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更新于：2025-12-05

Descartes-08

更新于：2025-12-05

概要

概要

基本信息

药物类型

自体CAR-T、mRNA CAR-T

别名

Anti-BCMA CAR-T cell therapy、Anti-BCMA chimeric antigen receptor T cell therapy、autologous T-cells expressing a chimeric antigen receptor directed against B-Cell maturation antigen

+ [2]

靶点

BCMA

作用方式

抑制剂

作用机制

BCMA抑制剂(B细胞成熟蛋白抑制剂)

治疗领域

肿瘤免疫系统疾病神经系统疾病+ [3]

在研适应症

重症肌无力抗中性粒细胞胞质抗体相关性血管炎皮肌炎

非在研适应症

多发性骨髓瘤系统性红斑狼疮

原研机构

Cartesian Therapeutics, Inc.

在研机构

Cartesian Therapeutics, Inc.

非在研机构-

权益机构-

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)-

特殊审评孤儿药 (美国)、罕见儿科疾病 (美国)、再生医学先进疗法 (美国)

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关联

项与 Descartes-08 相关的临床试验

NCT07089121

/ Not yet recruiting临床1/2期

Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis

Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis

开始日期2025-12-01

申办/合作机构

Cartesian Therapeutics, Inc.

NCT06799247

/ Recruiting临床3期

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients With Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis

The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study will last around 6 months. For eligible participants, Part 2 will last around 8 months.

开始日期2025-05-06

申办/合作机构

Cartesian Therapeutics, Inc.

NCT06038474

/ Active, not recruiting临床2期

Descartes-08 for Patients With Systemic Lupus Erythematosus

This is a Phase II study to evaluate the safety, tolerability and manufacturing feasibility of Descartes-08 CAR T-cells in patients with Systematic Lupus erythematosus (SLE).

开始日期2024-02-12

申办/合作机构

Cartesian Therapeutics, Inc.

100 项与 Descartes-08 相关的临床结果

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100 项与 Descartes-08 相关的转化医学

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100 项与 Descartes-08 相关的专利（医药）

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项与 Descartes-08 相关的文献（医药）

2023-07-01·The Lancet. Neurology

Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study

Article

作者: Miljković, Miloš D ; Slansky, Adam D. ; Pereira, Denise ; Chowdhury, Adam ; Brooks, Beverly Mackanzie ; Mozaffar, Tahseen ; Tosun, Mehmet ; Steele, Julie ; Habib, Ali Aamer ; Kurtoglu, Metin ; Singer, Michael S ; Jewell, Christopher M. ; Farias, Jerrica ; Duvernay, Matthew T. ; DeMaria, Gabrielle ; Requena, Naraly ; English, Emily ; Chahin, Nizar ; Benatar, Michael ; Vu, Tuan ; Howard, James F ; Feinberg, Marc H ; Suresh, Niraja ; Kamboh, Hafsa ; Komanduri, Krishna ; Benjamin, Cara ; Slansky, Adam ; Kireeva, Maria ; Jewell, Christopher M ; Sahagian, Gregory ; Granit, Volkan ; Dimitrova, Diana ; Howard, James F. ; Holley, Kelly ; Daniel, Shaji ; Kalayoglu, Murat V ; Ngo Casi, Minhtran ; De La Cruz, Luis ; Tenorio, April ; Fong, Julia Kimberly ; Chopra, Manisha ; Shan, Yufei ; Stewart, C Andrew ; Feinberg, Marc H. ; Paredes, Maria Elena

BACKGROUND:

Chimeric antigen receptor (CAR) T cells are highly effective in treating haematological malignancies, but associated toxicities and the need for lymphodepletion limit their use in people with autoimmune disease. To explore the use of CAR T cells for the treatment of people with autoimmune disease, and to improve their safety, we engineered them with RNA (rCAR-T)-rather than the conventional DNA approach-to target B-cell maturation antigen (BCMA) expressed on plasma cells. To test the suitability of our approach, we used rCAR-T to treat individuals with myasthenia gravis, a prototypical autoantibody disease mediated partly by pathogenic plasma cells.

METHODS:

MG-001 was a prospective, multicentre, open-label, phase 1b/2a study of Descartes-08, an autologous anti-BCMA rCAR-T therapy, in adults (ie, aged ≥18 years) with generalised myasthenia gravis and a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 6 or higher. The study was done at eight sites (ie, academic medical centres or community neurology clinics) in the USA. Lymphodepletion chemotherapy was not used. In part 1 (phase 1b), participants with Myasthenia Gravis Foundation of America (MGFA) disease class III-IV generalised myasthenia gravis received three ascending doses of Descartes-08 to determine a maximum tolerated dose. In part 2 (phase 2a), participants with generalised myasthenia gravis with MGFA disease class II-IV received six doses at the maximum tolerated dose in an outpatient setting. The primary objective was to establish safety and tolerability of Descartes-08; secondary objectives were to assess myasthenia gravis disease severity and biomarkers in participants who received Descartes-08. This trial is registered with clinicaltrials.gov, NCT04146051.

