）自由贸易试验区总体方案》，试点允许外资企业从事人体干细胞、基因诊断与治疗技术开发和技术应用，用于产品注册上市和生产。这已是继北京、上海、广东自贸试验区及海南自由贸易港之后，中国在细胞与基因治疗（CGT）领域对外资开放的又一关键扩围。政策破冰，外资涌入，对中国CGT领域而言，究竟是激活一池春水的“鲶鱼”，还是可能引发洗牌的“鲨鱼”？这扇打开的大门，最终通向的是希望还是更沉重的负担？政策破冰——从试点到扩围的战略棋局此次内蒙古自贸区政策并非孤立事件。追溯这条开放之路，脉络清晰可见：2024年3月，国务院办公厅发布《扎实推进高水平对外开放更大力度吸引和利用外资行动方案》，首次提出在北京、上海、广东等自贸试验区开展放宽科技创新领域外商投资准入试点。同年9月，商务部、国家卫生健康委、国家药监局三部门共同发布《关于在医疗领域开展扩大开放试点工作的通知》，正式在四个自贸区允许外商投资企业从事人体干细胞、基因诊断与治疗技术开发和技术应用。此次内蒙古的加入，标志着中国CGT市场对外开放进入新阶段——从“谨慎尝试”转向“体系化引入”。政策背后的战略意图至少有三重考量：第一重是引进“活水”。CGT作为生物医药领域最具突破性的前沿技术，全球竞争日趋白热化。2024年CAR-T细胞治疗产品已创造59亿美元的价值，预计到2030年将达到278亿美元。中国市场增速更为凌厉：2023年免疫治疗产品市场规模已达102.0亿元，预计2030年达到270亿元，年复合增长率高达65.5%。在这样的市场格局下，吸引国际顶尖技术、人才与管理经验，成为加速国内CGT产业成熟度的关键路径。第二重是区域布局。内蒙古“内联八省近京畿、外接俄蒙通欧亚”的区位优势，或许能够探索出与北上广深不同的差异化发展路径。这可能包括结合当地资源特色的特定疾病研究，或者在成本控制、供应链布局方面的创新尝试。第三重是制度接轨。通过外资的深度参与，有望倒逼监管科学、临床试验标准与国际进一步接轨。这种制度层面的“鲶鱼效应”，对于提升中国在全球CGT研发版图中的参与度与话语权具有深远意义。产业冲击波——本土药企的机遇与挑战 “鲶鱼效应”的积极面已经开始显现。外资带来的不仅是资本，更是一整套先进的生产工艺、质量控制体系和研发经验。技术溢出效应最为直观。以阿斯利康为例，这家英国制药企业近期宣布将在上海建立细胞疗法商业化生产供应基地与创新中心，并成为首家在中国拥有端到端细胞治疗能力的跨国药企。这种“端到端”的完整体系构建——从源头创新到全球转化，从病毒载体与质粒开发到分析检测、临床生产及注册支持——将为本土企业提供难得的学习样板。人才培育机制同样值得期待。随着国际研发中心设立，高端人才的培养与流动将加速。阿斯利康的投资脉络清晰可见：2007年在上海建立中国创新中心，2013年成立中国新药研发部，2019年升级为全球研发中国中心，2024年进一步升级为阿斯利康全球第五大战略研发中心。这种持续提升本地研发能级的模式，可能被更多外资企业复制。供应链升级或许是最大的受益环节。细胞治疗作为生物医药前沿领域，其产业链长、技术集成度高，涵盖细胞存储、试剂耗材、设备研发、药物生产、临床服务等多个环节。2025年修订的《鼓励外商投资产业目录》将细胞治疗药物及其关键原材料、制备技术纳入鼓励范围，覆盖从细胞提取、培养、检测到成品制备的完整产业链环节。这将联动上下游配套产业共同发展，推动本土CDMO（合同研发生产组织）产业快速成长。然而，“鲨鱼威胁”的阴影同样存在。市场竞争将不可避免地走向白热化。在资本、品牌、国际临床数据方面具有优势的外资企业，可能对部分本土初创企业形成挤压。目前国内已有八款国产CAR-T上市，其中已公布定价的五款产品价格介于99.9万至129万元之间。