RESET-MS: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Participants With Multiple Sclerosis

RESET-MS: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Participants with Multiple Sclerosis

开始日期2026-06-01

申办/合作机构

Cabaletta Bio, Inc.

NCT06359041

/ Active, not recruiting临床1/2期

RESET-MG: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Participants With Generalized Myasthenia Gravis

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis

开始日期2024-12-17

申办/合作机构

Cabaletta Bio, Inc.

NCT06328777

/ Recruiting临床1/2期

A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Systemic Sclerosis

RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis

开始日期2024-07-02

申办/合作机构

Cabaletta Bio, Inc.

100 项与 Resecabtagene Autoleucel 相关的临床结果

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100 项与 Resecabtagene Autoleucel 相关的转化医学

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100 项与 Resecabtagene Autoleucel 相关的专利（医药）

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项与 Resecabtagene Autoleucel 相关的文献（医药）

2024-11-01·MOLECULAR THERAPY

Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial

Article

作者: Miller, Claire ; Williams, Jazmean ; Little, Courtney ; Stadanlick, Jason ; Hadi-Nezhad, Fatemeh ; Furmanak, Thomas ; Thompson, Daniel ; Cicarelli, Justin ; Lam, Quynh ; Werner, Mallorie ; Ellis, Alexandra ; Schmitt, Chris ; Nunez, Daniel ; Basu, Samik ; Mozaffar, Tahseen ; Volkov, Jenell ; Chang, David ; Vorndran, Zachary

Under compassionate use, chimeric antigen receptor (CAR) T cells have elicited durable remissions in patients with refractory idiopathic inflammatory myopathies (IIMs). Here, we report on the safety, efficacy, and correlative data of the first subject with the immune-mediated necrotizing myopathy (IMNM) subtype of IIM who received a fully human, 4-1BBz anti-CD19-CAR T cell therapy (CABA-201) in the RESET-Myositis phase I/II trial (NCT06154252). CABA-201 was well-tolerated following infusion. Notably, no evidence of cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome was observed. Creatine kinase levels decreased, and muscular strength improved post-infusion. Peripheral B cells were depleted rapidly following infusion, and the subject achieved peripheral B cell aplasia by day 15 post-infusion. Peripheral B cells returned at 2 months post-infusion and were almost entirely transitional. Autoantibodies to SRP-9, SRP-72, SRP-54, and Ro-52, decreased relative to baseline, whereas antibodies associated with pathogens and vaccinations remained stable. The infusion product consisted of predominantly CD4⁺ effector memory T cells and exhibited in vitro cytolytic activity. Post-infusion, CABA-201 expansion peaked at day 15 and was preceded by a serum IFN-γ peak on day 8 with peaks in serum IL-12p40 and IP-10 on day 15. These data detail the safety, efficacy, and pharmacodynamics of CABA-201 in the first IMNM subject.

2024-06-01·Molecular Therapy-Methods & Clinical Development

Preclinical specificity & activity of a fully human 41BB-expressing anti-CD19 CART- therapy for treatment-resistant autoimmune disease

Article

作者: Alvarado, Andrea ; Stratton, Victoria ; Willis, Jonathan ; Guarneri, Soprina ; Patel, Darshil ; Lee, Jinmin ; Cassim, Hangameh ; Martynchuk, Ashley ; Wong, Steven ; Chen, Chien-Chung ; SantaMaria, Julia ; Dryer-Minnerly, Rebecca ; Basu, Samik ; Binder, Gwendolyn K ; Li, Yan ; Peng, Binghao J