FINDINGS:

We recruited 16 individuals for screening between Jan 7, 2020 and Aug 3, 2022. 14 participants were enrolled (n=3 in part 1, n=11 in part 2). Ten participants were women and four were men. Two individuals did not qualify due to low baseline MG-ADL score (n=1) or lack of generalised disease (n=1). Median follow-up in part 2 was 5 months (range 3-9 months). There was no dose-limiting toxicity, cytokine release syndrome, or neurotoxicity. Common adverse events were headache (six of 14 participants), nausea (five of 14), vomiting (three of 14), and fever (four of 14), which resolved within 24 h of infusion. Fevers were not associated with increased markers of cytokine release syndrome (IL-6, IL-2, and TNF). Mean improvements from baseline to week 12 were -6 (95% CI -9 to -3) for MG-ADL score, -7 (-11 to -3) for Quantitative Myasthenia Gravis score, -14 (-19 to -9) for Myasthenia Gravis Composite score, and -9 (-15 to -3) for Myasthenia Gravis Quality of Life 15-revised score.

INTERPRETATION:

In this first study of an rCAR-T therapy in individuals with an autoimmune disease, Descartes-08 appeared to be safe and was well tolerated. Descartes-08 infusions were followed by clinically meaningful decreases on myasthenia gravis severity scales at up to 9 months of follow-up. rCAR-T therapy warrants further investigation as a potential new treatment approach for individuals with myasthenia gravis and other autoimmune diseases.

FUNDING:

Cartesian Therapeutics and National Institute of Neurological Disorders and Stroke of the National Institutes of Health.

2023-01-19·Blood

Efficacy and safety of cilta-cel in patients with progressive multiple myeloma after exposure to other BCMA-targeting agents

Article

作者: Vogel, Martin ; Roccia, Tito ; Nesheiwat, Tonia ; De Braganca, Kevin C. ; Cohen, Adam D. ; Varsos, Helen ; Suvannasankha, Attaya ; Akram, Muhammad ; Paiva, Bruno ; Cohen, Yael C. ; Deraedt, William ; Corsale, Christina ; van de Donk, Niels W. C. J. ; Pacaud, Lida ; Zudaire, Enrique ; Martin, Thomas ; San-Miguel, Jesus ; Avivi, Irit ; Xu, Xiaoying ; Rodriguez-Otero, Paula ; Mistry, Pankaj ; Schecter, Jordan M. ; Mateos, María-Victoria ; Wang, Liwei

Abstract:

B-cell maturation antigen (BCMA)–targeting therapies, including bispecific antibodies (BsAbs) and antibody-drug conjugates (ADCs), are promising treatments for multiple myeloma (MM), but disease may progress after their use. CARTITUDE-2 is a phase 2, multicohort study evaluating the safety and efficacy of cilta-cel, an anti-BCMA chimeric antigen receptor T therapy, in various myeloma patient populations. Patients in cohort C progressed despite treatment with a proteasome inhibitor, immunomodulatory drug, anti-CD38 antibody, and noncellular anti-BCMA immunotherapy. A single cilta-cel infusion was given after lymphodepletion. The primary end point was minimal residual disease (MRD) negativity at 10−5. Overall, 20 patients were treated (13 ADC exposed; 7 BsAb exposed; 1 in the ADC group also had prior BsAb exposure). Sixteen (80%) were refractory to prior anti-BCMA therapy. At a median follow-up of 11.3 months (range, 0.6-16.0), 7 of 20 (35%) patients were MRD negative (7 of 10 [70.0%] in the MRD-evaluable subset). Overall response rate (95% confidence interval [CI]) was 60.0% (36.1-80.9). Median duration of response and progression-free survival (95% CI) were 11.5 (7.9—not estimable) and 9.1 (1.5—not estimable) months, respectively. The most common adverse events were hematologic. Cytokine release syndrome occurred in 12 (60%) patients (all grade 1-2); 4 had immune effector cell-associated neurotoxicity syndrome (2 had grade 3-4); none had parkinsonism. Seven (35%) patients died (3 of progressive disease, 4 of adverse events [1 treatment related, 3 unrelated]). Cilta-cel induced favorable responses in patients with relapsed/refractory MM and prior exposure to anti-BCMA treatment who had exhausted other therapies. This trial was registered at www.clinicaltrials.gov as NCT04133636.

2023-01-01·Frontiers in pharmacology

Effectiveness and safety of anti-BCMA chimeric antigen receptor T-cell treatment in relapsed/refractory multiple myeloma: a comprehensive review and meta-analysis of prospective clinical trials.