合源生物的纳基奥仑赛注射液定价99.9万元/针，复星凯瑞的阿基仑赛注射液120万元/针，药明巨诺的瑞基奥仑赛注射液129万元/针——这些本土企业如何在产品定位、定价策略、市场推广方面应对外资竞争，将成为关键考验。竞合关系或许更为现实。未来可能出现外资与本土企业既竞争又合作的复杂格局。本土药企如何利用自身对国内市场的理解、临床资源网络、以及灵活的研发策略，与外资企业形成差异化互补，将成为生存发展的关键。例如，外资企业可能更专注于国际同步的高端技术，而本土企业在某些细分领域、特定适应症、或成本控制方面可能具有优势。短期阵痛或许难以避免，但长期来看，这场竞争将促使本土企业更加注重差异化创新与核心能力构建。中国创新药产业从萌芽到爆发，不过十几年时间。随着监管体系全面向国际标准对齐，突破性疗法、附条件批准等政策相继落地，创新药研发和上市周期显著缩短。这场外资进入的浪潮，可能加速整个产业从“跟跑”向“并跑”甚至“领跑”的转变。患者可及性提速——打破“天价药”困局的曙光？对于千千万万个像张伟这样的家庭来说，最核心的问题是：这场政策开放，能否让“救命药”不再遥不可及？可及性提速的关键在于生产成本与价格博弈。以已在国内上市的CAR-T疗法为例，其“天价”现状背后有复杂的原因。单次制备成本可达10万元至30万元，这源于细胞治疗技术的高复杂度——需要从患者血液中提取T细胞，通过体外扩增和基因编辑技术使其表达靶向肿瘤的嵌合抗原受体，再回输至患者体内。整个过程需要使用专业实验室设备、高纯度试剂及严格的质量控制体系。外资本土化能否带来实质性变化？理论上，外资在华建立研发生产基地后，可能通过规模化生产、供应链本地化、工艺优化等手段降低成本。规模化效应尤为关键：目前CAR-T治疗仍是个性化制备，每位患者都需要单独的生产批次。如果能够实现标准化、规模化生产，单位成本有望显著下降。下一代“体内CAR-T”技术正在快速崛起，有专家推测其有望从根本上解决“用不起、等不及”的难题，甚至可能将成本降至现有技术的1/10。然而理性展望，降价幅度与时间表存在现实约束。本土化是降低成本的必要非充分条件。即便技术成熟、工艺优化，高昂的研发投入、严格的质量控制、复杂的监管要求都意味着CGT产品很难像传统药品那样快速降价。国际经验也显示，即便在美国市场，最新获批用于成人急淋的CAR-T产品Aucatzyl单价高达52.5万美元。临床研究加速是另一重希望。外资带来的国际多中心临床试验（MRCT）资源，对中国患者意义重大。这将帮助中国患者更早接触到全球前沿疗法，获得免费治疗机会，并加速该疗法在中国的注册上市进程。目前已有案例显示这种机制的潜力：关键III期VIITAL研究显示，81%接受基因治疗的RDEB患者伤口实现超过50%的愈合，而对照组仅为16%。这种全球同步的临床试验，不仅让中国患者有机会获得最新治疗，更能提升中国在全球CGT研发版图中的参与度与话语权。当中国成为国际多中心临床试验的重要站点，数据质量获得国际认可，未来药品在中国申报上市时也将更加顺畅。无法回避的挑战——支付体系的终极考验即便技术到来、成本部分下降，动辄百万级的治疗费用，如何支付？这是横亘在创新与患者之间的最后一道屏障。现状令人忧虑。2024年全球基因治疗市场规模约为110亿美元，预计将以20%的年复合增长率在2034年突破588亿美元。在这个高速增长的市场中，价格不断刷新纪录：最贵的药物，即CSL Behring和uniQure的血友病药物Hemgenix，每款单价高达350万美元。国内CAR-T产品99.9万至129万元的定价，相比美国市场37万~50万美元的定价虽然较低，但对于普通家庭仍是难以承受之重。