This phase II trial tests the effect of nemtabrutinib in combination with brexucabtagene autoleucel (brexu-cel) in treating patients with mantle cell lymphoma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Nemtabrutinib, a BTK inhibitor, may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chimeric antigen receptor (CAR) T-cell therapy, such as brexu-cel, is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's cancer cells is added to the T cells in the laboratory. The special receptor is called a CAR. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. Chemotherapy, such as fludarabine and cyclophosphamide, are given before CAR T cell therapy to help kill cancer cells in the body and help make room for the CAR T cells. Giving nemtabrutinib in combination with brexu-cel may be safe, tolerable, and/or effective in treating patients with relapsed or refractory mantle cell lymphoma.

开始日期2026-07-31

申办/合作机构

The Vanderbilt-Ingram Cancer Center

[+1]

NCT06553872

/ Recruiting临床2期IIT

A Phase 2, Open-Label, Randomized Study of Pirtobrutinib and Brexucabtagene Autoleucel in Patients With Relapsed or Refractory Mantle Cell Lymphoma

This is a phase 2, open-label, randomized, multicenter clinical trial in patients with relapsed/refractory mantle cell lymphoma (R/R MCL) who meet the criteria for standard-of-care FDA label for CD19 CAR T-cell therapy with brexucabtagene autoleucel (brexu-cel).

开始日期2024-08-23

申办/合作机构

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

NCT06253663

/ Active, not recruiting临床2期

A Phase 2 Multicenter Study Evaluating the Safety and the Efficacy of KTE-X19 in Adult Japanese Subjects With Relapsed/Refractory Mantle Cell Lymphoma or Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia

The goal of this clinical study is to learn more about KTE-X19, and how safe and effective it is in adult Japanese participants with relapsed/refractory (r/r) Mantle Cell Lymphoma (MCL) or r/r B-precursor Acute Lymphoblastic Leukemia (B-ALL).
The primary objectives of this study are to evaluate the efficacy of KTE-X19, as measured by:
* Objective response rate (ORR) per investigator assessment, in adult Japanese participants with r/r MCL
* Overall complete remission (OCR) defined as complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) per investigator assessment, in adult Japanese participants with r/r ALL

开始日期2024-03-18

申办/合作机构

Kite Pharma, Inc.

100 项与布瑞基奥仑赛相关的临床结果

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100 项与布瑞基奥仑赛相关的转化医学

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100 项与布瑞基奥仑赛相关的专利（医药）

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164

项与布瑞基奥仑赛相关的文献（医药）

2026-08-01·AMERICAN JOURNAL OF HEMATOLOGY

Post CAR ‐T Measurable Residual Disease Monitoring in Mantle Cell Lymphoma Enables Early Detection of Disease Relapse

Article

作者: Dahiya, Saurabh ; Simmons, Heidi ; Agarwal, Neha ; Jacob, Allison ; Kirsch, Ilan ; Smith, Melody ; Ananth, Snegha ; Sahaf, Bita ; Frank, Matthew J. ; Miklos, David ; Weng, Wen‐kai ; Bharadwaj, Sushma ; Lee, Lik Wee

ABSTRACT:

CD19‐directed chimeric antigen receptor (CAR) T‐cell therapy has transformed outcomes for patients with relapsed or refractory (r/r) mantle cell lymphoma (MCL), yet more than 40% relapse within one year. Early identification of patients at risk for progression could inform post CAR‐T surveillance and consolidation strategies. Measurable residual disease (MRD) has emerged as a powerful prognostic biomarker in frontline MCL, but its role after CAR T‐cell therapy remains incompletely defined. We retrospectively analyzed 37 patients with r/r MCL treated with brexucabtagene autoleucel (brexu‐cel). MRD was assessed using next‐generation immunoglobulin high‐throughput sequencing (Ig‐HTS) of peripheral blood mononuclear cells obtained before lymphodepletion, at 1‐ and 3‐months post‐infusion, and every 3 months thereafter. Clonotype identification was successful in 36 of 37 patients. Pre‐lymphodepletion MRD levels were lower in patients receiving bridging therapy (337 [0–198 449] vs. 21 213 [1–788 251]; p = 0.04), and MRD undetectability trended toward improved progression‐free survival (PFS; unreached vs. 28.5 months; HR 5.2; p = 0.07). Post‐infusion, patients with detectable Day 28 MRD had inferior PFS compared with those with undetectable MRD (10.9 vs. 51.5 months; HR 3.99; p = 0.002), whereas Day 28 PET‐CT response did not correlate with PFS ( p = 0.35; HR 1.8). Longitudinal MRD monitoring identified relapse a median of 6.5 months before PET/CT in most relapsing patients (15 out of 18). Early and serial MRD monitoring is thus a sensitive prognostic and surveillance tool in brexu‐cel treated MCL, with Day 28 MRD serving as an early predictor of long‐term outcomes.

2026-07-01·Lancet Haematology

Real-world patient-reported symptomatic adverse events and concordance with physician assessments after CAR T-cell therapy in patients with aggressive B-cell lymphomas: a prospective study

Article

作者: Gambella, Massimiliano ; Tisi, Maria Chiara ; Chiappella, Annalisa ; Di Rocco, Alice ; Martino, Massimo ; Cutini, Ilaria ; Bonifazi, Francesca ; Farina, Mirko ; Orciuolo, Enrico ; Zinzani, Pier Luigi ; Tartaglia, Francesca ; Fazi, Paola ; Ragaini, Simone ; Efficace, Fabio ; Bramanti, Stefania ; Corradini, Paolo ; Donzelli, Livia ; Vignetti, Marco ; Puglia, Serena ; Martelli, Maurizio ; Botto, Barbara

BACKGROUND:

Chimeric antigen receptor (CAR) T cells have shown considerable promise in treating patients with haematological malignancies. We aimed to investigate short-term patient-reported symptomatic adverse events in patients with aggressive B-cell lymphomas treated with CAR T-cell therapy and compare them with those reported by their treating physicians. We also examined factors predicting overall short-term burden of therapy.

METHODS:

This was a prospective, observational, multicentre study enrolling patients across 13 centres in Italy. Eligible patients were aged 18 years and older with a confirmed diagnosis of aggressive B-cell lymphoma. All patients were scheduled to undergo CAR T-cell therapy with one of the following products: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), or brexucabtagene autoleucel (brexu-cel). The primary objective of this analysis was to assess the prevalence of short-term patient-reported symptomatic adverse events overall and by sex and age categories. The primary objective was evaluated on day 10 after infusion via the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), which includes 19 items selected based on their clinical relevance, including: difficulty swallowing, decreased appetite, nausea, diarrhoea, swelling, hair loss, dizziness, concentration difficulty, memory difficulty, general pain, headache, muscle pain, joint pain, insomnia, fatigue, anxiety, discouragement, sadness, and chills. Prevalence of symptomatic adverse events was also assessed by type of CAR T-cell product. Additionally, the study was designed to allow a corresponding adverse event assessment via the CTCAE by treating haematologists for comparative analysis. Adverse events reported by physicians were matched with corresponding adverse events reported by patients. Furthermore, an overall symptom burden score was computed and used as outcome variable in multivariable analysis to examine factors predicting short-term burden of therapy, including sociodemographic and clinical data and pre-infusion patient-reported physical functioning by the EORTC QLQ-C30. The study is registered with ClinicalTrials.gov, NCT06026644, and is closed to accrual and follow-up is complete.

FINDINGS:

Between June 29, 2022, and Feb 26, 2024, 170 patients were included in the study. The median age of patients was 61·1 years (IQR 51·1-68·5) and the median follow-up was 23·7 months (17·7-24·6). 53 (31%) of 170 enrolled patients were female and 117 (69%) were male. Most patients were diagnosed with diffuse large B-cell lymphoma (118 [69%] patients), followed by mantle cell lymphoma (32 [19%] patients) and primary mediastinal large B-cell lymphoma (20 [12%] patients). 98 (58%) patients were infused with axi-cel, 39 (23%) with tisa-cel, and 32 (19%) with brexu-cel. 165 patients reached the day 10 timepoint, of whom 143 (87%) completed the corresponding PRO-CTCAE item list. We observed a prevalence (any grade) of more than 50% across 12 of the 19 symptomatic adverse events examined: fatigue (123 [87%] of 141 patients), decreased appetite (116 [83%] of 139 patients), insomnia (112 [79%] of 142 patients), chills (103 [73%] of 142 patients), diarrhoea (99 [70%] of 141 patients), sadness (81 [57%] of 141 patients), general pain (78 [55%] of 143 patients), dizziness (78 [55%] of 143 patients), joint pain (74 [52%] of 142 patients), muscle pain (73 [51%] of 143 patients), headache (73 [51%] of 143 patients), and impaired concentration (72 [51%] of 142 patients). Inspection of moderate to severe grades revealed that more than 30% of patients reported fatigue (78 [55%] of 141 patients), decreased appetite (74 [53%] of 139 patients), diarrhoea (63 [45%] of 141 patients), chills (49 [35%] of 142 patients), and insomnia (44 [31%] of 142 patients). Physicians frequently under-reported symptoms in their patients. For example, gastrointestinal symptoms were consistently under-reported, including difficulty swallowing (28 [78%] of 36 patients), decreased appetite (75 [77%] of 98 participants), nausea (43 [70%] of 61 participants), and diarrhoea (60 [71%] of 85 participants).

INTERPRETATION:

Our findings could assist clinicians in delivering more targeted and timely supportive care interventions and might inform patients about anticipated symptoms during the early post-CAR T-cell infusion period. Moreover, the observed discrepancies between patient-reported and clinician-reported symptoms suggest that more systematic use of patient-reported outcome measures might provide complementary insights beyond those captured through clinician assessment.

FUNDING:

Fondazione GIMEMA Franco Mandelli Onlus and Novartis Farma Italy.

2026-07-01·TRANSFUSION

Impaired hematopoiesis affects apheresis and CAR T‐cell product composition and treatment response

Article

作者: Brigitte Neuber ; Felix Korell ; Sandra Sauer ; Anita Ludwig-Husemann ; Miriam Stenzinger ; Michael Schmitt ; Peter Dreger ; Patrick Derigs ; Carsten Müller-Tidow ; Tim Sauer ; Hanna Kuhn ; Angela Hückelhoven-Krauss ; Kiavasch Farid ; Maria-Luisa Schubert ; Anita Schmitt

Abstract:

Background:

Chimeric antigen receptor (CAR) T‐cell therapy has transformed the treatment of hematologic malignancies, but its success depends on obtaining sufficient CD3 + T‐cell yields during leukapheresis. This can be difficult in heavily pretreated patients, who often show leukopenia and reduced T‐cell fitness.

Methods:

We analyzed 166 leukapheresis products from 154 patients undergoing manufacturing of CD19‐directed CAR T‐cells, including 146 from non‐Hodgkin's lymphoma (NHL) and 20 from acute lymphoblastic leukemia (ALL). Collections were performed for commercial CAR T‐cell products (axi‐cel, tisa‐cel, brexu‐cel, liso‐cel; n = 121) and the HD‐CAR‐1 trial with in‐house manufacturing (heidagenlecleucel; n = 45).

Results:

In 150/154 patients, a sufficient CD3+ T‐cell yield was achieved by a single leukapheresis. Pre‐apheresis lymphocyte count strongly predicted CD3+ T‐cell yield ( p < .001) and was associated with treatment response ( p = .044). Impaired hematopoiesis, reflected by reduced nucleated cell count ( p < .001), lymphocyte count ( p <.001), and hematocrit ( p = .017), was linked to poorer collection efficiency. Intensive prior therapies, including stem cell transplantation, reduced CAR T‐cell expansion during manufacturing ( p = .036). In patients with ALL , higher proportions of effector and CD8+CAR T‐cells in the product correlated with improved clinical outcomes ( p = .04; p = .023).

Conclusion:

These findings highlight the importance of early leukapheresis, ideally before intensive treatments, to optimize T‐cell yield, product quality, and therapeutic efficacy.