Review

作者: Nie, Xiaoyan ; Chen, Liming ; Yan, Diqin ; Chen, Min ; Dong, Wenliang ; Wang, Simin ; Zhang, Jiaojiao ; Hu, Dingyuan ; Niu, Suping ; Fang, Yi

Background: Chimeric antigen receptor T cells treatment targeting B cell maturation antigen (BCMA) is an emerging treatment option for relapsed/refractory multiple myeloma (RRMM) and has demonstrated outstanding outcomes in clinical studies. Objective: The aim of this comprehensive review and meta-analysis was to summarize the effectiveness and safety of anti-BCMA CAR-T treatment for patients with relapsed/refractory multiple myeloma (RRMM). Our research identifies variables influencing outcome measures to provide additional evidence for CAR-T product updates, clinical trial design, and clinical treatment guidance. Methods: The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) standard was followed for conducting this comprehensive review and meta-analysis, which was submitted to PROSPERO (CRD42023390037). From the inception of the study until 10 September 2022, PubMed, Web of Science, EMBASE, the Cochrane Library, CNKI, and WanFang databases were searched for eligible studies. Stata software (version 16.0) was used to assess effectiveness and safety outcomes. Results: Out of 875 papers, we found 21 relevant trials with 761 patients diagnosed as RRMM and were given anti-BCMA CAR-T treatment. The overall response rate (ORR) for the entire sample was 87% (95% CI: 80-93%) complete response rate (CRR) was 44% (95% CI: 34-54%). The minimal residual disease (MRD) negativity rate within responders was 78% (95% CI: 65-89%). The combined incidence of cytokine release syndrome was 82% (95% CI: 72-91%) and neurotoxicity was 10% (95% CI: 5%-17%). The median progression-free survival (PFS) was 8.77 months (95% CI: 7.48-10.06), the median overall survival (OS) was 18.87 months (95% CI: 17.20-20.54) and the median duration of response (DOR) was 10.32 months (95% CI: 9.34-11.31). Conclusion: According to this meta-analysis, RRMM patients who received anti-BCMA CAR-T treatment have demonstrated both effectiveness and safety. Subgroup analysis confirmed the anticipated inter-study heterogeneity and pinpointed potential factors contributing to safety and efficacy, which may help with the development of CAR-T cell studies and lead to optimized BCMA CAR-T-cell products. Systematic Review Registration: Clinicaltrials.gov, PROSPERO, CRD42023390037.