Over 4% of the global population is estimated to live with autoimmune disease, necessitating immunosuppressive treatment that is often chronic, not curative, and carries associated risks. B cells have emerged as key players in disease pathogenesis, as evidenced by partial responsiveness to B cell depletion by antibody-based therapies. However, these treatments often have transient effects due to incomplete depletion of tissue-resident B cells. Chimeric antigen receptor (CAR) T cells targeting B cells have demonstrated efficacy in refractory systemic lupus erythematosus. To this end, we developed an anti-CD19 CAR T cell product candidate, CABA-201, containing a clinically evaluated fully human CD19 binder (IC78) with a 4-1BB costimulatory domain and CD3 zeta stimulation domain for treatment refractory autoimmune disease. Here, we demonstrate specific cytotoxic activity of CABA-201 against CD19⁺ Nalm6 cells with no off-target effects on primary human cells. Novel examination of CABA-201 generated from primary T cells from multiple patients with autoimmune disease displayed robust CAR surface expression and effective elimination of the intended target autologous CD19⁺ B cells in vitro. Together, these findings support the tolerability and activity of CABA-201 for clinical development in patients with autoimmune disease.

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项与 Resecabtagene Autoleucel 相关的新闻（医药）

2026-04-16

·allsci.com

University Hospital Erlangen achieves CD19 CAR-T remission in autoimmune hemolytic anemia with ITP

Researchers at University Hospital Erlangen in Erlangen, Germany report that a single infusion of CD19 CAR-T therapy in autoimmune hemolytic anemia produced sustained remission in a patient with overlapping refractory AIHA, immune thrombocytopenia, and antiphospholipid syndrome, according to a study published April 10, 2026 in Med. The patient had failed nine prior treatment lines, including three courses of rituximab. The case involved a 47-year-old woman with concurrent cold- and warm-agglutinin autoimmune hemolytic anemia, ITP, and antiphospholipid antibody syndrome — a combination of autoantibody-driven disorders sharing a common pathogenic mechanism in dysregulated B cells. Prior therapies including steroids, intravenous immunoglobulin, azathioprine, bortezomib, and repeated rituximab had each failed to produce durable control. The treating team at Erlangen administered zorpocabtagene-autoleucel (Zorpo-cel; MB-CART19.1), an autologous 4-1BB-costimulated CD19-directed CAR-T cell product manufactured on the Miltenyi Prodigy platform, at a dose of 1 × 10⁶ CAR-positive T cells per kilogram following lymphodepletion with fludarabine and cyclophosphamide. Hematologic recovery was rapid. The patient achieved transfusion independence by day 7 and hemoglobin normalization above 12 g/dL by day 25. Hemolysis markers including LDH and haptoglobin normalized, and reticulocyte counts stabilized. Warm-reactive autoantibodies became undetectable, cold agglutinin titers fell, and antiphospholipid antibodies declined. Platelet counts improved without additional ITP-directed therapy. These responses were maintained across the 11-month follow-up period. The direct antiglobulin test showed conversion, and CAR-T cells demonstrated in vivo expansion detectable in peripheral blood following infusion. At approximately day 322, reconstituted B cells were 98% naïve phenotype, indicating that the reconstituting immune compartment lacked the autoreactive memory imprinting present before treatment. The safety profile was consistent with CAR-T cell therapy autoimmune hemolytic anemia experience in oncology settings. The patient developed Grade 1–2 cytokine release syndrome managed with tocilizumab and supportive care. No immune effector