749

项与布瑞基奥仑赛相关的新闻（医药）

2026-08-17

·BPDx生物药工艺

818/828落地，如何选择走“术”还是走“药”？

易贸医疗会议团队编辑 2026年5月，国务院令第818号《生物医学新技术临床研究和临床转化应用管理条例》与第828号《药品管理法实施条例》相继施行。一部管“术”，一部管“药”。这套被行业称为“双轨制”的监管框架运行百余天后，在2026BPD第九届生物药工艺发展大会论坛现场迎来了两场深度讨论，一场聚焦法规逻辑与路径选择，一场聚焦生产质量体系的落地实操。来自监管研究、学术机构、产业一线等不同领域的八位嘉宾，共同勾勒出行业在规则重塑期的集体思考。 1 818号令：从征求意见到正式施行 2026BPD上海 818号令的出台，源头可追溯至数年前基因编辑领域的重大伦理事件。那次事件暴露了法律层面的空白，新技术在临床研究中的边界、责任主体、违规后果，都缺少明确界定。此后，国家卫健委于2019年发布《生物医学新技术临床应用管理条例（征求意见稿）》，向社会公开征求意见。从征求意见到国务院正式公布条例，前后历时约六年。六年里，中国CGT产业从零星探索走向规模化发展，从跟随式创新逐步转向源头创新。当818号令最终落地时，政策制定者已将这一变化融入其中。中国药品监督管理研究会细胞与基因治疗产品监管研究专业委员会主任委员王越指出，818号令作为国务院条例，在法律阶位上仅次于立法。它的意义可以从两个层面理解。第一个层面是划清合规底线，此前的IIT研究存在大量灰色地带，有些甚至触及违规。第二个层面是在合规前提下鼓励创新，监管收紧的目的并非限制发展。中国药科大学生物医药卓越工程师学院教授郭炜参与了818号令配套实施指引的撰写。他透露，政策制定之初就确立了“原本能做的IIT尽量继续能做”的原则，国家不希望因监管收紧而扼杀了生物医药技术创新的势头。国家干细胞转化资源库副主任贾文文将818号令定位为“立规矩”，把原来靠灰色模式运作的空间用规则填满。在他看来，这对行业来说是进步，称不上约束。 2 路径选择：不是二选一，是对号入座 2026BPD上海两套监管框架并行，企业和研究者需要在“技术路径”与“药品路径”之间做出战略抉择。先看备案实践。以协和医院首批通过的项目为例，这些项目有几个共同特征：研究周期普遍较长，曾获国家专项支持，临床研究深入且有持续的数据支撑。王越认为，这些个案说明818号令的备案通道有其明确边界。他引用贾文文报告中的数据：85%到90%以上的项目其实并不适用这条路径，真正承压的是年轻的科学家和初创团队。从业者首先要做的，是对自身项目做客观评估，而不是反过来要求规则为自己让步。贾文文从企业决策角度给出了更具体的判断。如果产品疗效已在人体上得到明确验证，且具备药物属性，直接走药品路径是更务实的选择。818号令的技术路径更适合那些需要与医院深度绑定、进行复杂新技术联合开发的场景，比如骨科中需要结合3D建模与细胞修复的个性化治疗。企业在项目启动之初就应该向合作医院亮明自己的最终目标，为了技术收费，还是为了后续申报药品。这两条路，医院对项目的支持方式和路径设计完全不同。如果企业最终想靠药品获利，却试图通过技术路径绕道而行，在贾文文看来意义不大。即便技术路径最终实现收费，收益主体也是医院，企业从中分到的有限。郭炜从基因治疗的特殊性做了补充。基因治疗与细胞治疗在路径选择上差异显著。绝大多数基因治疗产品未来仍会走向药品方向，但超罕见病的情况不同，商业回报有限，药企缺乏投入动力，技术转化路径反而能为患者提供可及的治疗方案。他还提到一种“组合路径”：如果一个产品既有罕见病适应症又有常见病适应症，可以先走技术路径满足罕见病患者的需求，同时积累临床数据，再走药品路径申报常见病适应症。他还透露，卫健委对“同机制、同靶点”的界定原则仍在动态调整中。随着实践推进和意见反馈，界定原则会根据行业发展的实际需要不断优化。企业不必因为“别人已获批”就自我设限，关键在于自身产品的差异化竞争力。先声药业总监方淑平主持本场访谈，她在总结时梳理了讨论的共识：备案阶段仍以鼓励创新为主，多数IIT研究仍可按818号令继续推进。但转化的门槛确实较高，企业需要审慎评估自身条件再做出路选择。 3 质量体系：一套逻辑，无需两套 2026BPD上海与路径选择上的审慎权衡不同，生产与质量体系的讨论呈现出高度共识。艺妙神州联合创始人兼CTO齐菲菲表示，818号令的实施分为备案与转化两个阶段。备案阶段的要求与早期IIT差别不大，但到了转化阶段，理论上必须按药企标准执行。艺妙神州的文件体系始终按照药品GMP体系搭建，从未建立两套体系。科金生物首席技术官陈强更进一步指出，“两套体系”这个说法本身就不成立。如果项目是医生主导的医院项目，沿用医院原有体系即可。如果是企业发起的项目，冲着IND去的，就必须按GMP执行。亦诺微CTO黄青则从法规演进的角度补充了一个细节：818号令对生物医学新技术提出了“符合GMP标准”和“遵循QBD原则”的双重要求。QBD理念在药品监管中的引入不过二十余年，而GMP已推行近六十年。这意味着即便是“技术”路径，质量门槛也已向药品标准看齐。不过，陈强在讨论中抛出了一个值得深思的差异点。他观察到，在医院主导的IIT项目中，医生群体对GMP和CMC的认知与药企从业人员存在明显差距。他直言，“GMP like”是个伪概念——GMP就是GMP，非GMP就是非GMP，中间状态并不存在。这个提醒的背后，是IIT研究中大量存在“似是而非”的质量管理实践，尤其在医院主导的项目中更为突出。齐菲菲进一步指出，细胞治疗产品的物料控制要求远高于传统生物制品。细胞不能过滤除菌，也不能高温灭菌，所有物料的无菌与安全性保障必须达到更高标准。同时，他还澄清了一个常见的概念误区，所谓“GMP级别物料”其实并不存在。GMP是针对药品生产过程的规范，不是原材料的属性标签，“GMP级别”更多是供应商的营销话术。茵冠生物技术VP、本场主持人江颖纯在引导讨论时分享了她的观察。几位嘉宾的观点与茵冠生物的实践一致，两套监管体系共享同一套底层逻辑，都建立在GMP之上。批记录、放行等具体操作虽有调整空间，但法律法规的依据清晰且经过验证。茵冠生物内部奉行的原则是“就高不就低”——监管体系哪个标准更严格，就参照哪个执行，而不是等待法规给出宽松空间。CGT行业的生产质量极其严肃，企业不应指望监管放松要求。 4 工艺验证：自体产品的特殊挑战 2026BPD上海对于自体CAR-T这类个性化产品，工艺验证的策略需要专门设计。齐菲菲指出，自体产品的工艺验证首先要评估样本的代表性，用供者细胞还是患者细胞，这本身就是个关键决策。更重要的区别在于产能验证，传统生物制品生产一批可以供应大量患者，自体细胞产品真正的挑战在于多批次同时生产时，整个体系的稳定性。指导原则明确要求将工艺验证与产能验证融合，要在最大产能、最差条件下验证工艺是否仍能稳定运行。也正因如此，获批的产能本身就是监管审批的内容，企业能够同时启动多少批次、培养多少批次、收获多少批次，都要写入获批文件。上市后如果要扩大产能，必然会触发监管核查，监管部门需要确认体系在扩产条件下仍能稳定运行。在溶瘤病毒与CAR-T的差异方面，黄青提出溶瘤病毒的工艺验证策略更接近抗体药物，三批验证仍是基本要求。FDA近年对ATMP产品有所放宽，但溶瘤病毒领域仍遵循传统路径。陈强提醒，工艺验证的时机选择至关重要。在他看来，IIT阶段谈工艺验证为时过早，传统药企常在III期临床之后才做完整工艺验证。早期临床的质量标准应当留出足够的宽容度，因为研究者需要从中学习和发现产品的关键质量属性，不应该在数据尚不充分时就把自己限定在过窄的范围内。到了III期，标准自然会逐步收窄。而一旦收窄，再想放宽就得向药监部门提供充分理由，这个过程往往非常困难。 5 变更管理：细胞产品的“变”与“不变” 2026BPD上海从IND到BLA，CMC变更不可避免。2024年CDE在上海举办的相关培训中，专门就细胞治疗产品变更管理的疑难问题进行了详细解答。2025年7月，CDE发布了《细胞治疗药品药学变更研究与评价技术指导原则（征求意见稿）》；2026年1月，该指导原则正式施行。齐菲菲指出，这份原则之所以出台，是因为行业变更太多，而且对变更的认知过于轻率。指导原则在附录中列举了已上市产品的变更案例，明确了哪些属于重大、中等、微小变更。关键原材料的变更一律被视为重大变更，即便完成了完善的药学可比性研究，仍可能不足以支持变更，有时需要桥接非临床甚至临床研究。背后的原因在于：当前对细胞产品的分析表征手段远远不够。检测的十项、二十项指标呈现可比性，并不能证明变更前后的产品真正等同，可能只是没有用到足够灵敏的方法去发现差异。他举了两个案例。FDA批准的Yescarta和Tecartus两款CAR-T产品，在CAR分子和病毒载体上完全一致，区别仅在于后者增加了一个细胞分选步骤。在他看来，FDA正是认为加了一步操作之后的产品已不能视为同一产品，因此将其作为独立品种获批。再看国内，药明巨诺的倍诺达最早获批时使用的病毒载体由美国供应，后来为降低成本将载体生产转至国内。这一变更被CDE认定为重大变更，要求完成桥接临床研究后方可获批（目前该补充申请仍在审评中）。这两个案例揭示了一个深层逻辑：对于细胞这类“活的药物”，变更的审评尺度远比其他生物制品严苛。黄青从药品开发全周期的角度给出建议。药品开发分为不同阶段，质量管理也相应地从松到紧演进，一期临床的核心是安全性，标准可以相对宽泛；二期关注有效性；到了商业化阶段，标准必须高度收紧。变更也是同理，应当在工艺验证前完成重大变更，因为验证的目的是确认工艺在既定范围内的持续稳定运行，而不是探索边界。上市后的变更，尤其是涉及关键原材料的变更，难度会成倍增加。陈强用GMP的核心理念做了总结，任何变更都必须以文件记录为支撑，这是质量管理不变的铁律。陈强还分享了一个切身案例。他曾负责一个儿童适应症IND项目，在充分评估风险后决定不召开pre-IND会议，直接提交申请，最终比预期提前一个月获得默许。他的经验是：清晰的自我判断和对风险的充分认知，有时候比程式化地走流程更有效率。 6 临床数据的桥梁：前提比结论更重要 2026BPD上海 818号令产生的临床数据能否支持药品注册申报？这是企业最关心的问题之一。王越的观点很有代表性。IIT数据能否用于IND，关键在前端，备案阶段的方案设计是否已前瞻性地借鉴IND的基本原则，数据质量是否有严格把控。如果不谈这些前提条件，简单地要求用IIT数据作为新药申报的依据，这条路走不通。他进一步指出，如果研究者从一开始就考虑到未来申报药品的可能性，备案阶段的方案设计、数据采集、质量管理的标准都应该向IND靠拢，这能为后续数据的“被认可”创造条件。郭炜补充GCP标准是底线，研究方案应相当于临床一期的设计，生产应使用与未来IND申报同批次的工艺。满足这些条件越多，被认可的概率就越高。他还透露了关于第三方复核检验的要求，最初卫健委从细胞治疗的既往规定中引入了这一概念，但专家团队已建议参考药品IND的模式，临床研究阶段免除第三方复核，到转化应用阶段再做要求。这是正在讨论中的动向，卫健委正在认真考虑这个建议。贾文文则点出了一个逻辑上的矛盾。如果CMC和临床研究完全按药品标准执行，那可能一开始就不该走818号令，而应该直接走828号令。用“药品标准”做“技术路径”的研究，再用这些数据去申请“药品路径”的注册，在效率上未必划算。他还提醒，818号令的评审由卫健委独立组织，已不再是过去卫健与药监联合评审的机制，沟通需要分别进行。过去国家备案项目中那种联合评审的模式，在818号令实施后已经不再延续。方淑平在讨论收束时点明了问题的本质。IIT和IND面对的都是病人，安全性的底线是一致的。818号令的指引明确了申请人需要考虑的维度，但具体要做到什么程度，最终取决于企业对产品风险的理解和把控能力。 2026BPD上海两场讨论勾勒出818/828号令落地后的行业图景：合规的底线已经划清，创新的空间依然敞开，路径的选择需要战略眼光，质量的标尺没有第二条。同一套质量基石上，818/828号令为不同成熟度、不同属性的技术产品提供了差异化的转化通道。陈强说得直白，变是常态，但“documentation”是支撑一切变更的根基。黄青也强调，质量管理与药品开发阶段一脉相承，由松到紧但逻辑统一。齐菲菲反复提及贯穿始终的那条底线——“患者安全性”。而在讨论之后，落地才是关键。往期精选 2026.8.10 7000+工艺人年度集结！2026BPD第九届生物药工艺发展大会，今日盛大开幕九届深耕工艺，以CMC链接全球！2026BPD上海圆满闭幕！狮城待发！下一站新加坡 2026BPD新加坡生物药工艺发展大会 2026年9月22-23日新加坡会议规模 500+ 参会者，50+ 讲者会议亮点以工艺与生产能力，拓展亚太尤其东南亚地区市场潜力。国内限时免费注册快人一步扫码注册添加小易加速审核微站开启扫码了解完整内容参展咨询展位预订 18516293589 2026BPD新加坡讲者阵容 2026BPD新加坡日程模块