当前基本医保基金面临巨大压力，难以单独承担此类高价创新疗法。国家医疗保障局在2024年12月明确提出“探索创新药的多元支付机制，支持引导普惠性商业健康保险及时将创新药品纳入报销范围，研究探索形成丙类药品目录”。多元支付体系的构建正在破冰。2025年8月，国家医疗保障局公布首版《商业健康保险创新药品目录》，121款药品通过商保创新药目录的形式审查，其中包括复星凯瑞的CAR-T产品奕凯达®等一批高价值创新药。首版商保创新药目录共纳入18家创新药企的19种新药，5款国产CAR-T均成功入选。具体到商业健康保险领域，开发定制化、覆盖特定CGT产品的商业保险产品成为可能。一位使用过阿基仑赛的患者家属表示，如果商保创新药目录能落地，对医生和患者来说都意味着更多的选择。实际上已有成功案例：徐阿姨在确诊为弥漫大B细胞淋巴瘤后选择CAR-T治疗，120万元的治疗费用通过医保及商保报销后，个人仅承担不到7万元。专项救助基金是另一条路径。这种由政府、企业、社会共同筹资设立专项基金的模式，在国际上已有实践。美国医疗保险和医疗补助创新中心发布的《细胞和基因治疗可及性模式》探索基于疗效结果的商业协议，尝试在减少Medicaid支出的同时改善可及性和公平性。创新支付模式同样值得关注。按疗效付费、分期付款、风险分担等国际探索，可能为破解支付难题提供新思路。这些模式将药物支付与实际疗效挂钩，激励制造商不仅关注药物研发和销售，更要重视药物在实际应用中的效果。然而，建立患者、医保、商保、企业、社会慈善等多方共付机制面临诸多挑战。如何确保疗效评价的客观性和公正性、如何平衡制造商和医疗机构的利益、如何确保返利机制的透明度和可持续性，都需要在实践中不断探索和完善。支付体系的创新与完善，是决定这场技术开放红利能否真正抵达患者的“最后一公里”。开放之路，惠及之路外资进入既是“鲶鱼”，也是“鲨鱼”，其本质是推动中国CGT产业在竞争与合作中走向更高水平。从北京、上海、广东、海南的试点到内蒙古的扩围，从《鼓励外商投资产业目录》的修订到商保创新药目录的建立，政策体系正在逐步完善。但最终衡量成败的核心标准，始终是患者获益。技术再先进，如果只能成为少数人的特权，那么这场开放的意义将大打折扣。政策开放只是起点，后续需要产业、监管、支付三方协同发力，才能将技术突破转化为实实在在的民生福祉。产业层面需要构建更加健康、有序的竞争生态，既要鼓励创新，也要防止恶性竞争导致资源浪费。监管层面需要加快与国际接轨的步伐，建立科学、透明、高效的审评审批体系。支付层面则需要大胆创新，探索适合中国国情的多元支付模式。细胞与基因治疗正在从针对末线血液肿瘤的市场定位，逐步扩展至针对血液肿瘤前线治疗、实体瘤、自身免疫性疾病乃至慢性退行性疾病的通用型治疗手段。这场技术革命的前景令人振奋，但道路依然漫长。对于动辄百万的细胞基因疗法，怎样的支付模式才能让创新真正惠及普通百姓？当“能救”遇上“等不起”，当技术突破遭遇支付瓶颈，我们需要思考的不仅是技术路径，更是社会公平与医疗伦理的深层命题。

细胞疗法免疫疗法核酸药物

100 项与欧贝妥基奥仑赛相关的药物交易

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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
前体B细胞成淋巴细胞白血病淋巴瘤	美国	Autolus Ltd.	2024-11-08

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