116

项与 Descartes-08 相关的新闻（医药）

2025-11-26

·空之客

【药海听涛】CAR-T和TCE决战紫禁之巅：B细胞清除药物临床数据全景图

近些年来，自身免疫疾病的热度毋庸置疑，其中B细胞清除机制更是烈火烹油。如果说2024年是TCE之年，2025年则是invivo CAR-T之年，MNC动辄豪掷数以十亿计的真金白银，誓要守护未来自免治疗的旗帜。记得去年笔者在基金内部讨论这个领域时，经常产生的疑惑在于“为什么CAR-T被Schett验证之后大家要一窝蜂去买TCE”；到了今年，这个疑惑演变成了“为什么一窝蜂买完了TCE又要回头去一窝蜂搞invivo CAR-T”……面对这种挠头的局面，笔者作为外行又不可能给出机制孰优孰劣的说辞，只能化简就繁，从各家能拿出的临床数据入手，看看CAR-T和TCE决战紫禁之巅到底打出个什么结果了？（须做说明的是，目前B细胞清除机制的这些药物几乎全都在早期临床、甚至IIT，跨试验对比数据至多只有示意性意义；此外，虽然有少数管线尝试了多种自身免疫疾病，但本文只重点选取了针对交集最多的SLE适应症结果进行对比分析。） 1 自体CAR-T 上帝说CAR-T能治自身免疫疾病，于是便有了George Schett大神。在George教授几乎是以一己之力奠定B细胞清除机制在自免的天胡开局之后，三四年时间过去了，向身后看去似乎依然一骑绝尘，跟进的CD19 CAR-T们非但难以重现炸裂的有效性、甚至连细胞治疗惯有的CRS和ICANS风险都难以回避，只能寄希望于几家中国公司的双靶点CAR-T排列组合。 1.1 Dr. Schett：MB-CART19.1（CD19 CAR-T）作为开山祖师爷，Schett教授主导的CASTLE系列试验，无疑给全行业立下了“开局即巅峰”的丰碑。在10例SLE患者中有9例达到Doris缓解，B细胞清除中位时间达到120天，CAR-T峰值中位数达到120 cells/uL，所有患者均实现免疫抑制剂停用，且没有三级以上CRS或任何ICANS。目前工业界的CAR-T或其他机制，都还没有真正超越这样漂亮的临床结果。 1.2 Cabaletta：CABA-201（CD19 CAR-T） Cabaletta应该是最早正经用CAR-T冲击自免疾病的Biotech，一口气推了Myositis、SLE、SSc、gMG、MS、PV等一系列适应症。在初步公布的SLE结果中，4例接受治疗半年以上患者中有3例实现Doris缓解，B细胞清除中位时间超过50天，此外在SSc试验中还验证了淋巴结内深度的B细胞清除；所有患者中，CRS仅有1级，但有1例LN患者发生了4级ICANS和4级全血细胞减少。 1.3 药明巨诺：Relma-cel（CD19 CAR-T）作为国产CAR-T的先驱者之一，药明巨诺在自免方向的尝试较早。在最初8例患者中实现了5例Doris缓解，B细胞清除维持至少5周时间，CAR-T峰值中位数93 cells/uL、CAR基因峰值中位数6.5E4 copies/ugDNA；目前所有CRS均为1级，但有1例患者出现2级ICANS。 1.4 亘喜/AZ：GC-012F（CD19xBCMA FasT CAR-T）亘喜是最早被MNC整体收购的细胞治疗公司（甚至一度也是整个中国Biotech被收购的标杆），AZ买了自然就是看重冲击自免的预期。在IIT的10例患者中有7例实现Doris缓解，B细胞清除中位时间84天，CAR基因峰值2E4 copies/ugDNA；CRS全部为1/2级，没有ICANS，不过3级以上血液毒性发生率够高的。考虑到该试验较重的基线，应该说药效是比较亮眼的，不过这次ACR与半年前EULAR好像也没多少新东西，年初就启动的大手笔150例注册临床才更值得期待。 1.5 西比曼：C-CAR-168（CD20xBCMA CAR-T）西比曼是另一家早早就抱上J&J大腿的中国公司，不过这个双靶CAR-T似乎并不在合作范围内。目前已治疗超过半年的4例患者中有2例实现Doris缓解，B细胞清除时间约2月，CAR基因峰值约1E5 copies/ugDNA；CRS均为1/2级，没有ICANS。 1.6 Autolus：Obe-cel（CD20xBCMA CAR-T） Obe-cel作为已获批血液瘤的品种也继续向自免延伸，在治疗半年以上的6例患者中有5例实现Doris缓解，B细胞清除中位时间居然高达6月，CAR基因峰值9E4 copies/ugDNA；CRS全部为1级，没有ICANS。这应该是目前注册临床中最接近Schett的数据。 1.7 雅科生物：CD19xBCMA CAR-T 这个似乎连代号都不容易查到的管线，不声不响摸出了一篇Nat Med。15例患者中有12例实现了Doris缓解，B细胞清除时间约2月、特别是有3例患者进行了PBMC和BMMC的测序证实了深度的B细胞清除，CAR基因峰值100-500 copies/uL（分别对应CD19和BCMA）；所有CRS均为1级，没有ICANS。不过雅科似乎近年在一堆子刊上发了不少文章，注册临床的声音却仙踪难觅。 2 异体CAR-T 犹记得小甜甜invivo CAR-T现身之前，牛夫人UCAR-T才是下一代细胞治疗的希望之星，资本在寒冬中又兴趣寥寥，让这个方向的发展大打折扣。