预约演示
更新于：2026-08-01
Descartes-08
更新于：2026-08-01
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 |
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) |
非在研机构- |
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
7
项与 Descartes-08 相关的临床试验NCT07391605
A Randomized Double-Blind Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of Descartes-08 in Patients With Dermatomyositis and Antisynthetase Syndrome
NCT07089121
Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis
NCT06799247
A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients With Generalized Myasthenia Gravis (MG)
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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9
项与 Descartes-08 相关的文献（医药）2026-03-01NATURE MEDICINE
BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial
Article
作者: Slanksy, Adam ; Gwathmey, Kelly ; Siddiqi, Zaeem ; Stewart, C Andrew ; Miljkovic, Milos D ; Jewell, Christopher M ; Karam, Chafic ; Kalayoglu, Murat ; Fedak, Renee R ; Durmus, Hacer ; Sahagian, Gregory ; Shroff, Sheetal ; Kamboh, Hafsa ; Singer, Michael ; Howard, James F ; Badruddoja, Michael A ; Vu, Tuan ; Barnett-Tapia, Carolina ; Pasnoor, Mamatha ; Peltier, Amanda ; Vullaganti, Mithila ; Mozaffar, Tahseen ; Rivner, Michael ; Ragole, Thomas ; Kurtoglu, Metin ; Ruggerie, Rachel N ; Feinberg, Marc H ; Small, George ; Myers, Bennett ; Dimachkie, Mazen
Myasthenia gravis (MG) is driven by the secretion of autoantibodies from pathogenic B cell maturation antigen (BCMA)-expressing plasma cells. In this phase 2b randomized, controlled, double-blind trial, we evaluated Descartes-08, an autologous BCMA-directed mRNA chimeric antigen receptor T cell therapy, in patients with generalized MG (gMG). Patients (n = 26) were randomly allocated to receive once-weekly intravenous infusions of Descartes-08 (n = 15) or placebo (n = 11) over 6 weeks. The primary endpoint was a ≥5-point improvement in the MG Composite (MGC) score at month 3. Secondary endpoints included the mean change from baseline in MGC, MG Activities of Daily Living (MG-ADL) and Quantitative MG (QMG) scores by month 12. At month 3, the proportion of patients achieving a ≥5-point improvement in the MGC score was significantly higher for those treated with Descartes-08 compared to placebo in the overall population (66.7% (n = 10/15) versus 27.3% (n = 3/11), P = 0.0472) and in a subpopulation of those positive for autoantibodies to the acetylcholine receptor (63.6% (n = 7/11) versus 12.5% (n = 1/8), P = 0.0258). For patients treated with Descartes-08, the changes from baseline in mean MGC, MG-ADL and QMG scores at month 4 were -7.1, -5.5 and -4.8, respectively, with 83.0% of patients achieving a sustained and clinically meaningful response at month 12. Notably, 33.0% of patients achieved minimum symptom expression (MSE) (MG-ADL score ≤1) by month 6, which was sustained through month 12. Among biologic-naive patients, 55.60% achieved MSE by month 6, which was maintained through month 12 without additional treatment. Descartes-08 was generally safe and well tolerated. Infusion-related reactions were the most common adverse events reported (Descartes-08, 80.0% (n = 16/20); placebo, 56.3% (n = 9/16)). In summary, a single course of six once-weekly infusions of Descartes-08 was well tolerated and resulted in sustained clinically meaningful responses among patients with gMG. ClinicalTrials.gov identifier: NCT04146051 .