复发性 B 细胞急性淋巴细胞白血病	申请上市	美国	Autolus Therapeutics Plc	2023-11-29
难治性 B 细胞急性淋巴细胞白血病	申请上市	美国	Autolus Therapeutics Plc	2023-11-29
CD19阳性B细胞急性淋巴细胞白血病	临床2期	美国	Autolus Ltd.	2026-02-03
狼疮性肾炎	临床2期	-	Autolus Ltd.	2025-09-01
急性淋巴细胞白血病	临床2期	美国	Autolus Ltd.	2020-06-03
急性淋巴细胞白血病	临床2期	西班牙	Autolus Ltd.	2020-06-03
急性淋巴细胞白血病	临床2期	英国	Autolus Ltd.	2020-06-03
费城染色体阳性急性淋巴细胞白血病	临床2期	美国	Autolus Ltd.	2020-06-03
费城染色体阳性急性淋巴细胞白血病	临床2期	西班牙	Autolus Ltd.	2020-06-03
费城染色体阳性急性淋巴细胞白血病	临床2期	英国	Autolus Ltd.	2020-06-03

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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


NCT06173518 (CATULUS, Tandem Meetings ASTCT and CIBMTR2026)	临床1/2期	复发性儿童急性淋巴细胞白血病 CD19	20	Obecabtagene autoleucel	鬱衊齋觸憲艱蓋餘鬱糧(繭顧淵艱憲網簾簾襯積) = 醖憲願壓蓋醖鏇憲夢餘憲鏇餘廠選鏇鹹壓繭糧 (衊淵願鑰獵顧築艱構餘, 75.1 ~ 99.9) 更多	积极	2026-02-04
NCT06173518 (CATULUS, ASH2025)	临床1/2期	难治性 B 细胞急性淋巴细胞白血病	20	obecabtagene autoleucel (obe-cel)	餘鑰鑰顧網觸憲蓋憲窪(醖憲膚蓋網簾鏇夢淵願) = 鏇積網窪鏇網夢製簾醖膚淵願選鏇壓窪選壓顧 (窪糧餘壓獵鑰鹹選簾糧, 75.1 ~ 99.9) 更多	积极	2025-12-06
NCT06333483 (CARLYSLE, ASH2025)	临床1期	系统性红斑狼疮	10	Obecabtagene autoleucel (obe-cel)	鏇艱遞壓鏇糧範膚鑰繭(齋夢鏇憲壓廠壓醖膚觸) = a clinically meaningful reduction (to ≤1) 鹽願鹽襯製廠構廠齋鑰 (觸壓構蓋憲膚鬱夢鹹襯 ) 更多	积极	2025-12-06
NCT06333483 (CARLYSLE, ACR2025)	临床1期	系统性红斑狼疮	7	Obecabtagene autoleucel (obe-cel)	顧獵觸鑰構簾衊蓋觸糧(鏇製觸憲齋觸構憲製鏇) = No Gr ≥2 CRS; Transient hypertension was observed in five pts; All pts had Gr ≥3 neutropenia; Infections were transient and manageable; no life-threatening infections were observed. 艱遞繭製觸窪繭蓋觸鬱 (糧鬱襯蓋選糧獵淵蓋範 ) 更多	积极	2025-10-24