即使近期涌现出北恒和邦耀等亮眼的中国IIT数据，但UCAR-T原本最重要的成本优势，在invivo面前似乎可能遭遇降维打击，好像临床表现显得依旧意兴阑珊。 2.1 北恒生物：CTA-313（CD19xBCMA UCAR-T）北恒毫无争议是今年ACR上最靓的仔，数据漂亮不说，而且展现形式从容大方，全不似一个小公司的IIT。在14例患者中有6例实现Doris缓解、不过可惜的是高剂量组只有1/4反而偏低，B细胞清除中位时间超过6周、且重建后naive细胞占绝大多数，CAR基因峰值1E6 copies/ugDNA；仅5例发生CRS且全部为1级，高剂量组反而完全没有CRS。 2.2 邦耀生物：BRL-303（CD19 UCAR-T）虽然只有4例数据，邦耀也可圈可点，毕竟4例全都Doris缓解实属不易，B细胞清除也达到1月，CAR峰值1E4 copies/ugDNA、CAR-T峰值75 cells/uL；CRS只有1级，没有ICANS。 2.3 Fate：FT-819（iPSC来源CD19 UCAR-T）邪修版本的Fate，基本上算是废特了。试图减少清淋、甚至不清淋，然而无论是SLEDAI-2K评分、还是B细胞清除，都并不理想。 2.4 Cartesian：Decartes-08（mRNA转染BCMA UCAR-T）另一大邪修Cartesian在笛卡尔的名号加持下，居然还算有模有样。在相对较轻的基线背景下，治疗超过3月的3例患者中有2例实现Doris缓解，也没有3级以上不良反应。 3 体内CAR-T 火归火，invivo CAR-T玩家们普遍刚进入IIT，真正能拿出来临床数据者寥寥无几。而在为数不多的这些数据中，似乎带来的更多不是星辰大海的信心、而是满脸懵逼的困惑…… 3.1 虹信生物：HN-2301（tLNP CD19 invivo CAR-T）我当然理解早期探索的珍贵，但面对这篇顶着中国invivo CAR-T领跑者之名高居NEJM上的文章，依然表达十分的不李姐：论转染效率，不同患者之间异质性太大不稳定；论B细胞清除，也完全谈不上干净；论SLEDAI-2K评分降低，不说Doris缓解吧，基本是聊胜于无，而且是在多个患者基线就已经很低的情况下……只能说我们应该向前瞻的研究者和伟大的受试者们致敬。 3.2 EsoBiotec：ESO-T01（LVV BCMA invivo CAR-T） Eso是最早发表IIT结果（虽然是治疗MM）的invivo CAR-T公司之一，据称也因此率先被AZ纳入麾下。不过先驱一定带着更多的疑点，例如不同患者之间针对T细胞亚群转染效率的巨大异质性，CAR-T数量峰值偏低，以及瘆人的3级CRS和ICANS，特别是输注后的高热、低血压血氧、以至意识模糊（还记得会上听梅恒主任讲到几个医生被迫4小时轮班盯守的惊心动魄），都还值得持续关注。 4 TCE 去年全球各大药企们一窝蜂往TCE上砸钱，豪掷近百亿美元几乎到了“应买尽买”的程度，然而转眼一年多过去，好像连个水漂都没见着，尤其是被下了重注的那几个分子，既无IIT结果公布、又无自免的注册临床进展。至于已经浮出水面的这些临床结果，与一众CAR-T相比还是略显单薄，至少与当初MNC的热情似乎极不相称。 4.1 天劢源和：A-319（CD19xCD3双抗）天劢源和的IIT数据，在TCE里算是最亮眼：治疗九个月以上的10例患者中有4例实现Doris缓解，B细胞清除至少6周（虽然给药就持续了4周）；没有3级以上CRS或ICANS，但有1例因不良反应而脱落。它的问题不在于数据不好，而在于这样的数据早在去年ACR就有雏形（6例），且注册临床迟迟不见下文，生生把优等生搁在半路上了。 4.2 惠和生物：CC-312（CD19xCD28xCD3三抗）增加CD28第二信号无疑是个勇敢的举措，然而惠和这个分子似乎并没有达到设计的初衷。低剂量5ug组B细胞清除都不干净，高剂量10ug组也做不到SLEDAI-2K归零，只能说是有药效、但不多。 4.3 Roche：Mosunetuzumab（CD20xCD3双抗）罗氏旗下已经获批的Mosunetuzumab，在自免的早期尝试也并不算成功。priming dose剂量几乎没什么效果自不必说，15/45mg两个目标剂量虽然看到一定的B细胞清除，但爬到60mg似乎清除得反而不干净了，SLEDAI-2K评分的下降也只是聊胜于无。 5 其他抛开CAR-T与TCE之争，也有超出三界外、不在五行中的玩家。 5.1 宜明昂科：IMM-0306（CD47xCD20双抗） CD47“钉子户”宜明昂科如今也算找到了出口，B细胞清除持续12周相当亮眼，但15例患者中无一实现Doris缓解还是有点无奈。 5.2 恩瑞恺诺：KN-5501（CD19 UCAR-NK）最开始看到恩瑞恺诺这个临床结果是非常惊艳的，18例患者中有13例实现Doris缓解，B细胞清除时间1月；只有1级CRS，没有ICANS。然而，经大佬提醒，似乎作为一个通用细胞治疗，它需要的donor数量有点略多了，加之恩瑞恺诺的IND拖了快两年才于近期获批，多少会引发一些猜想。完整数据以及所有看过的文献都放在知识星球了，看官大佬们欢迎支持取用~