2026-03-01NATURE MEDICINE
BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial
Article
作者: Yu, David J. L. ; Peltier, Amanda ; Mozaffar, Tahseen ; Bodansky, Aaron ; Vullaganti, Mithila ; DeMaria, Gabrielle ; Fedak, Renee R. ; Myers, Bennett ; Kalayoglu, Murat V. ; Garcia, Samantha T. ; Siddiqi, Zaeem ; Curvino, Elizabeth J. ; Filev, Atanas ; Karafin, Matthew ; Rivner, Michael ; Zorn, Kelsey C. ; Mendoza, Meg ; Shaw, Jessica ; Biaksangi, Jennifer ; Gwathmey, Kelly ; Xu, Heng ; Durmuş, Hacer ; Miljković, Miloš D. ; Ragole, Thomas ; Sahagian, Gregory ; Cheng, Catherine ; Pasnoor, Mamatha ; Benson, Albina ; Badruddoja, Michael A. ; Requena, Naraly ; Ruggerie, Rachel N. ; Small, George ; Singer, Michael S. ; Park, Yara A. ; Quesnell, Tara ; Jewell, Christopher M. ; Karam, Chafic ; Shan, Yufei ; Trunnell, Amy ; Brunn, Carsten ; Vu, Tuan ; English, Adam ; Barnett-Tapia, Carolina ; Davis, Darnell ; Shroff, Sheetal ; Anderson, Mark S. ; Ngo-Casi, Minhtran ; Duvernay, Matthew T. ; Chopra, Manisha ; Pope, Chad A. ; Feinberg, Marc H. ; Habib, Ali A. ; Zhang, Yi ; DeRisi, Joseph L. ; Buensuceso, Charito ; Schneider, Dina ; Kamboh, Hafsa ; Tostanoski, Lisa H. ; de Sousa, Juliana F. ; Howard, James F. ; Traub, Rebecca E. ; Kurtoglu, Metin ; Irannejad, Mona ; Daniel, Shaji ; English, Emily P. ; Grover, Natalie S. ; Holley, Kelly ; Stewart, C. Andrew ; Slanksy, Adam
Chimeric antigen receptor (CAR)-T cell therapies have the potential to transform treatment of autoimmune disease by resetting the immune system. However, adoption of cell therapies in the autoimmune space is limited by hurdles such as inpatient administration, lymphodepletion and safety concerns around cytokine release syndrome and non-specific immunosuppression. RNA-based cell therapy has potential to address these limitations. Here we report prespecified exploratory analyses from a successful placebo-controlled, double-blind, randomized phase 2b trial in patients with generalized myasthenia gravis who received Descartes-08, an autologous, RNA-encoded anti-B cell maturation antigen (BCMA) CAR-T cell therapy. In 66.7% of patients (n = 10/15), transient targeting of BCMA with Descartes-08 administered in an outpatient setting without lymphodepletion resulted in durable clinical efficacy. Comparison of Descartes-08-treated (n ≤ 19) and placebo (n ≤ 15) cohorts by flow cytometry, serum profiling, multiplexing cytokine analysis and bulk/single-cell transcriptional analysis reveals a precision retuning of self-reactivity demonstrated by increased pro-immune function, decreased activity of BCMA+ plasma cells and plasmacytoid dendritic cells and reductions in disease-associated cytokines, such as IL-6. Furthermore, antibody and T cell receptor analysis revealed altered circulating repertoires of self-reactive antibodies and T cell clones among Descartes-08 participants. These effects occurred without immune suppression, indicated by the lack of decline in vaccine-specific antibodies or hypogammaglobulinemia. Our findings unveil a new type of immune reset and support the development of BCMA-targeted RNA cell therapies as a more accessible therapy for autoimmune diseases. ClinicalTrials.gov identifier: NCT04146051 .
2023-07-01The 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: Miloš D Miljković ; Adam Chowdhury ; Christopher M. Jewell ; Marc H Feinberg ; Beverly Mackanzie Brooks ; Yufei Shan ; Matthew T. Duvernay ; Naraly Requena ; Metin Kurtoglu ; Kelly Holley ; Christopher M Jewell ; Maria Kireeva ; Ali Aamer Habib ; Adam D. Slansky ; Mehmet Tosun ; Tuan Vu ; Manisha Chopra ; Michael S Singer ; Marc H. Feinberg ; April Tenorio ; Gabrielle DeMaria ; James F Howard ; Denise Pereira ; Diana Dimitrova ; James F. Howard ; Michael Benatar ; Maria Elena Paredes ; Krishna Komanduri ; C Andrew Stewart ; Jerrica Farias ; Nizar Chahin ; Murat V Kalayoglu ; Julie Steele ; Tahseen Mozaffar ; Cara Benjamin ; Hafsa Kamboh ; Gregory Sahagian ; Minhtran Ngo Casi ; Emily English ; Volkan Granit ; Shaji Daniel ; Adam Slansky ; Luis De La Cruz ; Julia Kimberly Fong ; Niraja Suresh
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.