cell-associated neurotoxicity syndrome was observed. Hypogammaglobulinemia, an expected consequence of B cell depletion, was managed with immunoglobulin replacement. No treatment-related mortality or life-threatening infections occurred. The biological rationale for CD19-directed CAR-T cells in this setting centers on the shared dependence of AIHA, ITP, and antiphospholipid syndrome on autoreactive B cells producing pathogenic autoantibodies. CD19 is expressed across the B cell lineage, including on subsets and in tissue compartments where the anti-CD20 monoclonal antibody rituximab achieves incomplete penetration. The Erlangen authors note that CAR-T cells achieved remission despite three prior rituximab failures, consistent with the hypothesis that deeper and more anatomically complete B cell depletion — including in lymph nodes and mucosal compartments — underlies the more durable response. The 4-1BB costimulatory domain incorporated into Zorpo-cel is associated with enhanced CAR-T cell persistence and memory differentiation, which may contribute to the duration of response observed. The authors describe the qualitative change in the reconstituted B cell compartment as a key mechanistic finding. The near-complete naïve phenotype of returning B cells at day 322 suggests that pathogenic memory clones were eliminated and that immune reconstitution proceeded from progenitors without the autoimmune programming that had driven disease. The authors characterize this as a B cell reset rather than simple depletion, distinguishing the approach from conventional immunosuppression, which does not eliminate autoreactive memory. The Erlangen group has previously reported CD19 CAR-T outcomes in systemic lupus erythematosus, inflammatory myositis, and systemic sclerosis, establishing the broader concept of CAR-T-mediated immune reset in connective tissue diseases. The current report extends that work to autoimmune cytopenias — a distinct disease category where dedicated CAR-T data remain limited. Refractory AIHA treatment options after rituximab failure are constrained. Fostamatinib, a spleen tyrosine kinase inhibitor, carries regulatory approval for warm AIHA but does not address the underlying autoantibody mechanism. Sutimlimab targets complement component C1s and is approved specifically for cold agglutinin disease but does not eliminate pathogenic B cells. Thrombopoietin receptor agonists for ITP and anticoagulation for antiphospholipid syndrome address downstream consequences without modifying the autoimmune source. In the commercial CAR-T space, Kyverna Therapeutics is advancing KYV-101, a CD19-directed autologous CAR-T product, in Phase II trials across multiple autoimmune indications including multiple sclerosis, myasthenia gravis, and systemic sclerosis. Cabaletta Bio is running Phase I/II RESET trials with CABA-201 in inflammatory myositis and lupus. Neither program is specifically focused on autoimmune cytopenias, and the current report from Erlangen represents one of the few published accounts of antiphospholipid syndrome CAR-T treatment alongside AIHA and ITP in a single patient. Prof. Dr. Fabian Müller of University Hospital Erlangen served as corresponding author. The work was funded by the Deutsche Forschungsgemeinschaft, Deutsche Krebshilfe, the Bundesministerium für Bildung und Forschung, the Bayerisches Zentrum für Krebsforschung, the Interdisciplinary Center for Clinical Research Erlangen, and the Staedtler Foundation. Miltenyi Biotechnology provided the manufacturing platform and research support to authors at the institution. Meta description: Erlangen researchers report CD19 CAR-T remission in refractory autoimmune hemolytic anemia with concurrent ITP and antiphospholipid syndrome. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