细胞疗法免疫疗法ASH会议

2025-11-15

·医麦客

针对儿童！红斑狼疮细胞疗法新突破，CAR-NK 与 CAR-T 双线领跑

免费早鸟票，200 张限量领取 2025 年 11 月 16 日医麦客新闻 eMedClub News 近日，2025 年美国肾脏病学年会（ASN）上，恩瑞恺诺公布了其自主研发的 CD19/BCMA CAR-NK 细胞疗法 KN5601 的最新临床研究数据。该疗法针对儿童复发/难治性系统性红斑狼疮（SLE），临床数据初步展现出「疗效与安全双优」的特征：受试者 SLEDAI 和 PGA 评分显著改善，蛋白尿水平降低或维持稳定，自身抗体滴度下降，且治疗期间未观察到治疗相关严重不良事件。根据官方新闻稿，KN5601 采用「通用化」设计，即通过 HTAS-RV™递送系统将 CAR 基因导入健康供体 NK 细胞，结合 NK 细胞扩增冻存技术，实现「即用型」生产。这意味着，它可以突破传统疗法需要从患者自身提取细胞、个体化制备的限制，能够快速响应临床需求。尤其是对于儿童患者中意义重大，儿童免疫系统发育不成熟，自体细胞采集难度高，通用型产品可避免等待期疾病进展风险。这一进展并非孤例。就在近期，国家药监局（NMPA）刚刚批准精准生物的「普基奥仑赛注射液」上市，这是国内首款治疗儿童白血病的 CAR-T 产品。从儿童白血病到儿童红斑狼疮，这些突破也揭示了一个重要趋势：细胞与基因治疗（CGT）领域，尤其是细胞疗法，正在向着儿童领域拓展，适应症也在向着更广泛的疾病领域延伸，尤其在 SLE 等自免疾病中，细胞疗法正成为重要研究方向之一。 SLE 细胞疗法管线进展 SLE 是一种典型的系统性自身免疫性疾病，主要表现为全身多系统多脏器受累、体内存在大量自身抗体等特征。如不及时治疗，可能导致肾脏、心血管及神经系统损伤等严重并发症。根据美国红斑狼疮基金会的数据，全球 SLE 患者数至少为 500 万，其中中国红斑狼疮患者约百万，患病率居世界第二。由于 SLE 病理机制较为复杂，目前临床尚无彻底的根治方法。传统治疗主要糖皮质激素和免疫抑制剂等，往往存在疗效有限、副作用大等问题。近年来，随着对 SLE 发病机制研究的深入，尤其是 B 细胞在自身免疫反应中的关键作用被逐步阐明，针对 B 细胞的靶向治疗成为研究热点。细胞治疗作为一种新兴治疗策略，通过直接靶向致病性 B 细胞或调节免疫平衡，为 SLE 治疗提供新思路。推荐阅读：GSK、罗氏等纷纷押注 SLE 蓝海，细胞疗法与 TCE 双赛道竞速 CAR-NK CAR-NK 疗法兼具靶向性和 NK 细胞的天然安全性（低 CRS 风险、无 GVHD），在 SLE 中的应用多数处于早期探索阶段。国内外布局这一领域的企业包括 Nkarta、Century、恩瑞恺诺、先博生物、荣谷生物等。国际层面，Nkarta 的同种异体 CD19 CAR-NK 细胞 NKX019 于 2023 年获 FDA 批准治疗狼疮性肾炎的 IND 申请，目前正处于 1 期临床阶段。该产品被设计通过表达膜结合型 IL-15 增强细胞持久性。除了针对狼疮性肾炎外，NKX019 还有一项针对系统性硬化症、ANCA 相关性血管炎和特发性炎性肌病的临床研究也正在进行中。此外，2025 年 5 月 21 日，Nkarta 还开展了一项 NKX019 治疗重症肌无力（MG）临床试验的招募。国内方面，先博生物、恩瑞恺诺相关管线均已进入临床阶段且已有早期临床数据。 SNC103 是先博生物研发的国内首个获批 IND 治疗 SLE 的通用型 CAR-NK 疗法。官方新闻稿指出，临床前研究中，其 CAR 结构展示出比同类 CAR 设计更优效的活性。2025 年 6 月 26 日，先博生物公布的临床研究结果显示，SNC103 在治疗复发/难治性 SLE 病人中实现快速深度缓解，并展示出卓越的安全性。具体而言，入组的 5 多线治疗无效患者（平均患病超 9 年且伴有多器官受损），100% 在 1 个月达到 SRI-4 应答，其中 1 例随访时间超 12 个月仍维持应答；60% 的患者在 3 个月内达到 DORIS 缓解；全部患者经治后实现激素停用或减量，未出现 CRS、ICANS、GVHD 等不良反应。 KN5501 是恩瑞恺诺的另一款 CAR-NK 细胞治疗药物，与文章开头提到的 KN5601 一样，采用了独创的 HTAS-RV 递送系统和细胞扩增冻存技术。 2025 年 9 月亚太风湿病学大会（APLAR）上，KN5501 治疗 SLE 的 2 年最新随访结果显示，20 例患者的 SRI-4 应答率 95% 提示患者疾病活动度显著改善；LLDAS 应答率 70%；Doris 缓解率 60%；达到 Doris 缓解的受试者，最长随访时间超过 24 个月，仍保持缓解未复发。所有受试者的 SLEDAI 和 PGA 评分显著持续改善，无一例受试者复发。 CAR-T 相较于 CAR-NK，CAR-T 疗法在 SLE 领域的探索更早、布局更为广泛，已从自体走向通用型，从 CD19 单靶点扩展至多靶点。据不完全统计，目前已有超 20 条管线处于不同的临床阶段，来自国外 Novartis、BMS 等头部，国内百暨基因、西比曼、邦耀生物、药明巨诺、精准生物、瑞顺生物等诸多企业。其中，进展比较快的已经处于 2 期临床阶段，如 Cartesian 的 Descartes-08，这是一款基于 mRNA 技术的 BCMA CAR-T，已经在去年 7 月完成了针对系统性红斑狼疮患者的 2 期临床试验首次给药。西比曼生物的 CD20/BCMA 双靶点 CAR-T 细胞疗法 C-CAR168 治疗自免疾病的早期临床数据也显示，达到 6 个月随访的 4 位狼疮性肾炎患者全部实现 SRI-4 应答标准。其中，2 人达到完全肾脏缓解（CR）标准，1 人达到了部分 CR。今年 4 月，一篇题为「Safety and clinical efficacy of Relmacabtagene autoleucel (relma-cel) for systemic lupus erythematosus: a phase 1 open-label clinical trial」的文章报道了瑞基奥仑赛治疗中重度活动性 SLE 患者的一项 IIT 研究初步结果。研究结果显示，所有患者经治后临床症状均显著改善。6 个月时，患者 SELENA-SLEDAI 平均评分从基线时的 11.75 分降至 1.625 分；PGA 平均评分从基线时的 1.82 分降至 0.52 分；BILAG 总分从基线时的 6.50 分降至 0.88 分。在 8 例患者中，4 例在 1-4 个月内达到 SLE 缓解定义（DORIS）的临床缓解标准，7 例在 1-4 个月内达到狼疮低疾病活动状态（LLDAS）标准，且整体安全性良好。结语 SLE 作为一种复杂的系统性自身免疫性疾病，长期以来一直缺乏有效的根治手段。随着科学技术的不断进步，SLE 治疗领域正迎来前所未有的发展机遇，细胞疗法等创新疗法在 SLE 治疗中展现出巨大的潜力和广阔的应用前景。尽管细胞疗法为 SLE 治疗带来历史性突破，但也仍面临治疗成本、生产工艺复杂等诸多挑战。未来，通用型细胞疗法将可能成为重点发展方向，联合治疗策略、靶点优化及生产工艺改进等也将进一步推动这一领域向前发展。责任编辑丨玄枵校对丨玄枵参考资料： 1.NEJM 365;22 december 1, 2011 2.各企业官网官微 3.公开资料精彩直播预告长按识别二维码立即参与↓ 11 月 19 日 19:00-20:30 ADC 研发前沿工具：基于细胞的高通量筛选技术推荐 2 11 月 20 日 15:00-16:00 外泌体作为新型递送系统在 RNAi 药物领域的技术应用声明：本文旨在于传递行业发展信息、探究生物医药前沿进展。文章内容仅代表作者观点，并不代表医麦客立场，亦不构成任何价值判断、投资建议或医疗指导，如有需求请咨询专业人士投资或前往正规医院就诊。版权说明：欢迎个人转发文章至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。如需转载请在文章下方留言获取授权。封面来源网络，如有侵犯版权，请联系删除点点“分享”、“点赞”和“在看”，给我充点儿电吧~