273
项与 Descartes-08 相关的新闻（医药）2026-07-31
·今日头条
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, 后查看更多信息
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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 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 加拿大 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 意大利 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 波兰 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 塞尔维亚 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 西班牙 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 土耳其 | 2025-05-06 | |
| 重症肌无力 | 临床3期 | 英国 | 2025-05-06 | |
| 抗合成酶综合征 | 临床2期 | 美国 | 2026-04-28 | |
| 皮肌炎 | 临床2期 | 美国 | 2026-04-28 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 重症肌无力 BCMA | 30 | 壓製獵壓窪膚願膚餘構(積衊鹽鑰選壓築艱餘淵) = 壓鹹鹹願鑰顧憲選齋齋 範餘構鑰壓築簾鏇獵膚 (齋築遞憲糧醖構簾繭製 ) | 积极 | 2026-01-09 | ||
临床2期 | 36 | 廠範構選衊廠壓獵製餘(蓋製觸糧繭鑰鏇窪醖艱) = 繭膚顧製獵艱鑰衊齋襯 繭醖窪鹹齋獵獵鏇醖艱 (鏇顧繭廠網衊艱膚淵範, 1.1) 更多 | 积极 | 2025-04-08 | |||
(without prior biologic therapies) | 廠範構選衊廠壓獵製餘(蓋製觸糧繭鑰鏇窪醖艱) = 鹹遞構鬱製簾製憲夢鹽 繭醖窪鹹齋獵獵鏇醖艱 (鏇顧繭廠網衊艱膚淵範, 1.5) 更多 | ||||||
临床2期 | 31 | 鏇顧構構積選窪廠鬱餘(獵網蓋膚廠遞夢鏇壓獵) = 積積願網鏇鹽網襯艱繭 糧壓糧鹹鏇鹽齋範夢獵 (夢鹹範構廠製糧製鹽鬱 ) 更多 | 积极 | 2025-04-07 | |||
Placebo | 鏇顧構構積選窪廠鬱餘(獵網蓋膚廠遞夢鏇壓獵) = 築醖憲淵憲顧齋簾選廠 糧壓糧鹹鏇鹽齋範夢獵 (夢鹹範構廠製糧製鹽鬱 ) 更多 | ||||||
临床2期 | 36 | 襯淵糧願繭築網蓋糧餘(淵糧膚鹹築鏇糧糧鬱遞) = 簾遞艱廠衊衊簾選鏇築 糧觸衊艱鬱觸構觸廠範 (壓襯築鑰選蓋積製艱鬱 ) 更多 | 积极 | 2024-12-03 | |||
(without prior exposure to biologic therapies) | 襯淵糧願繭築網蓋糧餘(淵糧膚鹹築鏇糧糧鬱遞) = 願襯願衊夢構選襯窪簾 糧觸衊艱鬱觸構觸廠範 (壓襯築鑰選蓋積製艱鬱 ) 更多 | ||||||
临床2期 | 13 | 網窪憲遞齋繭窪鹽醖鏇 = 鏇窪獵襯壓窪願窪夢夢 鑰鬱獵廠製衊壓製蓋觸 (積醖構蓋構範顧繭糧襯, 膚範壓蓋蓋蓋觸觸網鹽 ~ 餘遞範齋製淵廠窪鹹膚) 更多 | - | 2024-10-08 | |||
临床2期 | 36 | 齋遞餘壓構醖鏇憲觸願(蓋廠窪窪鏇鏇築窪鬱鏇) = 襯膚簾衊窪繭鏇獵獵構 蓋窪顧壓鬱繭製艱網願 (獵鏇選蓋構廠繭鹽構鹹 ) 达到 更多 | 积极 | 2024-07-02 | |||
Placebo | 齋遞餘壓構醖鏇憲觸願(蓋廠窪窪鏇鏇築窪鬱鏇) = 觸獵積廠夢製廠築壓鑰 蓋窪顧壓鬱繭製艱網願 (獵鏇選蓋構廠繭鹽構鹹 ) 达到 更多 | ||||||
临床2期 | 7 | 遞淵齋繭壓積網憲艱製(膚齋範淵鏇鹽積鹽鏇壓) = 蓋構糧廠願衊餘糧襯艱 鏇鑰廠繭築蓋鹹蓋衊構 (鬱遞憲蓋積願範築鬱選 ) 更多 | 积极 | 2024-01-08 | |||
临床1/2期 | 14 | 範願鑰選簾簾獵壓鏇選(鹹選積築淵壓簾艱鹽艱) = 鹹鬱餘壓衊壓簾構鏇觸 糧繭餘選膚遞繭遞窪醖 (鹽艱鏇鹽憲築衊選鹽糧 ) 更多 | 积极 | 2023-07-01 | |||
临床1/2期 | 2 | 觸觸願衊艱齋鹹獵獵淵(遞糧醖鏇齋憲夢構醖餘) = over 50% three months after Descartes-08 administration. 窪築網範遞壓鬱鑰蓋簾 (齋衊夢蓋積鏇襯廠繭餘 ) | 积极 | 2022-05-10 |
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