2026-04-15

·BioSpace

First Patients Dosed with Cabaletta Bio’s Rese-cel Manufactured on Cellares’ Automated Cell Shuttle™ Platform

First two GMP doses of rese-cel manufactured on the Cell Shuttle™ met all release specifications, were delivered on time, and have been infused into patients within Cabaletta Bio's RESET™ clinical trial program. SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Cellares, the first Integrated Development and Manufacturing Organization (IDMO), today announced that Cabaletta Bio Inc.’s (“Cabaletta Bio” or “Cabaletta”) (Nasdaq: CABA) investigational CAR T cell therapy rese-cel (resecabtagene autoleucel) has been infused into the first two patients using drug product manufactured on Cellares' Cell Shuttle ™ automated manufacturing platform. The administration of an autologous cell therapy, which met all release criteria and was manufactured on an automated manufacturing platform, represents an important step on the journey to realizing a future where scalable manufacturing of autologous products to supply thousands of patients per year can be achieved with minimal capital investment and a low cost of goods. While the transformative clinical benefits of autologous CAR T cell therapy are well established in oncology, the high manufacturing costs, lack of scalability, process inconsistency, and operational inflexibility associated with the current highly manual way of manufacturing have created meaningful barriers to patient access, reducing patient accessibility to these therapies. As autologous CAR T products continue to demonstrate compelling clinical impact on patients with autoimmune diseases, the challenges associated with achieving unprecedented scale have been magnified. The Cell Shuttle and Cell Q systems offer the potential of highly scalable production capacity that can be secured by biotech and pharmaceutical companies alike with minimal capital investment and a low cost of goods to address large market opportunities. In 2023, Cabaletta recognized the potential of Cellares' fully automated, industrialized approach to fundamentally address the cost and scale challenges associated with autologous CAR T manufacturing. Over the past three years, the two companies collaborated closely to adapt the rese-cel manufacturing process to the Cell Shuttle and to generate comparability data across multiple process development and engineering runs, benchmarking the automated manufacturing process against Cabaletta’s established process. The resulting dataset supported the submission and subsequent FDA clearance of an Investigational New Drug amendment to use the Cell Shuttle platform for clinical manufacturing of rese-cel. "This is an important milestone that reflects focused collaboration between the teams at Cabaletta and Cellares since 2023," said Steven Nichtberger, M.D., Co-founder, Chairman, and Chief Executive Officer of Cabaletta Bio. "The dosing of these first two patients is an important demonstration of Cellares’ GMP manufacturing and supply chain capabilities with their automated manufacturing platform and thus represents a significant achievement towards our goal of securing high-capacity flexible supply with minimal capital investment and a low cost of goods." "This milestone is a transformative moment for the field of autologous cell therapy," said Fabian Gerlinghaus, Co-founder and Chief Executive Officer of Cellares. "For years, the promise of autologous CAR T has been constrained by manufacturing models that were never designed to scale. The successful dosing of patients with rese-cel manufactured on the Cell Shuttle demonstrates that automation can deliver GMP drug products on time and within specification. This system can deliver autologous cell therapies at a scale and cost structure that makes these therapies accessible for much larger patient populations." The Cell Shuttle's automated, end-to-end manufacturing workflow reduces manual touchpoints, minimizes variability, and enables standardized execution across runs, equipment, and facilities. When combined with the Cell Q™ high-throughput quality control and release testing platform, the integrated Cellares solution is designed to meet the demands of scalable commercial production while maintaining the rigorous quality standards required for clinical and commercial-scale use. About Rese-cel Rese-cel (formerly referred to as CABA-201) is an investigational, autologous CAR T cell therapy engineered with a fully human CD19 binder and a 4-1BB co-stimulatory domain, designed specifically for the treatment of autoimmune diseases. Administered as a single, weight-based infusion, rese-cel is intended to transiently and deeply deplete CD19-positive cells, with the goal of resetting the immune system and achieving durable clinical responses without the need for chronic therapy. Cabaletta is evaluating rese-cel in the RESET™ (REstoring SElf-Tolerance) clinical development program, which includes multiple ongoing company-sponsored trials across a diverse and growing range of autoimmune diseases in rheumatology, neurology and dermatology. About Cabaletta Bio Cabaletta Bio (Nasdaq: CABA) is a late-stage clinical biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases. The CABA™ platform encompasses two complementary strategies which aim to advance the discovery and development of engineered T cell therapies with the potential to become deep and durable, perhaps curative, treatments for a broad range of autoimmune diseases. The lead CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy is prioritizing the development of rese-cel, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. Rese-cel is currently being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical development program spanning multiple therapeutic areas, including rheumatology, neurology and dermatology. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA. For more information, please visit and connect with us on LinkedIn. About Cellares Cellares is the first Integrated Development and Manufacturing Organization (IDMO), providing global cell therapy development and manufacturing services through an Industry 4.0 approach to the mass manufacture of the living drugs of the 21st century. The company enables biopharmaceutical partners to develop, scale, and commercialize cell therapies with the capacity, reliability, and economics required to meet total patient demand. Cellares’ fully automated platforms - Cell Shuttle™ for end-to-end cell therapy manufacturing and Cell Q™ for automated in-process and release quality control - are deployed across its network of IDMO Smart Factories worldwide. These technologies deliver industry-leading manufacturing economics, higher process success rates, and the ability to produce up to 10× more cell therapy batches than conventional CDMOs with comparable footprint and headcount, resulting in the lowest cost of manufacturing in the industry. Headquartered in South San Francisco, California, Cellares operates its first commercial-scale IDMO Smart Factory in Bridgewater, New Jersey, with additional facilities under construction in Europe and Japan. Through its global manufacturing network, Cellares is purpose-built to support both clinical and commercial programs and to expand access to life-saving cell therapies worldwide. For more information, visit and follow Cellares on LinkedIn . Contacts Cellares Contacts Business Development: bd@cellares.com Investors: ir@cellares.com Media: pr@cellares.com