2026-08-14

·PRNewswire

CAR T-Cell Therapy for Acute Lymphoblastic Leukemia Market to Grow Significantly at a CAGR of 6.8% During the Forecast Period (2026-2036), Driven by Emerging Therapies and Expanding Pipeline | DelveInsight

The dynamics of CAR T-cell therapy in ALL in the market are anticipated to change in the coming years owing to improvements in research and development activities for efficient treatment options to be available in the market. The launch of emerging therapies such as Lasme-cel (UCART22) (Cellectis), WU-CART-007 (Wugen), CTD402 (Imviva), Obe-cel and AUTO1/22 (Autolus Therapeutics), and others is expected during the forecast period of 2025–2034. LAS VEGAS, Aug. 12, 2026 /PRNewswire/ -- Recently published CAR T-Cell Therapy for ALL Market Insights report includes a comprehensive understanding of current treatment practices, CAR T-cell therapy for ALL emerging drugs, market share of individual therapies, and current and forecasted market size from 2022 to 2036, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. CAR T-Cell Therapy for ALL Market Summary The market size for CAR T-cell therapy for ALL was found to be USD 3.2 billion in the 7MM in 2025. The United States accounted for the largest CAR T-cell therapy for ALL treatment market size in the 7MM in 2025, compared to other major markets, including the EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The total incident cases of ALL in the 7MM were approximately 12,000 in 2025. Leading CAR T-cell therapy for ALL companies, such as Cellectis, Wugen, Imviva, Autolus Therapeutics, Fate Therapeutics, PeproMene Bio, and others, are developing new CAR T-cell therapy for ALL treatment drugs that can be available in the CAR T-cell therapy for ALL market in the coming years. The promising CAR T-cell therapies for ALL in clinical trials include Lasme-cel (UCART22), WU-CART-007, CTD402, Obe-cel, AUTO1/22, FT839, PMB-101, and others. Discover what is the future of the CAR T-cell therapy for ALL treatment market @ Key Factors Driving the Growth of the CAR T-Cell Therapy for ALL Market Rising Incidence of Relapsed/Refractory ALL: An increasing number of patients with relapsed or refractory ALL continues to create demand for effective treatment options. CAR T-cell therapy offers a promising alternative for patients who have limited success with conventional chemotherapy or stem cell transplantation. Advancements in CAR T-Cell Technology: Continuous innovation in CAR design, manufacturing processes, and next-generation constructs is improving treatment efficacy, persistence, and safety. The development of allogeneic and dual-target CAR T-cell therapies is expected to further expand the market. Superior Clinical Outcomes and Durable Responses: CAR T-cell therapies have demonstrated high complete remission rates and durable long-term responses, particularly in pediatric and young adult B-cell ALL. These encouraging clinical outcomes are driving greater physician confidence and broader adoption. Strong Pipeline of Investigational Therapies: Numerous clinical-stage CAR T-cell candidates such as Lasme-cel (UCART22) (Cellectis), WU-CART-007 (Wugen), CTD402 (Imviva), Obe-cel and AUTO1/22 (Autolus Therapeutics), FT839 (Fate Therapeutics), PMB-101 (PeproMene Bio), and others are advancing through development. This robust pipeline is expected to introduce new treatment options and sustain long-term market growth. As per Aparna Thakur, Project Manager of Forecasting at DelveInsight, CAR-T cell therapies need more data supporting their use in earlier lines of therapy. Patients are likely to have better outcomes if they receive CAR-T cell therapy before multiple lines of chemotherapy, like those in the clinical trials that led to FDA approval. Moreover, even though the reimbursement of CAR-T therapies represents a challenge for healthcare providers, insurers, and government agencies, they have successfully reached the majority of the markets by considering the patient pool demand. CAR T-Cell Therapy for ALL Market Analysis CAR-T cell therapy has transformed the treatment landscape by offering the potential for durable, long-lasting responses with a single administration. This approach may reduce the need for intensive salvage chemotherapy and autologous stem cell transplantation, thereby minimizing treatment-related toxicity in patients with high-risk disease. Its introduction has redefined the standard of care for individuals with primary refractory disease or those who experience an early relapse after frontline treatment. The rapid evolution of immunotherapy has been marked by the emergence of CAR-T cell therapies, which represent a significant advancement in the management of Acute Lymphocytic Leukemia (ALL). A major milestone was achieved in 2017 when the US FDA approved KYMRIAH (tisagenlecleucel), the first CAR-T cell therapy indicated for ALL. This was followed in October 2021 by the approval of TECARTUS (brexucabtagene autoleucel) for adults aged 26 years and older with relapsed or refractory (R/R) B-cell precursor ALL. Further progress was made in November 2024 with the FDA approval of AUCATZYL (obecabtagene autoleucel), a CD19-directed genetically engineered autologous T-cell therapy, for adults with relapsed or refractory B-cell precursor ALL. The CAR-T therapy pipeline continues to expand with promising investigational candidates, including Lasme-cel (UCART22) (Cellectis), WU-CART-007 (Wugen), CTD402 (Imviva), Obe-cel and AUTO1/22 (Autolus Therapeutics), FT839 (Fate Therapeutics), PMB-101 (PeproMene Bio), and several others. Many of these emerging therapies have demonstrated encouraging efficacy and favorable safety profiles in early- and mid-stage clinical studies, highlighting their potential to further improve outcomes for patients with ALL. CAR T-Cell Therapy for ALL Competitive Landscape Some of the CAR T-cell therapies for ALL under development include Lasme-cel (UCART22) (Cellectis), WU-CART-007 (Wugen), CTD402 (Imviva), Obe-cel and AUTO1/22 (Autolus Therapeutics), FT839 (Fate Therapeutics), PMB-101 (PeproMene Bio), and others. Cellectis' Lasme-cel is expected to address a significant share of the eligible market by offering several potential advantages over CD19-directed therapies, including a distinct therapeutic target, a single-dose treatment approach, off-the-shelf availability, and the ability to achieve deep minimal residual disease (MRD) responses in patients receiving third-line or later therapy. Wugen's WU-CART-007 is an investigational allogeneic, off-the-shelf CAR-T cell therapy targeting CD7. It has been engineered with a fratricide-resistant design to overcome key manufacturing and biological challenges associated with developing CAR-T therapies for CD7-positive hematologic malignancies. In August 2025, Wugen secured USD 115 million through an equity financing round led by Fidelity Management & Research Company, with additional investments from RiverVest Venture Partners, Lightchain Capital, Abingworth, ICG, LYZZ Capital, Tybourne Capital Management, Aisling Capital Management, and other prominent life sciences investors. The funding is intended to accelerate the ongoing pivotal T-RRex trial evaluating WU-CART-007 in patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). The anticipated launch of these emerging therapies are poised to transform the CAR T-cell therapy for ALL market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the CAR T-cell therapy for ALL market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about which pipeline drugs could transform CAR T-cell therapy for ALL treatment @ CAR T-Cell Therapy for ALL Drugs Recent Developments in the CAR T-Cell Therapy for ALL Market In June 2026, Cellectis announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to lasmecabtagene timgedleucel (lasme-cel), its investigational CD22-directed allogeneic CAR-T cell therapy, for the treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). In April 2026, Wugen announced that the FDA has selected its investigational allogeneic CAR-T cell therapy, Sofi-cel, for participation in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program. The company was invited to join the initiative, which annually selects up to nine development programs across the FDA's biologics and drug review centers. In April 2026, Autolus Therapeutics unveils new ROKA Real-World and pediatric data supporting obe-cel in ALL. In January 2026, the FDA awarded Orphan Drug Designation (ODD) to CTD402, an innovative allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of patients with relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and lymphoblastic lymphoma (T-LBL). CAR T-Cell Therapy for ALL Epidemiology Segmentation The CAR T-cell therapy for ALL epidemiology section provides insights into the historical and current CAR T-cell therapy for ALL patient pool and forecasted trends for the leading markets. Among the type-specific cases of ALL in the EU4 and the UK, B-cell lymphoblastic leukemia cases were almost 4 times higher than cases of T-cell lymphoblastic leukemia in 2025. The CAR T-cell therapy for ALL treatment market report proffers epidemiological analysis for the study period 2022–2036 in the leading markets, segmented into: Total Incident Cases of ALL Gender-specific Incident Cases of ALL Age-specific Incident Cases of ALL Subtype-specific Incident Cases of ALL Genetic mutation-specific Incident Cases of ALL Total Treated Cases of ALL Scope of the CAR T-Cell Therapy for ALL Market Report CAR T-Cell Therapy for ALL Patient Population Forecast CAR T-Cell Therapy for ALL Therapeutics Market Size CAR T-Cell Therapy for ALL Pipeline Analysis CAR T-Cell Therapy for ALL Market Size and Trends CAR T-Cell Therapy for ALL Market Opportunity CAR T-Cell Therapy for ALL Market Unmet Needs KOL's Views on CAR T-Cell Therapy for ALL CAR T-Cell Therapy for ALL Market Access and Reimbursement Download the report to understand the top emerging CAR T-cell therapies for ALL @ CAR T-Cell Therapy for ALL Clinical Trials Table of Contents Related Reports CAR T-Cell Therapy Market CAR T-Cell Therapy Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key CAR T-cell therapy companies, including CytoAgents, Genentech, Incyte Corporation, Caribou Biosciences, Chimeric Therapeutics, Allogene Therapeutics, Kyverna Therapeutics, iCell Gene Therapeutics, Synthekine, Janssen Research & Development, SecuraBio, ImmPACT Bio, A2 Biotherapeutics, Gracell Biopharmaceuticals, Wugen, Sana Biotechnology, Oncternal Therapeutics, Vor Biopharma, CARsgen Therapeutics, Autolus Limited, Arcellx, Kite, Novartis, Lyell Immunopharma, ImmPACT Bio, Tmunity Therapeutics, Poseida Therapeutics, Precigen, AffyImmune Therapeutics, 2seventy bio, Takeda, Cartesian Therapeutics, Cabaletta Bio, Legend Biotech, Miltenyi Biomedicine, Beam Therapeutics, and others. CAR-T Market CAR-T Market Size, Target Population, Competitive Landscape & Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key CAR-T companies, including Alnylam Pharmaceuticals, JW Therapeutics, Gilead Sciences, Janssen Pharmaceuticals, Innovent Biologics, Sorrento Therapeutics, Cartesian Therapeutics, CASI Pharmaceuticals, Juventas Cell Therapy, Novartis, Poseida Therapeutics,Shanghai Unicar-Therapy Bio-medicine Technology, Sinobioway Cell Therapy, Tessa Therapeutics, Wuhan Bio-Raid Biotechnology, Miltenyi Biomedicine, Bristol-Myers Squibb, Autolus Limited, Beijing Immunochina Medical Science and Technology, Carsgen Therapeutics, Cellular Biomedicine Group, Chongqing Precision Biotech, Eureka Therapeutics, Formula Pharmaceuticals, Guangzhou Bio-gene Technology, Hebei Senlang Biotechnology, Mustang Bio, MolMed, Aurora BioPharma, Atara Biotherapeutics, Autolus, Bellicum Pharmaceuticals, Kecellitics Biotech Company, Yake Biotechnology, Minerva Biotechnologies, Allogene Therapeutics, PersonGen BioTherapeutics (Suzhou), Precision BioSciences, Pregene (ShenZhen) Biotechnology Company, Shanghai GeneChem, Shanghai Longyao Biotechnology, Shenzhen BinDeBio, and others. Acute Lymphoblastic Leukemia Market Acute Lymphoblastic Leukemia Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ALL companies, including AbbVie, Roche (Genentech), Janssen Research & Development, Pharmacyclics (an AbbVie Company), Incyte Corporation, Novartis, Jazz Pharmaceuticals, Autolus Therapeutics, Syndax Pharmaceuticals, Cellectis, ADC Therapeutics, Wugen, Servier, Allogene, Amgen, Astellas Pharma, Gilead Sciences, Syndax, Incyte, Orca Biosystems , and others. Acute Lymphoblastic Leukemia Clinical Trial Analysis Acute Lymphoblastic Leukemia Pipeline Insight – 2026 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ALL companies, including AbbVie, Roche (Genentech), Janssen Research & Development, Pharmacyclics (an AbbVie Company), Incyte Corporation, Novartis, Jazz Pharmaceuticals, Autolus Therapeutics, Syndax Pharmaceuticals, Cellectis, ADC Therapeutics, Wugen, Servier, Allogene, Amgen, Astellas Pharma, Gilead Sciences, Syndax, Incyte, Orca Biosystems, and others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us: Shruti Thakur [email protected] +14699457679 SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo

免疫疗法细胞疗法上市批准孤儿药ASCO会议

2026-08-10

·全球GCT研究

免疫重置革命：自免成为全球MNC和CGT主战场

从一位狼疮患者的困境说起:3-5亿自免患者·自免药物TOP22销售$1,207亿(CGT的16倍)·免疫重置让94%患者停药·Top50药企自免管线图谱·七笔in vivo并购$177亿·iPSC/表观遗传/体细胞全景·未来十年爆炸性成果·对全人类健康长寿影响 GCT前沿·Gangtise/各公司年报/NEJM/ARM·📖 约 13078 字 · 32 分钟·🔗 NEJM 2025体内CAR-T SLE数据一、引言:一场改写数亿人命运的"免疫重置革命" 想象一位28岁的红斑狼疮(SLE)患者:22岁确诊,过去6年每天服用激素和免疫抑制剂,脸因激素浮肿,反复发烧、关节痛、肾脏报警,医生告诉她:"这种病要终身用药,只能控制,无法治愈"。全球像她这样的自身免疫病患者有3-5亿人——这个数字,是癌症患者总数的4倍以上[^7][^32]。如果你是一名患者或普通人——你最关心的是:“我的病有没有可能被治好、不再终身吃药?”过去两年,答案正在改变:全球首个“免疫重置”疗法让难治性狼疮患者实现长期无药缓解,4年随访持续有效[^4];2025年全球自免药物TOP22销售额$1,207亿——这是一个比CGT全市场大16倍的市场,意味着药企有巨大动力为你研发新药[^32]。如果你是一名医生——你看到的是治疗范式的转换:从“终身抑制免疫”(激素/生物制剂)到“一次性重置免疫”(CAR-T/TCE清除致病B细胞)。BMS zola-cel让94%的难治性自免患者停用免疫抑制治疗[^2];AZD0120在SLE实现90%缓解[^3]。你需要知道:哪些患者适合、何时转诊、风险如何管理——这可能是你职业生涯中最重要的治疗工具升级。如果你是一名生物学研究者——这是免疫学最激动人心的时刻:CAR-T靶向CD19(B细胞)/BCMA(浆细胞)深度清除后,B细胞再生重建免疫耐受,免疫系统“重新学习认识自我”[^8][^9];表观遗传编辑(不切DNA调控表达)、iPSC现货细胞工厂、体内递送技术,正把“免疫重置”从血液肿瘤复制到自免、纤维化、神经退行[^19][^25]。如果你是一名biotech从业者——资本已经用脚投票:2025-2026年in vivo CAR-T七笔并购约$177亿(礼来$70亿Kelonia、强生$33.65亿Sail)[^4][^5][^9];Top50药企中30+家涉足CGT、累计投入$800亿+[^1];自免取代肿瘤成为CGT主战场,BD窗口2027-2029年最为确定[^37]。全景数据:2025年CGT产品全球销售约$75亿(CAR-T六款占89%)[^1];2026年上半年Carvykti约$12.5亿、Casgevy单季$7,600万创历史新高[^12][^13][^14][^15];in vivo CAR-T七笔重磅交易合计约$177亿[^3][^4][^5][^6][^7][^8][^16]。本文要回答的核心问题:免疫重置革命的技术逻辑是什么?全球MNC如何布局自免管线?哪些公司最可能成为BD买方?未来十年哪些技术将带来爆炸性成果?这场革命对全人类的疾病治疗与健康长寿意味着什么?——无论你属于哪类读者,这篇文章都与你有关。二、存量格局:2025年CGT销售Top10与商业化验证图1|2025年全球CGT产品销售额Top10(CAR-T六款主导约$58亿) · 查看原文表1|2025年全球CGT产品销售额Top10 # 产品公司类型靶点适应症 2025销售同比 1 CARVYKTI 强生/传奇 CAR-T BCMA 多发性骨髓瘤 $18.87亿 +95.9% 2 YESCARTA 吉利德/Kite CAR-T CD19 LBCL $14.95亿 -5% 3 BREYANZI BMS CAR-T CD19 LBCL/MZL $13.58亿 +82% 4 ZOLGENSMA 诺华 AAV基因治疗 SMN1 SMA ~$10亿 -5% 5 ELEVIDYS Sarepta AAV基因治疗微肌营养不良蛋白 DMD $8.99亿 +119% 6 TECARTUS 吉利德/Kite CAR-T CD19 MCL/ALL $4.8亿 +12% 7 ABECMA BMS/蓝鸟 CAR-T BCMA 多发性骨髓瘤 $4.5亿 -8% 8 KYMRIAH 诺华 CAR-T CD19 ALL/DLBCL $3.81亿 -14% 9 CASGEVY Vertex/CRISPR Tx CRISPR基因编辑 BCL11A SCD/β-地贫 $1.16亿放量初期 10 LUXTURNA Spark/罗氏 AAV基因治疗 RPE65 遗传性视网膜病 ~$1亿平稳关键洞察一:前线拓展驱动爆发式增长。Carvykti(+95.9%至$18.87亿)与Breyanzi(+82%至$13.58亿)凭借向更前线适应症(二线/一线)拓展实现爆发增长——这证明CAR-T的商业化天花板远未触及,每一次适应症前移都在再造一个"新药"。关键洞察二:首代产品进入平台期。Yescarta(-5%)、Kymriah(-14%)、Abecma(-8%)增速停滞,反映首代CAR-T在末线市场的饱和——行业需要新一代技术(异体、体内、自免适应症)打开第二增长曲线。关键洞察三:基因治疗与基因编辑进入放量前夜。Zolgensma(FDA 2025.12扩龄至≥2岁后2026年有望回升至~$14亿)、Elevidys(+119%至$8.99亿)证明AAV基因治疗在大适应症(DMD/SMA)上的商业化潜力;Casgevy($1.16亿,全球首款CRISPR疗法)仍处放量初期,Goldman Sachs峰值预期$39亿[^1][^2]。二·五、2026年上半年(H1)已上市CGT药品销售全景表6|2026年上半年(H1)已上市CGT药品全球销售数据(截至2026.8.9) 产品公司类型 2026 H1/Q2销售同比/环比要点 CARVYKTI 强生/传奇 CAR-T(BCMA) H1约$12.5亿持续增长前线拓展(CARTITUDE-4)驱动,继续稳居全球最畅销CAR-T BREYANZI BMS CAR-T(CD19) Q2 $4.84亿超华尔街预期 LBCL/MZL放量,带动BMS上调2026全年指引至~$490-500亿 YESCARTA 吉利德/Kite CAR-T(CD19) Q2 $3.46亿 -12% YoY 美国需求仍增但竞争加剧;环比+2%企稳 CASGEVY Vertex/CRISPR Tx CRISPR基因编辑 Q2 $7,600万(H1约$1.19亿) +151% YoY/+78%环比创上市以来单季新高,放量全面加速 TECARTUS 吉利德/Kite CAR-T(CD19) Q2 $7,000万 -24% YoY Kite Q2细胞治疗合计$4.17亿(-14% YoY) ABECMA BMS/蓝鸟 CAR-T(BCMA) Q2 $7,200万 -17% YoY 受Carvykti前线拓展挤压,份额承压 ZOLGENSMA 诺华 AAV基因治疗未见H1单独披露 2025全年约$10亿 FDA 2025.12扩龄至≥2岁后2026年有望回升至~$14亿 ELEVIDYS Sarepta AAV基因治疗未见H1单独披露 2025全年$8.99亿(+119%) DMD适应症扩展与全球上市推进中 2026 H1关键洞察:(1)Casgevy进入放量快车道——Q2销售$7,600万,同比+151%、环比+78%,创上市以来单季新高,基因编辑的"商业化验证"正在兑现[^14][^15];(2)Carvykti继续领跑——H1约$12.5亿,前线拓展战略持续兑现,进一步拉开与Yescarta/Breyanzi的差距[^12];(3)首代CAR-T分化加剧——Yescarta Q2 -12%、Tecartus -24%、Abecma -17%,而Breyanzi Q2 $4.84亿超预期(带动BMS上调2026全年指引至~$490-500亿)[^13];(4)Kite整体Q2细胞治疗销售$4.17亿(-14% YoY)但环比+2%,美国需求企稳[^17]。(注:Zolgensma/Elevidys未见H1单独披露,以2025全年为基准。)三、第一梯队:七大MNC的完整CGT能力谱系表2|第一梯队:七大MNC完整CGT能力谱系(2026) 公司 CGT技术平台核心CGT资产/管线自免布局 2025-2026关键动作礼来(市值~$1.07万亿) in vivo CAR-T(LNP+慢病毒双路径) Orna LNP-oRNA、Kelonia iGPS、Verve碱基编辑、Adverum AAV B细胞自免 $24亿Orna+$70亿Kelonia收购,全球最激进CGT布局诺华(~$2,810亿) 自体CAR-T+AAV+siRNA Kymriah、YTB323(SLE Ph2)、Zolgensma SLE/LN/SSc/肌炎(Ph2关键) 收购Kate(下一代AAV)、DTX(siRNA);自免CAR-T临床最靠前吉利德(~$2,190亿) Kite自体CAR-T+异体+in vivo Yescarta+Tecartus、anito-cel(Ph3)、Interius in vivo CM336 BCMA/CD3自免双抗 $3.5亿Interius+$21.75亿Arcellx(2026.2);制造能力行业最强强生(~$5,500亿) 自体CAR-T(Carvykti)+in vivo(双平台) Carvykti、传奇TaVec(LB2501)、Sail体内CAR-T Carvykti+传奇in vivo+Sail(自免免疫重置) $7.85亿首付+$25.8亿收购选择权绑定Sail(2026.7.29);传奇LB2501 EHA 2026:100% ORR/83.3% CR BMS(~$1,100亿) 自体CAR-T+in vivo(LNP) Breyanzi、zola-cel(CD19自免Ph3)、Orbital SLE/SSc/肌炎/RA(Breakfree研究) $15亿Orbital(2025.10);zola-cel自免CAR-T先锋,有望最先获批阿斯利康(~$2,710亿) 慢病毒in vivo+TCE+Treg EsoBiotech ENaBL、AZD0120(BCMA/CD19)、亘喜GC012F、Quell Treg AZD0120 SLE 90%DORIS缓解 $10亿EsoBiotech(2025.3)+$12亿亘喜(2023.12)+西比曼+Quell(≤$2B) 艾伯维(~$3,830亿) tLNP in vivo CAR-T Capstan tLNP-CD19 CAR-T(CPTX2309 Ph1) 自免(Ph1启动,2026数据待读) $21亿Capstan(2025.6);免疫学全球领导地位+通用CAR-T战略协同礼来——全球最激进的CGT买家。市值约$1.07万亿的礼来,在2026年2-4月连续以$24亿收购Orna(LNP-oRNA体内CAR-T)、$70亿收购Kelonia(iGPS慢病毒体内CAR-T),叠加此前$13亿Verve(体内碱基编辑PCSK9,LDL降62%)、$15亿Adverum(AAV湿性AMD),累计CGT投入超$100亿[^4][^5][^8]。其"LNP-mRNA瞬时表达+慢病毒持久表达"双路径策略,无论在哪种递送技术先突破都能掌握主动权——这是2025-2026年最值得研究的CGT战略样本。诺华——自体CAR-T自免临床最靠前。拥有Zolgensma+Kymriah双上市产品,其新一代CD19 CAR-T YTB323在SLE的关键性Ph2数据预计2027-2028年读出,是第一梯队中自体CAR-T自免方向临床进度最前的公司[^1][^2]。吉利德/Kite——制造能力行业最强+异体卡位。Kite建立的自体CAR-T制造体系被公认为行业最高效(Yescarta+Tecartus约$19.8亿);2026年2月以$21.75亿收购Arcellx获得anito-cel(BCMA异体CAR-T,Ph3,96% ORR)[^7],2025年8月$3.5亿收购Interius进入in vivo[^6]。强生/传奇——Carvykti现象级放量+in vivo储备。Carvykti(+95.9%至$18.87亿)成全球最畅销CAR-T;传奇生物TaVec慢病毒in vivo平台(LB2501 CD19/CD20双靶)EHA 2026展示100% ORR/83.3% CR,2026H2将披露更多in vivo数据[^1][^2]。 BMS——自免CAR-T先锋,有望最先获批。zola-cel(CD19 CAR-T)治疗系统性硬化症(SSc)的Phase 3(Breakfree研究)已启动,叠加$15亿Orbital(体内CAR-T,OTX-201 CD19自免,2026Q3申报IND)[^3],BMS是最有可能在全球率先获批"自免CAR-T"的药企。阿斯利康——自免+肿瘤双栖的最全弹药库。$10亿EsoBiotech(慢病毒in vivo ENaBL)+$12亿亘喜(GC012F双靶CAR-T)+西比曼权益+Quell(≤$2B,Treg细胞治疗),AZD0120(BCMA/CD19双靶)在SLE实现90% DORIS缓解——AZ的自免CAR-T临床数据目前全球领先[^1][^2]。艾伯维——免疫学霸主+体内CAR-T新军。