细胞疗法免疫疗法孤儿药基因疗法临床研究

2025-11-14

·EndPoints

Despite positive data, Cartesian to pause work in form of lupus in favor of other indications

Cartesian Therapeutics said it will focus development of its lead cell therapy candidate on patients with myasthenia gravis and myositis, and will pause work in another immune condition known as systemic lupus erythematosus. That’s despite reporting positive data in a small trial of the lupus patients. The therapy, called Descartes-08, led to remission in two of the three people. Systemic lupus erythematosus, or SLE, affects more than 1.5 million people in the US and can be deadly, inflaming and damaging organs. It currently has no cure, according to Cartesian. Cartesian said it will instead put its resources toward myositis, which can also be deadly and can lead to weak muscles, pain, heart issues and challenges breathing and swallowing. The company said that its data led it to believe that Descartes-08 could have “strong mechanistic alignment” for treating the disease. It hopes to begin a pivotal trial by the middle of next year. It has an ongoing Phase 3 trial in myasthenia gravis. “While we saw very encouraging data and we agree that significant unmet need remains in SLE, we feel myositis represents a more attractive area of development,” Cartesian said in an emailed response to a question from Endpoints News . “Myositis has significant unmet [need] with very limited therapeutic options currently available.” In addition to pausing Descartes-08 in SLE, it said it will also halt work on Descartes-15, an autologous anti-BCMA CAR-T that it called a next-generation version of its treatment. Like Descartes-08, it was designed not to need preconditioning with chemotherapy. Descartes-15 had just completed a three-patient, Phase 1 safety trial with positive results. The pullback will extend the company’s cash through mid-2027, it said, and will let it complete the Phase 3 myasthenia gravis trial and start the Phase 2 myositis trial for Descartes-08. Last year, the biotech raised $130 million through a private placement that was meant to help expand its work. Editor’s note: This story has been updated with a comment from the the company.