2026-04-11

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411期🧬 CAR-T每日追踪 | 2026年4月

📰 今日要点速览 🔬 技术突破：中国医学科学院团队开发活性Rac1工程化CD33 CAR-T，显著增强骨髓浸润，为AML治疗开辟新路径 🧬 创新平台：LNP递送TYRP1-CAR mRNA实现体内原位CAR-巨噬细胞生成，用于脉络膜黑色素瘤 ⚠️ 安全警示：Mayo Clinic大样本研究揭示cilta-cel治疗后免疫效应细胞相关肠结肠炎（IEC-EC）的临床特征与管理策略 🧊 儿童突破：双胞胎MLL-AF4+婴儿ALL病例报告——序贯CD19/CD22 CAR-T成功挽救 📖 行业视角：CAR-T先驱Renier Brentjens博士接受采访，阐述领域瓶颈与未来方向📄 论文速递1️⃣ [创新策略] 活性Rac1介导骨髓滞留增强CD33 CAR-T对CD33⁺白血病细胞的杀伤效力 Wang S, Li L, Xing H, Xu Y et al.FASEB J. 2026 Apr 30;40(8):e71739.DOI: 10.1096/fj.202503832RR | PMID: 41961479 核心发现：骨髓微环境是白血病细胞（尤其是化疗耐药细胞）的关键"保护伞"，也是CAR-T治疗髓系肿瘤的主要障碍——T细胞难以有效浸润骨髓。研究团队通过工程化表达持续活性Rac1（Rac1V12）的CD33 CAR-T细胞，靶向Rac1 GTPase（细胞骨架动力学的核心调节因子）： ✅ 活性Rac1显著增强T细胞和CD33 CAR-T细胞的迁移能力，促进其在骨髓中定植 ✅ Transwell迁移依赖性杀伤实验证实Rac1V12 CAR-T体外杀伤力增强 ✅ 异种移植模型中，Rac1V12 CD33 CAR-T实现更强的白血病抑制，显著延长生存期 ✅ 机制：骨髓中Rac1V12 CAR-T呈增强的免疫记忆表型和更低的强直信号（tonic signaling） 🔑 意义：为AML等髓系恶性肿瘤的CAR-T治疗提供了通过分子工程增强组织浸润的新策略。2️⃣ [创新平台] LNP递送CAR-mRNA实现体内原位CAR-巨噬细胞生成用于脉络膜黑色素瘤 Chen W, Wang D, Li J, Ling J et al.Curr Eye Res. 2026 Apr 10;1-11.DOI: 10.1080/02713683.2026.2648868 | PMID: 41960698 核心发现：研究开发了F4/80抗体偶联的MPLA-LNP递送系统，封装TYRP1-CAR mRNA，通过玻璃体内注射将肿瘤相关巨噬细胞（TAM）原位重编程为功能性CAR-巨噬细胞： 🔬 脉络膜黑色素瘤（CNM）小鼠模型：肿瘤相关巨噬细胞呈M2优势表型 💉 F4/80/MPLA-LNP-TYRP1-CAR mRNA玻璃体内注射后，促进M1型极化，原位生成功能性TYRP1-CAR巨噬细胞 📉 治疗组显著减少肿瘤负荷（生物发光+肿瘤重量），延长生存期 🛡️ 血液生化、组织学分析未见全身毒性 🔑 意义：首次实现LNP介导的体内CAR-巨噬细胞生成，开辟了"off-the-shelf"实体瘤治疗新路径。3️⃣ [药物警戒] Cilta-cel治疗后免疫效应细胞相关肠结肠炎的诊断与管理 Lim KJ, Lee HE, Chen ZE, Bledsoe A, Parrondo RD et al. (Mayo Clinic)Blood Adv. 2026 Apr 10.DOI: 10.1182/bloodadvances.2025018853 | PMID: 41961998 核心发现（229例Mayo Clinic cilta-cel治疗患者回顾性分析）： 🧪 9例（3.9%）出现非顽固性、非血性、3级腹泻，常需长期肠外营养（TPN） ⏰ CAR-T输注至症状发作中位时间：85天（范围35-166天） 🦠 大多数病例伴有胃肠道共感染 🔬 病理：十二指肠活检多呈GVHD样黏膜损伤模式，2例呈T细胞淋巴增殖性疾病特征（CD4⁺/CD8⁺各1例） ⚠️ 独立危险因素：高危疾病（IMS/IMWG定义）、延长的CRS、前期延迟神经毒性 💊 一线治疗（激素、胆汁酸螯合剂、IVIG、抗菌）效果差，无持久应答 ✅ 生物制剂：维多珠单抗（2/2有效）、英夫利昔单抗（1/3有效）——无胃肠道共感染者疗效更佳 🔑 临床建议：早期胃肠镜评估（含十二指肠活检）至关重要；激素短期失败后应尽早启动生物制剂。4️⃣ [综述] 靶向COX-2/PGE2轴增强脑肿瘤NK和T细胞免疫治疗 Shen CJ, Florentino-Krasnov J, Liao YC, Tang HW et al.Cancer Immunol Immunother. 2026 Apr 10;75(5):141.DOI: 10.1007/s00262-026-04372-5 | PMID: 41961113 核心发现：胶质母细胞瘤（GBM）等侵袭性脑肿瘤面临三重屏障：血脑屏障（BBB）、显著的瘤内异质性、深度免疫抑制的肿瘤微环境（TME）。前列腺素E2（PGE2）是TME免疫抑制的关键驱动因子：通过EP2/EP4受体→升高cAMP→损害NK细胞激活受体（NKG2D、NKp30）、诱导CD8⁺ T细胞耗竭、促进Treg扩增 COX-2/PGE2轴介导对检查点抑制剂、CAR-T和化疗的耐药治疗策略：靶向该通路可恢复NK/T细胞功能，增敏免疫治疗；联合PGE2调控与CAR-NK/CAR-T平台有望重新定义GBM治疗范式。5️⃣ [临床综述] 弥漫大B细胞淋巴瘤（DLBCL）治疗 Falade AS, Ansell SM.Mayo Clin Proc. 2026 Apr 10.DOI: 10.1016/j.mayocp.2026.01.017 | PMID: 41961024 要点：DLBCL是最常见的非霍奇金淋巴瘤，占全球病例30%-40%。当前治疗进展：一线：R-CHOP±缩短疗程±ISRT Polatuzumab vedotin替代长春新碱对特定患者有益CAR-T已成为早期复发（<12个月）或难治性患者的首选方案二次进展后可用双特异性抗体、抗体药物偶联物（ADC）或新型抗体联合方案6️⃣ [临床综述] CAR-T治疗儿童神经自身免疫疾病：机制、应用与展望 Ling W, Xu C, Tang J, Lu S, Wu C.Front Pediatr. 2026 Mar 25;14:1740566.DOI: 10.3389/fped.2026.1740566 | PMID: 41960033📌 PMC免费全文要点：儿童神经自身免疫疾病（PNADs）包括MOGAD、NMDAR脑炎等，传统免疫治疗存在局限性。CAR-T通过靶向清除病理性B细胞，具有"免疫系统重置"潜力。早期证据支持在MOGAD和SLE等疾病中的治疗潜力。当前挑战：治疗成本高、CRS/神经毒性安全风险、儿童长期安全性数据缺乏。未来需优化全程管理流程、改进CAR设计、建立神经发育监测体系。7️⃣ [临床研究] R/R原发性纵隔B细胞淋巴瘤（PMBCL）真实世界多中心研究 Gugliotta G, Argnani L, Cantelli M, Pinto A et al.Am J Hematol. 2026 Apr 10.DOI: 10.1002/ajh.70317 | PMID: 41960655 要点：意大利真实世界多中心数据（2020年后）显示：R/R PMBCL患者总生存（OS）显著改善，主要归因于CAR-T细胞治疗作为挽救治疗的应用。研究支持帕博利珠单抗或纳武利尤单抗联合本妥昔单抗（nivo-BV）作为R/R PMBCL挽救治疗的安全性和有效性。