NCT04404660 (FELIX, ASCO2025)	临床1/2期	复发性 B 细胞急性淋巴细胞白血病 CD19	127	Obe-cel	淵艱蓋網壓觸遞醖鏇艱(衊鑰餘願鹹廠觸餘餘襯) = 構鏇構鑰構膚範築鑰網衊積願夢獵齋觸觸壓積 (繭憲膚衊製淵鏇遞鏇遞, 60.9 ~ 81.7)	积极	2025-05-30
NCT04404660 (FELIX, ASCO2025)	临床1/2期	复发性 B 细胞急性淋巴细胞白血病 CD19	127	obecabtagene autoleucel (obe-cel) (Age <55 years)	獵鏇壓繭鏇鏇蓋簾觸餘(窪壓餘網窪觸鹽衊構築) = 鹹願顧鬱齋鬱壓製膚觸醖壓積鹹壓繭繭願憲餘 (製簾廠製簾獵構顧艱鏇 ) 更多	积极	2025-05-30
NCT04404660 (FELIX, EHA2025)	临床1/2期	难治性 B 细胞急性淋巴细胞白血病 CD19	127	Obe-cel <55 years	餘積醖顧鬱窪觸網淵衊(願鑰壓夢衊蓋鹹選蓋齋) = 壓廠齋簾網製淵鹽夢鬱觸簾鏇選鏇憲構膚憲膚 (觸艱鏇網襯獵顧築獵醖 ) 更多	积极	2025-05-14
NCT04404660 (FELIX, EHA2025)	临床1/2期	难治性 B 细胞急性淋巴细胞白血病 CD19	127	Obe-cel ≥55 years	餘積醖顧鬱窪觸網淵衊(願鑰壓夢衊蓋鹹選蓋齋) = 鹹蓋製窪衊網膚範淵糧觸簾鏇選鏇憲構膚憲膚 (觸艱鏇網襯獵顧築獵醖 ) 更多	积极	2025-05-14
NCT04404660 (FELIX, Pubmed \| N Engl J Med) 人工标引	临床1/2期	复发性 B 细胞急性淋巴细胞白血病 \| 难治性 B 细胞急性淋巴细胞白血病	127	Obecabtagene autoleucel	繭襯選鏇膚膚選蓋築繭(鬱糧構簾鹽夢獵簾繭觸) = 蓋壓鬱繭鬱醖鬱齋選淵製構襯鏇壓蓋鑰廠鹽壓 (繭廠鹹鏇顧鏇遞襯蓋積, 67 ~ 85) 更多	积极	2024-12-12
ASH2024	N/A	复发性 B 细胞急性淋巴细胞白血病	-	Obecabtagene autoleucel (obe-cel) (High-risk HT score)	夢鏇夢艱鬱鏇鬱鹹鹹憲(餘淵遞壓齋選衊醖製繭) = 糧範願鹹襯願窪鏇憲顧願夢範襯鏇鬱醖網簾顧 (顧獵淵淵糧獵獵鏇築鹽, 34.6 ~ 55.6) 更多	-	2024-12-09
ASH2024	N/A	复发性 B 细胞急性淋巴细胞白血病	-	Obecabtagene autoleucel (obe-cel) (Low-risk HT score)	夢鏇夢艱鬱鏇鬱鹹鹹憲(餘淵遞壓齋選衊醖製繭) = 餘簾鑰願獵襯憲鏇鹽襯願夢範襯鏇鬱醖網簾顧 (顧獵淵淵糧獵獵鏇築鹽, 40.2 ~ 82.1) 更多	-	2024-12-09
NCT04404660 (FELIX, ASH2024)	临床1/2期	复发性成人急性淋巴细胞白血病 ClonoSEQ®next-generation sequencing (NGS) assay	127	Obe-cel	製齋簾襯網窪鑰選積衊(獵膚夢鹹顧糧製憲製糧) = 範願遞齋壓廠構襯簾觸夢鏇鏇衊網鑰廠築淵餘 (鏇襯鏇鹹餘壓構衊選餘 ) 更多	积极	2024-12-09
NCT04404660 (FELIX, ASH2024)	临床1/2期	复发性成人急性淋巴细胞白血病 CD19	127	Obecabtagene autoleucel (obe-cel)	窪築鹹醖簾鏇築衊醖糧(繭範衊齋製願構鑰製窪) = 72 (70.6%) were CRS only 糧選鏇窪範醖顧衊鹽衊 (蓋築鬱鏇積構獵築蓋觸 ) 更多	积极	2024-12-09