$21亿Capstan(tLNP体内CD19 CAR-T,CPTX2309 Ph1)与Skyrizi/Rinvoq($20B+/年自免收入)形成极高战略协同——若Capstan读出积极数据,艾伯维将同时拥有"小分子/生物制剂+细胞治疗"的自免全谱系武器[^4]。四、第二梯队:技术缺口与自免引进急迫性表3|第二梯队:技术缺口与自免CGT引进急迫性公司现状核心缺口驱动因素引进急迫性辉瑞 2016年Bamboo AAV;血友病B基因治疗晚期 CAR-T与自免CGT几乎为零后COVID收入真空+管线压力最高(2028-2030) 默沙东 $13亿Curon CN201 CD3/CD19双抗(自免TCE) 无任何CAR-T平台 Keytruda 2028专利悬崖极高(通用/in vivo CAR-T契合规模化生产) 赛诺菲 3条in vivo CAR-T临床前(tLNP) 细胞治疗能力≈0,无自有递送平台 Dupixent专利逐步到期高安进 BiTE平台(blinatumomab自免Ph2:SLE/RA) 无CAR-T,走TCE替代路线若TCE自免数据未达注册终点→转向in vivo 中高(2028-2029转向概率大) Vertex CASGEVY(全球首款CRISPR疗法) CAR-T与自免方向完全空白战略重心在CF/肾病/疼痛低(对自免CAR-T缺乏兴趣) 安斯泰来 2024年合作布局in vivo CAR-T(肿瘤) 自免方向无明确管线日本iPSC/基因编辑学术优势中(日本最活跃CGT药企) 辉瑞:急迫性最高。CGT布局停留在2016年Bamboo(AAV),CAR-T与自免CGT几乎为零,后COVID收入真空+管线压力使其成为2028-2030年最可能的CGT大买家[^2]。默沙东:专利悬崖驱动。Keytruda 2028年核心专利到期是硬性时间压力,已通过$13亿收购Curon CN201(CD3/CD19双抗)切入自免B细胞清除,但无CAR-T平台——通用/in vivo CAR-T契合其规模化生产核心能力,是首选收购方向[^2]。赛诺菲:Dupixent到期倒逼。3条in vivo CAR-T临床前但无自有递送平台,细胞治疗能力≈0,2027年后Dupixent专利到期将倒逼补强[^2]。安进:TCE替代路线的变数。BiTE平台(blinatumomab)已在SLE/RA启动自免Ph2——安进认同"B细胞清除治自免"逻辑但选TCE路线;若TCE自免数据未达注册终点,2028-2029年转向in vivo CAR-T的概率将显著上升[^2]。Vertex:CRISPR领袖但缺CAR-T。拥有全球首个获批CRISPR疗法Casgevy,但CAR-T/自免方向完全空白,战略重心在CF/肾病/疼痛[^2]。五、第三梯队:布局浅或空缺(含三大退出者) 诺和诺德:100%聚焦代谢/心血管,无CGT资产,但GLP-1现金流空前充裕——若自免被探索为第二增长引擎,不排除大规模收购一次性切入(低概率高影响力)[^2]。Moderna:mRNA-LNP递送与in vivo CAR-T底层共通,与Carisma合作in vivo CAR-M,但后COVID收入锐减,短期系统性投入概率低[^2]。BioNTech:mRNA平台转向肿瘤个体化疫苗,CAR-T管线规模极小[^2]。Bayer:BlueRock(iPSC帕金森Ph3)+AskBio(AAV-GDNF Ph2),聚焦神经CGT[^2]。Biogen:Sangamo合作(锌指基因调控)聚焦神经退行[^2]。三大战略退出者(未来3-5年不会引进):Takeda(2025.10退出细胞治疗)、GSK(已剥离基因治疗)、Regeneron(停止CAR-T)——它们的退出说明CGT并非所有药企的必选项,只有"免疫学+肿瘤+制造能力"三者兼备者才能驾驭[^1][^2]。六、2026年MNC研发重点与收购/投资热点图2|2025-2026年in vivo CAR-T六笔主要并购(合计$143.5亿) · 查看原文热点一:in vivo CAR-T——2026年最拥挤的赛道。2025.3 AZ-EsoBiotech($10亿)→2025.6艾伯维-Capstan($21亿)→2025.8吉利德-Interius($3.5亿)→2025.10 BMS-Orbital($15亿)→2026.2礼来-Orna($24亿)→2026.4礼来-Kelonia($70亿)→2026.7.29强生-Sail($7.85亿首付+$25.8亿收购选择权),合计约$177亿[^3][^4][^5][^6][^7][^8][^16]。强生通过Sail绑定体内CAR-T+自免免疫重置能力,与传奇TaVec形成“双平台”——这一交易密度刷新了单一技术赛道的并购纪录。热点二:自免取代肿瘤成为CGT新主战场。2025年10月NEJM发表全球首个in vivo CD19 CAR-T治疗SLE人体数据——LNP递送实现B细胞耗竭、疾病活动度下降、无重大毒性(DOI:10.1056/NEJMc2509522)[^9][^10]。叠加BMS zola-cel SSc Ph3、诺华YTB323 SLE Ph2、AZD0120 SLE 90% DORIS,自免CAR-T的概念验证已全面建立。热点三:递送平台成为估值核心。AZ从JCR引进($825M)、辉瑞从Voyager引进($630M)、Capstan tLNP、4DMT合成衣壳——递送是体内CAR-T与基因编辑的咽喉,平台公司持续获得溢价[^2]。热点四:体内基因编辑。Intellia NTLA-2002(HAE,发作减少95%)Ph3推进;Verve VERVE-102(碱基编辑PCSK9,LDL降62%)——in vivo基因编辑将重定义赛道[^2][^11]。七、2028-2030引进方向研判:三大主线主线一:通用型(异体)CAR-T的自免适应症。与自体CAR-T相比,通用CAR-T实现现货供应(off-the-shelf)、无需清淋、成本降至1/10;自免治疗窗口不追求"长期持久",中短期深度B细胞清除即可实现免疫重置和长期无药缓解。2026 EULAR大会披露的中国北恒生物CTA313异体通用型CD19 CAR-T与Artiva AB-101异体NK+CAR已展示门诊给药可行性[^2]。主线二:in vivo CAR-T与体内基因编辑。省去体外采集/改造/扩增/回输全流程,实现真正"现货药物"。礼来双路径(LNP-mRNA+慢病毒)覆盖互补技术路线;若NTLA-2002在2027-2028达注册级,将引爆新一轮in vivo基因编辑平台交易[^2][^11]。主线三:新型递送载体(LNP/新型AAV衣壳/VLP)。NEJM 2025论文证明LNP递送效率已足够启动注册临床[^9][^10]。市场价值测算:若体内或通用CAR-T在SLE/MS等大适应症读出注册级数据(2028-2029窗口),仅SLE全球市场空间即超$150亿;一个门诊给药+1/10成本的SLE CAR-T,峰值可达$50-100亿——这解释了MNC为何在技术验证早期就投入数十亿美元[^2]。七·五、iPSC分化细胞治疗全景:从神经前体细胞到肝前体细胞表7|iPSC分化细胞治疗管线全景:从神经前体细胞到肝前体细胞(2026) 分化细胞类型适应症代表管线/机构阶段关键进展多巴胺神经元帕金森病 Bayer/BlueRock Bemdaneprocel Ph3 30-35% UPDRS评分改善;全球iPSC细胞治疗进度最前管线神经前体细胞(NPC) 脑卒中、颅脑损伤、脊髓损伤 CU Anschutz等神经干细胞试验;基因工程NPC(分泌神经营养因子,如天冬氨酰酶iPSC-NPC) 早期临床 “重启脑修复”概念验证;卒中后神经再生成为iPSC热点方向心肌细胞晚期心力衰竭 Heartseed HS-001(日本,与Novo Nordisk合作) I/II期 2023年启动全球首个iPSC心肌细胞心衰试验,细胞片移植重建心肌胰岛β细胞 1型糖尿病日本iPSC胰岛细胞片(2026.3.6批准) 全球首款获批iPSC产品日本批准全球首个iPSC衍生治疗产品(胰腺胰岛细胞);中国干细胞治疗T1D/T2D逆转个案肝细胞/肝前体细胞肝硬化、晚期肝病新型肝细胞疗法(2026.5临床结果);MSC肝病研究早期临床首个针对晚期肝病的细胞疗法临床试验结果积极;肝前体细胞定向分化推进中视网膜色素上皮(RPE) 黄斑变性日本iPSC-RPE(全球首个iPSC人体试验延续) 临床黄斑变性细胞替代治疗,日本积累最长随访数据 iPSC-NK/iPSC-T 肿瘤免疫 Fate等iPSC现货NK/T I/II期现货细胞工厂方向,与MNC肿瘤管线协同 iPSC是“下一代现货细胞工厂”,其核心价值在于可分化为几乎所有体细胞类型。截至2026年,全球iPSC衍生细胞管线已覆盖神经、心脏、肝脏、胰岛、眼、肾等主要器官[^19][^21]:(1)神经前体细胞(NPC)——正被用于脑卒中、颅脑损伤与脊髓损伤的“重启脑修复”试验,部分管线通过基因工程让NPC分泌神经营养因子(如天冬氨酰酶iPSC-NPC)增强保护作用[^23];(2)肝细胞/肝前体细胞——2026年5月首个针对晚期肝病的细胞疗法临床试验结果积极,肝硬化患者迎来“细胞替代+再生”新希望[^22];(3)心肌细胞——Heartseed HS-001(日本,与Novo Nordisk合作)2023年启动全球首个iPSC心肌细胞心衰试验,细胞片移植直接重建心肌[^21];(4)胰岛β细胞——2026年3月6日日本批准全球首款iPSC衍生治疗产品(胰腺胰岛细胞片治疗1型糖尿病),这是iPSC细胞治疗的历史性节点[^24];(5)多巴胺神经元(Bemdaneprocel Ph3)、RPE(黄斑变性)、iPSC-NK/T(肿瘤)同步推进[^2]。对MNC的意义:Bayer(BlueRock)、Novo Nordisk(Heartseed合作)、住友制药等已重仓iPSC——未来十年,iPSC将补足CGT在“退行性疾病/器官修复”维度的空白,与体内CAR-T(免疫重置)、基因编辑(遗传病矫正)构成三大技术支柱。七·六、表观遗传基因治疗:从罕见病到慢病表8|表观遗传基因治疗:从罕见病到慢病(2026) 公司/管线技术平台适应症阶段关键进展 Tune Therapeutics TUNE-401 TEMPO表观沉默平台(不切断DNA,直接沉默cccDNA) 慢性乙肝(HBV) Ph1b/2a EASL 2026公布概念验证:首个直接表观沉默HBV cccDNA的临床证据,有望实现"功能性治愈" Epigenic Therapeutics(中国) CRISPR表观遗传编辑慢性乙肝临床推进中 2026年完成$6,000万B轮融资,加速乙肝表观治愈管线表观沉默疗法表观编辑(FSHD靶点) 面肩肱型肌营养不良(FSHD) 早期临床首个表观编辑临床试验数据:病毒生物标志物清除,可重复给药 Chroma Medicine等表观编辑平台高胆固醇血症、罕见遗传病临床前→IND 心血管/代谢慢病成为表观编辑新战场(PCSK9等靶点) 印迹障碍治疗表观编辑恢复印记基因表达 Beckwith-Wiedemann等印记障碍临床前表观编辑独特优势:修复印记缺陷(纯基因编辑无法实现) 表观遗传编辑是“不切断DNA的基因治疗”——通过dCas9-效应蛋白靶向DNA甲基化/组蛋白修饰,只调控基因表达、不改变序列,因此可逆、可重复给药、安全性更高[^20][^25]。2026年两大里程碑:(1)TUNE-401(EASL 2026概念验证)——直接表观沉默HBV cccDNA,是“功能性治愈乙肝”的首个临床证据,对全球2.5亿慢性乙肝患者意义重大[^19];(2)Epigenic Therapeutics完成$6,000万B轮(中国,CRISPR表观编辑治疗乙肝)[^20]。此外,FSHD肌营养不良早期临床数据已显示病毒生物标志物清除;高胆固醇血症(PCSK9)、印记障碍(Beckwith-Wiedemann)等罕见病与慢病管线同步推进[^25]。从罕见病到慢病的路径:表观编辑先在最“可治疗”的罕见病(FSHD、印记障碍)验证安全性,再向慢病(乙肝、高胆固醇血症、代谢病)扩展——这一路径与基因编辑(Casgevy→慢病)完全一致,且表观编辑在“可逆性”上具有独特优势,是未来十年CGT版图中被低估的赛道[^25]。七·七、体细胞治疗:从肝到肺到肾——器官纤维化攻坚表9|体细胞/组织前体细胞治疗:从肝到肺到肾(2026) 细胞类型适应症代表管线/机构阶段关键进展工程化再生巨噬细胞(IL-10/MMP9) 肝硬化、终末期肝病 Resolution Therapeutics RTX001(爱丁堡大学衍生) Ph2(MATCH研究) 4年随访:治疗组死亡/移植风险30.8% vs 对照显著降低,首个晚期肝病细胞疗法;NCT06823713 肝细胞/肝前体细胞肝硬化、MASH相关肝纤维化新型肝细胞疗法(2026.5临床结果);iPSC肝细胞分化早期临床首个针对晚期肝病的细胞疗法临床试验结果积极;MASH肝纤维化成为下一目标自体肺球体干细胞(LSC) 特发性肺纤维化(IPF) NCT04262167(自体LSC静脉输注) 临床(美国) 肺纤维化缺乏有效治疗,LSC移植是“肺再生”代表方案肺泡上皮干细胞(AT2)/肺前体细胞肺纤维化、支气管扩张 UC-MSC+肺干细胞联合方案(2026 Protocol);AT2作为纤维化核心调控细胞早期临床/临床前 AT2细胞数量维持是抗纤维化关键靶点,前体细胞替代策略推进中肾自体细胞治疗(REACT) 慢性肾病(CKD) rilprencel(自体肾细胞,ProKidney) Ph3 被称为CKD“第五支柱”;自体肾小管细胞回输延缓肾功能恶化肾前体细胞注射肾衰竭、肾纤维化 Hospital Clínic de Barcelona(自体重编程肾前体细胞注入衰竭肾) 早期临床一次性注射自体肾前体细胞修复肾脏;Mayo再生肾病学同情用药同步推进体细胞/组织前体细胞治疗正在从“血液与肿瘤”走向“器官纤维化与退行”的广阔战场。