8️⃣ [病例报告] 单卵双胞胎MLL-AF4⁺婴儿急性淋巴细胞白血病：序贯CAR-T成功挽救 Li C, Tong X, Yang J, Gao H, Li Z.Front Pediatr. 2026 Mar 25;14:1777726.DOI: 10.3389/fped.2026.1777726 | PMID: 41960026📌 PMC免费全文病例：单绒毛膜单卵双胞胎同时发生MLL-AF4⁺ ALL（极为罕见的MLL驱动的婴儿ALL侵袭性亚型）。St. Jude TOTXV方案获得完全缓解后出现分子学复发，序贯CD19和CD22 CAR-T细胞治疗成功挽救。因家属意愿及经济因素，未行异基因造血干细胞移植。9️⃣ [行业视角] CAR-T先驱Renier Brentjens博士访谈：障碍与机遇 Brentjens RJ, MD, PhD（CAR-T领域先驱，Roswell Park综合癌症中心）Cancer Discov. 2026 Apr 10;OF1.DOI: 10.1158/2159-8290.CD-NW2026-0032 | PMID: 41961988 Brentjens博士回顾了CAR-T的早期工作、当前局限及未来研究方向，为理解领域发展脉络提供了权威视角。🏥 临床试验动态 NCT号标题申办方阶段状态更新日期NCT06121297 RESET-SLE: CABA-201（CD19 CAR-T）治疗活动性SLE Cabaletta Bio I/II期 🟢 招募中 2026-04-09NCT05621291 NGS监测指导B-ALL的CAR-T后B细胞恢复管理 NCI 观察性 🟢 招募中 2026-04-09NCT05826535 Ronde-cel（CD19/CD20双靶点）治疗侵袭性LBCL Lyell Immunopharma I/II期 🟢 招募中 2026-04-09NCT04960579 P-BCMA-ALLO1异体CAR-T治疗多发性骨髓瘤 Poseida Therapeutics I期 🟢 招募中 2026-04-09NCT06575712 HCT患者应对技能培训干预研究 Duke University 观察性 🟢 招募中 2026-04-09📜 专利速报 Google Patents访问受限。基于近期研究热点，CAR-T专利布局聚焦于： 🧬 新靶点与结构：BCMA、GPRC5D、NKG2D、FAP、TYRP1等靶点CAR设计优化 🏭 体内递送平台：LNP递送mRNA实现体内CAR细胞生成（如Chen W et al.研究） 🛡️ 安全控制：逻辑门控CAR、诱导型系统、iCasp9自杀基因 🤝 联合策略：CAR-T与双特异性抗体、检查点抑制剂、COX-2抑制剂的联合方案 🧊 适应症扩展：从血液肿瘤→自身免疫→纤维化→眼部肿瘤📊 趋势总结🔥 本日三大看点 1. 髓系肿瘤新策略——Rac1工程化CAR-TWang S et al.的研究通过Rac1分子工程解决CAR-T骨髓浸润不足的老大难问题，这是实体瘤/髓系肿瘤"递送优化"路线的重要进展。 2. 体内CAR新范式——LNP递送CAR-mRNAChen W et al.首次实现体内原位CAR-巨噬细胞生成，"无需体外细胞制备"的新平台对CAR-T可及性和成本具有颠覆性潜力。 3. Cilta-cel安全性精细化管理Lim KJ et al.的Mayo Clinic大样本分析系统阐明了IEC-EC这一cilta-cel特异性毒性的特征——随着CAR-T适应症扩展，识别和管理产品特异性毒性成为关键课题。📈 大趋势追踪维度演进方向适应症血液肿瘤 → 实体瘤 → 自身免疫 → 眼部肿瘤/纤维化技术平台自体CAR-T → 异体/通用型 → 体内递送（非细胞）递送策略静脉输注 → 局部递送（心包内、玻璃体内） → 微环境响应释放安全性管理 CRS管理 → 产品特异性毒性精细识别 → 个体化预防策略扫一扫联系我们