细胞疗法临床3期临床2期临床1期免疫疗法

100 项与 Descartes-08 相关的药物交易

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研发状态

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
重症肌无力	临床3期	美国	Cartesian Therapeutics, Inc.	2025-05-06
重症肌无力	临床3期	塞尔维亚	Cartesian Therapeutics, Inc.	2025-05-06
重症肌无力	临床3期	西班牙	Cartesian Therapeutics, Inc.	2025-05-06
重症肌无力	临床3期	土耳其	Cartesian Therapeutics, Inc.	2025-05-06
抗中性粒细胞胞质抗体相关性血管炎	临床2期	-	Cartesian Therapeutics, Inc.	2025-12-01
皮肌炎	临床2期	-	Cartesian Therapeutics, Inc.	2025-12-01
系统性红斑狼疮	临床2期	美国	Cartesian Therapeutics, Inc.	2024-02-12
多发性骨髓瘤	临床2期	美国	Cartesian Therapeutics, Inc.	2021-12-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04146051 (Company_Website) 人工标引	临床2期	重症肌无力	36	Descartes-08	獵鑰獵築齋壓淵簾繭衊(壓築積製壓築膚鹹醖積) = 顧簾壓獵鹹鑰願齋願鏇鑰範積積願遞壓醖觸襯 (壓艱選積築鹽範選夢餘, 1.1) 更多	积极	2025-04-08
				Descartes-08 (without prior biologic therapies)	獵鑰獵築齋壓淵簾繭衊(壓築積製壓築膚鹹醖積) = 淵蓋繭蓋積齋繭鹹憲遞鑰範積積願遞壓醖觸襯 (壓艱選積築鹽範選夢餘, 1.5) 更多
NCT04146051 (MG-001, Neurology \| AAN2025) 人工标引	临床2期	重症肌无力	31	Descartes-08	窪繭範夢築廠網鑰齋積(窪積範鑰鹽選艱醖鹹憲) = 構築襯觸築鏇觸製鑰壓鹹蓋簾簾窪壓齋製構襯 (憲襯願鏇鬱築艱淵窪夢 ) 更多	积极	2025-04-07
				Placebo	窪繭範夢築廠網鑰齋積(窪積範鑰鹽選艱醖鹹憲) = 選鹽範艱獵襯廠糧廠廠鹹蓋簾簾窪壓齋製構襯 (憲襯願鏇鬱築艱淵窪夢 ) 更多
NCT04146051 (Company_Website) 人工标引	临床2期	重症肌无力	36	Descartes-08	窪艱壓遞遞選憲範繭壓(願簾壓鏇築廠鹹鑰糧範) = 築窪構鏇壓鹹獵獵蓋憲鏇蓋艱遞觸膚網顧淵繭 (鏇齋鏇積獵顧鹹鏇餘艱 ) 更多	积极	2024-12-03
				Descartes-08 (without prior exposure to biologic therapies)	窪艱壓遞遞選憲範繭壓(願簾壓鏇築廠鹹鑰糧範) = 網衊齋願襯壓選襯選顧鏇蓋艱遞觸膚網顧淵繭 (鏇齋鏇積獵顧鹹鏇餘艱 ) 更多
NCT04816526 (CTgov)	临床2期	多发性骨髓瘤	13	Descartes 08	繭餘範鏇廠選襯鬱衊衊 = 製選鹹繭獵壓積築憲鬱網憲艱範餘範襯淵製鹽 (鹽鑰餘鏇夢範觸獵鏇餘, 襯築積壓窪觸獵鑰廠鬱 ~ 糧餘獵獵醖觸糧獵繭築) 更多	-	2024-10-08
NCT04146051 (Biospace) 人工标引	临床2期	重症肌无力	36	Descartes-08	鹽憲膚鹽鬱衊淵壓壓鹹(鏇夢夢廠襯範鏇襯襯醖) = 觸選膚醖繭觸顧淵願獵構艱築積壓觸範膚膚艱 (廠選積齋艱鹹鬱淵願窪 ) 达到更多	积极	2024-07-02
				Placebo	鹽憲膚鹽鬱衊淵壓壓鹹(鏇夢夢廠襯範鏇襯襯醖) = 網築鹽鑰築鹽憲獵範繭構艱築積壓觸範膚膚艱 (廠選積齋艱鹹鬱淵願窪 ) 达到更多
Corporate Publications 人工标引	临床2期	重症肌无力	7	Descartes-08	糧選構積蓋鹽選襯醖積(鏇願鹹鹹鹹鏇鬱觸鹹鹽) = 蓋衊範鬱糧繭製壓顧憲窪鑰憲蓋積蓋鏇繭蓋衊 (觸壓鬱顧構鑰繭願遞窪 ) 更多	积极	2024-01-08
NCT04146051 (MG-001, Pubmed 1 \| Pubmed 2 \| Lancet Neurol) 人工标引	临床1/2期	重症肌无力	14	Descartes-08	顧積蓋餘壓艱範蓋齋衊(選蓋夢獵鏇壓淵鹽獵壓) = 顧鏇襯積觸遞鏇範憲繭築簾膚壓顧築鏇繭鬱醖 (膚憲製糧夢製廠憲觸鹽 ) 更多	积极	2023-07-01
NCT04146051 (Corporate Publications) 人工标引	临床1/2期	重症肌无力	2	Descartes 08	廠選廠餘簾齋鏇憲淵範(窪築膚壓壓鹹網範鹽鏇) = over 50% three months after Descartes-08 administration. 鹽齋衊網願選遞壓淵艱 (糧獵窪鏇壓顧齋糧憲醖 )	积极	2022-05-10