全球肝、肺、肾三大器官的纤维化疾病(肝硬化、MASH、IPF、CKD)患者数以亿计,现有药物只能延缓进展,细胞治疗则尝试“修复器官”:(1)肝脏——Resolution Therapeutics的工程化再生巨噬细胞RTX001(IL-10/MMP9增强抗炎与基质降解)在MATCH Ph2研究中展示4年数据:治疗组死亡/移植风险30.8%,显著优于对照,是全球首个在晚期肝病中显示长期获益的细胞疗法[^26][^27];肝前体细胞与iPSC肝细胞同步推进,MASH相关肝纤维化成为下一个攻克目标[^22];(2)肺脏——特发性肺纤维化(IPF)目前仅有两款抗纤维化药物且无法逆转,自体肺球体干细胞(LSC)移植(NCT04262167)与肺泡上皮干细胞(AT2)替代策略正在改写“肺不可再生”的认知[^28][^29];(3)肾脏——ProKidney的rilprencel(REACT肾自体细胞治疗)已进入Ph3,被称为CKD治疗的“第五支柱”[^30];巴塞罗那Hospital Clínic则直接将自体重编程肾前体细胞注入衰竭肾脏,探索“一次性修复”[^31]。对MNC与产业的意义:器官纤维化(肝/肺/肾)患者规模远超罕见病,是CGT从“小众治愈”走向“大众慢病管理”的关键跳板——Resolution(爱丁堡)、ProKidney等“体细胞修复”公司正是这一赛道的先行者;未来十年,“前体细胞修复+基因编辑抗纤维化+表观调控”的组合方案,有望把肝硬化、IPF、CKD从“不可逆”变为“可逆转”[^29][^30]。七·八、全球自免药物市场:2025年TOP22销售$1,207亿表10|2023-2025年全球自免药物销售额TOP15(亿美元,自免市场巨大之证) # 药物公司靶点 2023 2024 2025 25同比 1Dupixent 赛诺菲/再生元 IL-4Rα 125 141177.7 +26.2% 2Skyrizi 艾伯维 IL-23 80 117175.6 +49.9% 3Ocrevus 罗氏 CD20 72 7884.7 +8.6% 4Rinvoq 艾伯维 JAK1 43 6083.0 +39.1% 5Cosentyx 诺华 IL-17A 65 6666.7 平稳 6Prolia 安进 RANKL 50 5865.0 +12.1% 7Entyvio 武田 α4β7 58 6264.1 +3.4% 8Stelara 强生 IL-12/23 104 10460.8 -41.3%(生物类似药) 9Xolair 罗氏/诺华 IgE 46 5154.4 +6.7% 10Tremfya 强生 IL-23 28 3751.6 +40.5% 11Ultomiris 阿斯利康 C5 32 4047.2 +18.0% 12Humira 艾伯维 TNF-α 90 6845.4 -49.5%(生物类似药) 13Kesimpta 诺华 CD20 25 3544.3 +26.6% 14Vyvgart argenx FcRn 12 2241.5 +88.7% 15Orencia BMS CTLA-4-Ig 36 3737.1 平稳TOP22合计(华西证券/Gangtise口径)$937亿$1,062亿$1,207亿 +13.7% 自免市场之巨大,远超想象:仅全球自免药物TOP22,2025年合计销售额即达$1,207亿(同比+13.7%)[^32][^33][^34]。单是Dupixent($177.7亿)与Skyrizi($175.6亿)两个超级重磅,就超过CGT全产品2025年销售额($75亿)的四倍——这就是“自免取代肿瘤成为CGT新主战场”的最硬逻辑:即使CGT只拿下自免市场的一小块,也是再造数个Carvykti的体量[^1][^32]。值得注意的是生物类似药冲击(Stelara -41.3%、Humira -49.5%)正在创造“老药退、新药进”的换挡窗口,CAR-T/TCE等新一代疗法的机会正在变大[^32][^35]。七·九、Top50药企自免管线图谱:SLE/SS/SSc/AIHA四病种表11|Top50药企自免管线图谱:SLE/SS/SSc/AIHA四病种(2026) 公司自免收入2025 SLE SS干燥综合征 SSc硬皮病 AIHA溶贫BMS Orencia $37.1亿 CD19 CAR-T SLE Ph2 —zola-cel SSc Ph3(全球首个) —阿斯利康 Saphnelo $6.9亿Saphnelo已上市+AZD0120(90% DORIS)+SLE Ph3 — — —诺华 Cosentyx/Kesimpta YTB323 SLE Ph2ianalumab III期成功,2026H2美国上市(首个SS创新药) — —艾伯维 Skyrizi+Rinvoq $258.6亿 ABBV-619 CD19 CAR-T SLE Ph1+CD19 ADC SLE — — —强生 Tremfya+Stelara $112.4亿 Teclistamab(BCMA/CD3 TCE)SLE IIT→Ph2 — Nipocalimab(FcRn)Ph2 —罗氏 Ocrevus+Xolair $139亿莫妥珠(CD20 TCE)SLE Ph3+RO7507062(CD19)SLE Ph1 — — —默沙东无自免重磅 CN201(CD3/CD19 TCE)SLE Ph1→Ph2 — — —安进 Prolia $65亿 blinatumomab(BiTE)SLE/LN Ph2 SS探索 — —吉利德无自免重磅 — — — CM336(BCMA/CD3)AIHA IIT→Ph2GSK Benlysta $23.2亿Benlysta已上市(SLE/LN) — — —赛诺菲 Dupixent $177.7亿 — — — —UCB Bimzelx $25.2亿 ATG-201 TCE探索 — — Cizutamig(MG Ph2)礼来无自免重磅 Kelonia/Orna体内CAR-T(B细胞自免)Ph1 — — —中国CGT公司 — 通用型CD19 CAR-T/STAR-T(SLE-LN 100% SRI-4)IND/Ph1 通用CAR-T探索通用CAR-T探索通用CAR-T(5/5 CR)IND 四病种全景(患者规模):SLE全球约820万(高收入国家75-100万)、干燥综合征SS约400-500万、硬皮病SSc约100-150万、自身免疫性溶血性贫血AIHA约10-20万——四病种均存在中重度患者未满足需求[^36]。关键格局:(1)BMS在SSc领跑(zola-cel Ph3全球首个);(2)阿斯利康在SLE布局最全(已上市Saphnelo+双靶CAR-T AZD0120);(3)诺华ianalumab即将成为首个SS创新药(2026H2美国上市);(4)罗氏(莫妥珠Ph3)、默沙东(CN201)、安进(BiTE)走TCE双抗路线;(5)吉利德CM336切入AIHA;(6)中国CGT公司的通用型CAR-T(STAR-T/CD19)在SLE-LN(100% SRI-4)、AIHA(5/5 CR)等重症适应症已展示差异化数据,成为MNC自免CGT引进的重要供给源[^36][^37]。 BD买家画像(自免维度):极度关注(默沙东-Keytruda 2028悬崖+CN201已验证TCE路线、辉瑞-后COVID真空+自免管线为零);高度关注(诺华-若YTB323遇“自体成本陷阱”则转通用型、艾伯维-免疫学霸主需临床阶段通用管线、阿斯利康-自免+肿瘤双栖、BMS-缺off-the-shelf);潜在关注(赛诺菲-Dupixent到期倒逼、强生-Carvykti延伸、安进-TCE数据未达则转向)[^37]。八、未来十年爆炸性成果预测(2026-2036) 图3|全球CGT市场规模:2024年$212.8亿→2034年$1,174.6亿(CAGR 18.6%) · 查看原文表4|未来十年爆炸性成果预测时间轴(2026-2036) 阶段技术突破预期成果标志性事件预判 2026-2028(数据兑现期) 体内CAR-T概念验证完成 NEJM 2025.10已发表首个in vivo CD19 CAR-T治疗SLE人体数据;B细胞耗竭+疾病活动度下降+无重大毒性体内CAR-T注册性试验启动;传奇LB2501、KLN-1010数据读出自免CAR-T首个获批 BMS zola-cel(SSc Ph3)有望成为全球首个自免CAR-T获批诺华YTB323 SLE关键Ph2数据(2027-2028) 体内CRISPR注册级数据 Intellia NTLA-2002(HAE,发作减少95%)Ph3 引爆新一轮in vivo基因编辑平台交易 2028-2030(注册获批期) 通用CAR-T自免获批 off-the-shelf现货供应+成本降至1/10+门诊给药 SLE/SSc/RA等大适应症;SLE市场$150亿+ 体内CAR-T注册临床礼来双路径(LNP-mRNA+慢病毒)覆盖互补技术首个体内CAR-T新药申请;门诊给药成标准实体瘤CAR-T放量 2026.6 NMPA已批准全球首个实体瘤CAR-T(CLDN18.2) CLDN18.2/GPC3/间皮素实体瘤管线全面临床后期 2030-2036(治愈时代) 体内基因编辑范式跃迁 ex vivo→in vivo,单次给药体内修复致病基因肝脏/眼/神经/心脏遗传病全覆盖 iPSC现货细胞工厂 iPSC-T/NK/心肌/多巴胺神经元全谱系帕金森(Bemdaneprocel Ph3)、黄斑变性、心衰细胞治疗神经退行性疾病突破 AAV基因治疗+细胞治疗+碱基编辑联合帕金森/亨廷顿/ALS首个疾病修饰疗法健康长寿方向表观遗传重编程、衰老细胞清除、器官再生 CGT从"治病"走向"抗衰";市场$1,174.6亿 2026-2028(数据兑现期):体内CAR-T概念验证完成(NEJM已发表首个SLE数据[^9][^10]);BMS zola-cel有望成为全球首个自免CAR-T获批;NTLA-2002若达注册级将引爆体内基因编辑交易潮[^2][^11]。 2028-2030(注册获批期):通用CAR-T自免获批、体内CAR-T注册性试验、实体瘤CAR-T放量(2026.6 NMPA已批准全球首个实体瘤CAR-T,CLDN18.2靶点)——CGT进入"门诊给药+现货供应"时代[^1][^2]。 2030-2036(治愈时代):体内基因编辑实现ex vivo→in vivo范式跃迁;全球十大药企的iPSC现货细胞工厂(帕金森Bemdaneprocel Ph3[^2]、黄斑变性、心衰)全面落地;神经退行性疾病出现首个疾病修饰疗法;表观遗传重编程与衰老细胞清除开启"健康长寿"赛道。九、对全人类疾病治疗与健康长寿的影响表5|CGT十年发展对人类疾病治疗与健康长寿的影响矩阵疾病领域全球患者规模 CGT十年后预期代表技术社会影响血液肿瘤年新发~50万(全球) CAR-T成为标准疗法,治愈率显著提升自体/异体/体内CAR-T 从"化疗时代"进入"细胞治愈时代" 自身免疫病全球3-5亿患者 CAR-T免疫重置实现长期无药缓解(非"终身用药") 自体CAR-T、通用CAR-T、体内CAR-T 自免从慢病管理→一次性重置,医疗支出革命罕见遗传病全球3亿+罕见病患者单次基因治疗/编辑治愈 AAV基因治疗、CRISPR/碱基/先导编辑罕见病从"无药可医"→"一次治愈" 神经退行性疾病帕金森~1,000万、AD~5,000万 iPSC细胞替代+基因治疗疾病修饰 iPSC多巴胺神经元、AAV、碱基编辑延缓衰老相关神经退化,健康寿命延长心血管/代谢病数亿级体内基因编辑单次降脂(LDL降62%) LNP-CRISPR/碱基编辑(PCSK9) 慢病从"终身服药"→"一次编辑" 健康长寿全人类细胞重编程/衰老细胞清除/器官再生表观遗传重编程、iPSC、基因编辑健康寿命(healthspan)延长,老龄化社会变革影响一:自免疾病从"终身用药"到"一次性重置"。全球3-5亿自免患者,目前主流是终身生物制剂(每年$2-5万)。CAR-T免疫重置若获批,将是医学史上首次"重置免疫系统"的疗法——对SLE、硬皮病、MS、RA、1型糖尿病的治疗范式是颠覆性的[^2]。影响二:罕见遗传病从"无药可医"到"一次治愈"。全球3亿+罕见病患者,AAV基因治疗+CRISPR/碱基/先导编辑将实现单次治愈——这是"一刀切掉终身治疗"的经济学革命[^1]。影响三:慢病从"终身服药"到"一次编辑"。LNP-CRISPR体内编辑PCSK9(LDL降62%)预示着心血管/代谢慢病可能被"单次注射"管理——这对全球数亿高血脂、糖尿病患者的健康管理是革命性的[^2][^11]。影响四:神经退行与健康长寿。iPSC细胞替代(帕金森)、AAV基因治疗、表观遗传重编程——CGT正从"治病"走向"抗衰",健康寿命(healthspan)延长将深刻改变老龄化社会的医疗与经济结构[^2]。影响五:经济学重构。