免疫疗法细胞疗法信使RNA

100 项与 Resecabtagene Autoleucel 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
抗合成酶综合征	临床3期	美国	Cabaletta Bio, Inc.	2023-12-20
抗合成酶综合征	临床3期	英国	Cabaletta Bio, Inc.	2023-12-20
皮肌炎	临床3期	美国	Cabaletta Bio, Inc.	2023-12-20
皮肌炎	临床3期	英国	Cabaletta Bio, Inc.	2023-12-20
特发性炎症性肌病	临床3期	美国	Cabaletta Bio, Inc.	2023-12-20
特发性炎症性肌病	临床3期	英国	Cabaletta Bio, Inc.	2023-12-20
免疫介导的坏死性肌病	临床3期	美国	Cabaletta Bio, Inc.	2023-12-20
免疫介导的坏死性肌病	临床3期	英国	Cabaletta Bio, Inc.	2023-12-20
复发性多发性硬化	临床2期	-	Cabaletta Bio, Inc.	2026-06-01
慢性进行性多发性硬化	临床2期	-	Cabaletta Bio, Inc.	2026-06-01

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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


NCT06154252 (RESET-Myositis, ACR2025)	临床1/2期	特发性炎症性肌病	6	Rese-cel	窪廠壓鹽膚齋壓鏇憲鏇(鏇築餘衊襯餘繭鹽艱製) = Grade 1 cytokine release syndrome (CRS) in 3 of 6 patients, and no Grade 2 or higher CRS, dose-limiting toxicity or immune effector cell-associated neurotoxicity syndrome (ICANS). 顧選廠廠網廠遞顧簾鹹 (鬱繭憲遞鹹遞鹽積觸鑰 )	积极	2025-10-24
NCT06121297 (RESET-SLETM, ACR2025)	临床1/2期	狼疮性肾炎 \| 系统性红斑狼疮	7	Rese-cel	膚糧顧觸衊艱窪簾範膚(壓衊糧廠積窪糧構淵鹹) = 網鏇衊蓋廠網廠鹹糧齋顧簾齋鏇糧壓憲糧齋廠 (鑰衊糧膚糧窪蓋簾選繭 ) 更多	积极	2025-10-24
NCT06154252 \| NCT06328777 \| NCT06121297 (RESET-SSc, Company_Website) 人工标引	临床1/2期	肌炎 \| 系统性硬皮病 \| 系统性红斑狼疮	10	resecabtagene autoleucel (RESET-Myositis)	-	积极	2025-02-18
	临床1/2期	肌炎 \| 系统性硬皮病 \| 系统性红斑狼疮	10	resecabtagene autoleucel (RESET-SLE)	膚獵鹹夢衊鬱膚膚憲觸(糧鹽糧繭鹹繭壓醖廠觸) = 艱醖窪願顧糧積積鹽繭鬱繭膚夢醖鑰遞艱淵鹹 (簾獵選襯選鬱壓觸積鏇 ) 更多	积极	2025-02-18
RESET-Myositis (GlobeNewswire) 人工标引	临床1/2期	特发性炎症性肌病 \| 系统性硬皮病 \| 狼疮性肾炎 ... 更多	8	CABA-201	鑰鏇鑰鹹網鹽遞築窪構(網廠壓積製醖憲夢淵築) = 鏇鏇選繭鹹觸顧襯壓衊糧鹽衊構艱遞糧繭窪憲 (餘鏇鹹願顧簾蓋觸糧製 ) 更多	积极	2024-11-18
NCT06121297 (RESET-SLE, Biospace) 人工标引	临床1/2期	自身免疫性疾病 \| 系统性红斑狼疮 \| 免疫介导的坏死性肌病	2	CABA-201	膚醖鬱簾淵繭窪選顧鏇(鏇蓋壓鹹蓋襯鹽廠窪網) = None 願願製淵淵繭繭廠憲窪 (願廠築鹹願餘願網衊蓋 ) 更多	积极	2024-06-14