CGT市场2034年预计$1,174.6亿(CAGR 18.6%)[^2];但更重要的是"一次性治愈"vs"终身治疗"的支付逻辑重构——每一次治愈都为社会节省数十年的治疗费用与生产力损失。十、给各方建议10.1 给投资人 (1)关注"自免CAR-T"标的:zola-cel/YTB323/AZD0120数据读出节点即估值重估点;(2)in vivo CAR-T头部标的已被MNC瓜分,关注递送平台(LNP/AAV衣壳)与体内基因编辑(Intellia类)的二级机会;(3)中国CGT资产(异体CAR-T、体内CAR-T、基因编辑)仍是全球BD的性价比供给源,许可中位首付$1.41亿存在价值修复空间;(4)用"并购潮领先融资潮18-24个月"周期规律,2027年提前布局一级市场。10.2 给CGT创业者 (1)自免是融资与BD的最热叙事,但必须绑定"门诊给药+成本1/10+可制造"的临床路径;(2)体内CAR-T拥挤,错位竞争:实体瘤、神经、代谢新适应症;(3)参考EsoBiotech/Orbital路径——临床前到I期即被MNC收购,提前构建BD叙事与数据包;(4)安全设计(自杀开关/可控CAR)从第一天嵌入——在辉大事件后,这是监管与并购尽调的共同底线。10.3 给跨国药企 (1)in vivo CAR-T版图未完成:Capstan类LNP路线仍有标的,递送平台是下一轮军备竞赛;(2)辉瑞/默沙东/赛诺菲应加速"通用/in vivo CAR-T自免"补位,错过2028-2030窗口将再等五年;(3)中国CGT资产是性价比供给源,但尽调需关注中美监管互认与安全记录;(4)支付创新前置:疗效挂钩/分期支付从获批第一天设计。10.4 给临床医生 (1)关注自免CAR-T临床试验入组(BMS zola-cel SSc、诺华YTB323 SLE、AZD0120);(2)熟悉基因治疗15年长期随访与继发肿瘤监测;(3)参与真实世界登记,为CGT商业化提供长期安全数据;(4)客观传达"一次性治愈"与"终身监测"的双重现实。10.5 给患者与家庭 (1)CGT获批加速(FDA年批10-20款),在ClinicalTrials.gov检索针对自身疾病的管线;(2)自免CAR-T若获批将改变"终身用药"命运,但以数据读出为准,警惕宣传话术;(3)咨询慈善援助、分期支付与临床试验免费用药机会;(4)保持理性:治愈性疗法伴随长期随访与未知风险,知情同意务必逐条确认。10.6 给政策制定者 (1)建立"自免CAR-T"支付与准入框架(疗效挂钩/分期/专项基金);(2)完善体内编辑疗法的肝毒性监测标准与长期随访监管(参考FDA定义更新);(3)支持供应链本土化与GMP产能建设;(4)促进中美欧监管互认,加速全球同步可及;(5)在辉大事件后,用透明与纪律重建全球对中国CGT临床数据的信任。十·五、患者视角:这轮革命对普通人意味着什么? 用“电脑重装系统”理解免疫重置:自身免疫病(红斑狼疮、硬皮病、类风湿等)就像免疫系统被“病毒程序”劫持——本来保护身体的卫士开始攻击自己的器官。传统药物像“杀毒软件”,需要天天用、用一辈子;而CAR-T免疫重置像“重装系统”——把出错的白细胞(B细胞)清掉,让新的免疫细胞重新学会“认识自己”,有望一次治疗、长期停药[^2][^4]。患者最关心的五个问题:(1)要住院多久?现有自体CAR-T需住院2-4周,体内CAR-T目标是“门诊注射即走”;(2)多少钱?目前自体CAR-T约$35-50万,未来通用/体内方案目标降至$3-15万,且中国五款CAR-T已闯关商保(自费有望降至4万/针级)[^38];(3)能彻底治好吗?目前看是“长期无药缓解”(4年随访持续缓解),能否终身治愈仍需更久随访[^4];(4)副作用大吗?需警惕细胞因子释放综合征(CRS)与感染,自免患者通常比肿瘤患者耐受更好,但仍需在有经验的医疗中心进行;(5)什么时候普通人能用上?2027-2028年自免CAR-T有望首个获批,2030年后逐步普及。给患者的建议:(1)难治性SLE/SSc/肌炎/MG患者可咨询主治医生是否有CAR-T临床试验机会(ClinicalTrials.gov检索);(2)警惕“高价自费试验”——正规临床试验免费,任何要求自筹巨款的项目都需警惕(2026年8月中国582万自费试验事件教训)[^20];(3)自免药物市场规模巨大(TOP22超$1,200亿),意味着药企有足够动力研发——患者是这场变革的最大受益者[^32]。十一、结语:从狂潮到治愈全球市值Top50药企,30+家涉足CGT,累计投入超$800亿——这不是泡沫,而是制药工业对"治愈性技术"的集体下注。2025年Carvykti近$19亿的爆发、NEJM首个体内CAR-T SLE数据、礼来$70亿的Kelonia收购、强生$33.65亿绑定Sail,共同指向一个确定的未来:CGT正在从"肿瘤末线的最后手段"变为"自免/罕见病/慢病的一线治愈方案"。未来十年,在七大MNC与第二梯队买家的推动下,我们将见证:首个自免CAR-T获批(2027-2028)、首个体内CAR-T新药申请(2028-2030)、iPSC现货细胞工厂落地(2030-2036)、以及CGT从"治病"走向"健康长寿"。对全人类,这意味着自免患者从终身用药到免疫重置、罕见病患者从无药可医到一次治愈、慢病患者从终身服药到一次编辑、老龄化社会从"延寿"到"健康延寿"。从狂潮到治愈——这一次,资本与技术终于同向而行。 📚 参考文献溯源 [1]Gangtise数据平台:《全球市值Top50药企CGT细胞基因治疗业务全景分析》(2026.7.13,用户提供) · Gangtise/各公司年报/ARM 2026 [2]Gangtise数据平台:《全球市值Top50药企CGT全景分析》(2026.7.13,含三大梯队/引进方向研判,用户提供) · Gangtise(银河/摩根士丹利/国泰海通/高盛/JPM研报) [3]BMS官方:BMS收购Orbital Therapeutics($15亿,2025.10,体内CAR-T) · BMS官方公告 [4]AbbVie官方:收购Capstan Therapeutics(最高$21亿,2025.6.30,tLNP体内CAR-T) · AbbVie官方公告 [5]Lilly官方:收购Orna Therapeutics(最高$24亿,2026.2,LNP-oRNA体内CAR-T) · Eli Lilly官方公告 [6]Fierce Biotech:吉利德$3.5亿收购Interius(体内CAR-T,2025.8) · Fierce Biotech [7]Gilead官方:收购Arcellx最大化anito-cel长期价值(2026) · Gilead官方公告 [8]Lilly官方:收购Kelonia Therapeutics(首付$32.5亿,最高$70亿,2026.4,iGPS体内CAR-T) · Eli Lilly官方公告 [9]NEJM 2025:In Vivo CD19 CAR T-Cell Therapy for Refractory SLE(全球首个体内CAR-T治疗SLE人体数据) · N Engl J Med 2025;393:1542-1544 · DOI:10.1056/NEJMc2509522 · PMID:40961420 [10]BioSpace:MagicRNA体内CAR-T治疗SLE首个临床数据发表于NEJM(2025.10) · BioSpace [11]行业预测:全球CGT市场规模2024年$212.8亿→2034年$1,174.6亿(CAGR 18.6%) · Fortune Business Insights/行业预测 [12]J&J 2026 H1业绩:Carvykti销售约$12.5亿(行业整理/J&J Q2 2026) · J&J Q2 2026业绩/行业整理 [13]BMS Q2 2026财报:Breyanzi $4.84亿、Abecma $7,200万(-17%),上调2026指引 · BMS Q2 2026投资者报告 [14]Vertex Q2 2026财报:Casgevy $7,600万(+151% YoY/+78%环比,历史新高) · Vertex官方Q2 2026财报 [15]Reuters:Vertex上调2026全年指引,Casgevy放量加速(2026.8.3) · Reuters [16]J&J官方:与Sail Biomedicines合作推进体内CAR-T与自免免疫重置,$785M首付+$25.8亿收购选择权(2026.7.29) · Johnson & Johnson官方公告 [17]Gilead Q2 2026财报:Yescarta $3.46亿(-12%)、Tecartus $7,000万(-24%),Kite合计$4.17亿(-14%) · Gilead官方Q2 2026财报 [18]Bloomberg:J&J获得以$25.8亿收购Sail Biomedicines的选择权(2026.7.29) · Bloomberg [19]Tune Therapeutics官方:TUNE-401表观沉默HBV cccDNA Ph1b/2a概念验证数据(EASL 2026) · Tune Therapeutics/EASL 2026 [20]Endpoints News:中国Epigenic Therapeutics完成$6,000万B轮推进CRISPR表观乙肝疗法(2026) · Endpoints News [21]Nature Signal Transduction:干细胞产品临床转化(含Heartseed iPSC心肌细胞心衰I/II期,2023启动) · Signal Transduction and Targeted Therapy · DOI:10.1038/s41392-026-02582-y [22]News-Medical:首个针对晚期肝病的细胞疗法临床试验结果(2026.5.25) · News-Medical [23]PMC综述:神经干细胞治疗脑卒中/颅脑损伤/脊髓损伤临床试验 · PMC(神经干细胞临床试验综述) [24]日本2026.3.6批准全球首款iPSC衍生治疗产品(胰腺胰岛细胞片,1型糖尿病) · 行业报道/日本PMDA [25]MedPath:表观编辑进入临床——首个试验数据与管线重塑基因治疗(FSHD等) · MedPath Trial News [26]Resolution Therapeutics官方:MATCH Ph2 4年随访数据,工程化再生巨噬细胞显著延长肝硬化患者无移植生存(AASLD 2025) · Resolution Therapeutics/AASLD 2025 [27]PubMed:MATCH01 Ph2长期随访,巨噬细胞治疗组死亡/移植风险30.8% vs 对照 · PubMed(MATCH LTFU) · PMID:42184827 [28]CenterWatch:NCT04262167自体肺球体干细胞(LSC)静脉输注治疗特发性肺纤维化 · ClinicalTrials.gov NCT04262167 [29]PMC综述:干细胞治疗肺纤维化——新兴希望(肺泡上皮/AT2机制) · PMC(肺纤维化干细胞治疗综述) [30]NephJC:REACT(rilprencel)肾自体细胞治疗,慢性肾病“第五支柱” · NephJC [31]Galway大学/行业报道:巴塞罗那Hospital Clínic自体重编程肾前体细胞一次性注射修复衰竭肾脏 · University of Galway/Hospital Clínic报道 [32]华西证券《自免药物深度报告》(2026.6)/Gangtise:2023-2025自免药物TOP22销售(2025合计$1,207亿,+13.7%) · 华西证券/Gangtise [33]Liang Chang:20 Global Pharma Q4 2025 Earnings(Dupixent FY2025 $17.8B,Stelara $6,078M,Ocrevus CHF 7,010M) · 20 Global Pharma Q4 2025 Earnings汇总 [34]Mordor Intelligence:AbbVie 2025 Skyrizi $17.562B+Rinvoq免疫学组合$30.406B · Mordor Intelligence [35]Forbes:Boom Times For Autoimmune Disease Therapies(2026.7.8,Skyrizi 2032年预计$33B) · Forbes [36]Gangtise《全球Top50药企CGT与自免全景分析》(2026.7.29,用户提供):SLE 820万/SS 400-500万/SSc 100-150万/AIHA 10-20万患者规模与四病种管线 · Gangtise(2026.7.29) [37]Gangtise《全球Top50药企CGT与自免全景分析》:BD买家画像(默沙东/辉瑞T0,诺华/艾伯维/AZ/BMS/礼来T1,赛诺菲/强生/安进T2) · Gangtise(2026.7.29) [38]腾讯新闻:五款百万级CAR-T闯关商保谈判,自费负担有望降至4万/针(2026.8.4) · 腾讯新闻细胞基因治疗前沿每日一篇 CGT 顶刊深度解读 · 数据源自 Nature/Cell/Science/Nat Biotechnol/ASGCT/ESGCT/AACR 内容仅供学术交流，不构成医疗建议

100 项与布瑞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	澳大利亚	Gilead Sciences Pty Ltd.	2022-09-30
复发性套细胞淋巴瘤	欧盟	Kite Pharma EU BV	2020-12-14
复发性套细胞淋巴瘤	冰岛	Kite Pharma EU BV	2020-12-14
复发性套细胞淋巴瘤	列支敦士登	Kite Pharma EU BV	2020-12-14
复发性套细胞淋巴瘤	挪威	Kite Pharma EU BV	2020-12-14
难治性套细胞淋巴瘤	欧盟	Kite Pharma EU BV	2020-12-14
难治性套细胞淋巴瘤	冰岛	Kite Pharma EU BV	2020-12-14
难治性套细胞淋巴瘤	列支敦士登	Kite Pharma EU BV	2020-12-14
难治性套细胞淋巴瘤	挪威	Kite Pharma EU BV	2020-12-14
急性淋巴细胞白血病	美国	Kite Pharma, Inc.	2020-07-24
套细胞淋巴瘤	美国	Kite Pharma, Inc.	2020-07-24

未上市

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适应症	最高研发状态	国家/地区	公司	日期
伯基特淋巴瘤	临床2期	美国	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	奥地利	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	法国	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	德国	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	意大利	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	荷兰	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	西班牙	Kite Pharma, Inc.	2022-11-01
伯基特淋巴瘤	临床2期	瑞士	Kite Pharma, Inc.	2022-11-01
毛细胞白血病	临床2期	美国	Kite Pharma, Inc.	2022-11-01
毛细胞白血病	临床2期	奥地利	Kite Pharma, Inc.	2022-11-01

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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


ASGCT2026	N/A	B细胞白血病 \| B细胞淋巴瘤 \| 多发性骨髓瘤 ... 更多	469	Idecabtagene vicleucel (Abecma)	遞積鏇築獵鑰憲糧醖蓋(築範糧鏇窪鬱築構構繭) = 蓋壓網築鏇憲憲夢鏇憲願鏇積糧醖襯壓鑰襯艱 (艱鏇餘鹽糧蓋鏇鑰積鹹 ) 更多	积极	2026-05-13
				Lisocabtagene maraleucel (Breyanzi)	遞積鏇築獵鑰憲糧醖蓋(淵廠衊獵壓積築衊蓋繭) = 獵簾築糧觸繭範顧鏇積鏇襯鏇簾廠鬱選鏇網繭 (壓繭範夢遞鹹蓋齋廠遞 )
EHA2026	N/A	套细胞淋巴瘤	28	Brexucabtagene autoleucel (brexu-cel)	膚壓窪壓齋構構築網餘(鹽鹽製鑰觸餘觸窪繭蓋) = 顧鏇繭鏇願築艱鏇獵襯選簾窪簾築壓壓獵醖齋 (壓鬱齋簾膚選窪積齋觸 ) 更多	积极	2026-05-12
EHA2026	N/A	急性淋巴细胞白血病	27	Brexucabtagene autoleucel (brexu-cel)	鏇膚願遞齋淵鏇餘壓壓(鹽獵鑰鑰製壓膚醖願窪) = 艱鏇夢膚窪襯窪鬱鹹壓憲願鏇廠鹽衊網選鬱觸 (憲鹽廠糧壓遞餘壓鹹鑰 ) 更多	积极	2026-05-12
EBMT2026	N/A	大B细胞淋巴瘤 \| 套细胞淋巴瘤 \| 滤泡性淋巴瘤 ... 更多	198	Axi-cel	憲餘鹽鏇繭壓範顧蓋蓋(網艱遞築鏇糧網蓋簾願) = 窪遞齋艱廠鏇鹽範網觸簾鏇鬱壓範觸觸窪鏇鏇 (鹹膚窪築餘鹽醖簾簾糧 ) 更多	积极	2026-03-22
				Brexu-cel	簾獵膚選廠窪獵鬱夢選(鏇簾築襯網顧艱願選遞) = 憲蓋構壓獵簾範醖鏇網鏇製遞壓選積繭積糧憲 (遞襯夢積襯網憲醖願壓 )
NCT05537766 (ZUMA-25, CTgov)	临床2期	毛细胞白血病 \| 伯基特淋巴瘤 \| 华氏巨球蛋白血症难治 ... 更多	19	Fludarabine+Cyclophosphamide+Brexucabtagene Autoleucel (Substudy A (Relapsed/Refractory Waldenstrom Macroglobulinemia): Brexucabtagene Autoleucel)	積夢糧糧鏇簾齋鏇顧遞 = 鹽網繭醖遞積鑰壓鹽簾獵遞構獵鑰衊淵窪壓範 (願觸鑰蓋簾鹹蓋繭蓋鑰, 獵齋鏇衊願鏇淵觸蓋範 ~ 鬱觸選繭構夢蓋壓繭鏇)	-	2026-03-10
				Fludarabine+Cyclophosphamide+Brexucabtagene Autoleucel (Substudy B (Relapsed/Refractory Richter Transformation): Brexucabtagene Autoleucel)	鬱膚夢窪鹹淵醖蓋鑰選(鏇膚蓋鬱憲膚觸構積鬱) = 鹽鏇糧構網構衊廠願憲艱製鹹醖鹹襯網齋製鬱 (艱憲蓋繭蓋鏇鑰鬱鹹製, 築遞餘鹽齋醖憲鏇衊膚 ~ 淵夢廠廠鹹鬱築夢壓醖) 更多
Tandem Meetings ASTCT and CIBMTR2026	N/A	难治性 B 细胞急性淋巴细胞白血病	8	Brexucabtagene Autoleucel (Brexu-cel)	蓋淵鹹顧廠網範鏇積蓋(構夢鹹鏇淵網構願廠鏇) = 鏇繭製齋獵鑰遞衊顧衊廠艱襯艱壓遞觸憲構壓 (齋遞襯餘淵鬱衊窪觸顧 ) 更多	积极	2026-02-04
Tandem Meetings ASTCT and CIBMTR2026	N/A	B细胞淋巴瘤	235	Axicabtagene ciloleucel (Yescarta)	衊廠醖蓋齋齋淵淵膚構(鹹積廠襯淵鬱構積鬱繭) = 衊糧蓋鹽遞鑰範夢夢製築築鬱選糧顧齋醖夢廠 (艱鏇選鏇遞範夢衊繭築, 14.2 ~ 14.61) 更多	积极	2026-02-04
				Brexucabtagene autoleucel (Tecartus)	衊廠醖蓋齋齋淵淵膚構(鹹積廠襯淵鬱構積鬱繭) = 積鏇廠醖選鏇窪廠憲夢築築鬱選糧顧齋醖夢廠 (艱鏇選鏇遞範夢衊繭築, 14.79 ~ 16.46) 更多
Tandem Meetings ASTCT and CIBMTR2026	N/A	血液肿瘤	10	CAR-T	糧觸鹽艱獵構夢鹹願衊(夢鹹蓋衊獵鑰積製襯顧) = 衊壓構鬱鹹簾鏇鏇顧憲鑰獵壓壓壓醖壓壓衊鹽 (糧鹽網鬱遞壓遞襯衊淵 ) 更多	积极	2026-02-04
NCT03642626 (MT2017-45, CTgov)	临床2期	费城染色体阳性急性淋巴细胞白血病 \| 大B细胞淋巴瘤 \| 急性淋巴细胞白血病 ... 更多	150	YESCARTA+Fludarabine+Cyclophosphamide (ARM B: Yescarta for Refractory Diffuse Large B Cell Lymphoma (DLBCL))	願鏇積餘顧蓋窪憲構網 = 製衊糧夢選築廠夢鹹網願衊窪壓願蓋艱簾廠遞 (範鹹築淵艱衊壓衊鹽製, 窪蓋範顧獵餘鹽壓壓齋 ~ 憲構鹹艱膚築鹽膚膚艱) 更多	-	2026-01-23
				Fludarabine+Cyclophosphamide+KYMRIAH (ARM C: Kymriah for Refractory Diffuse Large B Cell Lymphoma (DLBCL))	願鏇積餘顧蓋窪憲構網 = 壓觸範簾憲鏇襯製醖醖願衊窪壓願蓋艱簾廠遞 (範鹹築淵艱衊壓衊鹽製, 淵齋網糧憲糧獵齋獵糧 ~ 淵積繭淵膚鹽鬱鏇繭構) 更多
ChiCTR2300073872 (ASH2025) 人工标引	临床2期	前体B细胞成淋巴细胞白血病淋巴瘤	28	KTE-X19	鑰廠衊鑰壓蓋獵製鑰襯(遞齋構淵齋鑰製膚壓遞) = 鹽繭願範壓淵糧遞廠築壓範簾顧淵遞衊壓遞觸 (夢範壓憲鏇壓糧範齋鹹, 62.0 ~ 90.2) 更多	积极	2025-12-06