Descartes-08

嵌合抗原受体（CAR）是经人工改造的合成蛋白，可改变人体免疫细胞的作用模式，使其识别并清除病变有害细胞。这类蛋白被固定在T细胞表面，赋能免疫细胞对抗疾病。目前，CAR-T细胞疗法已获批上市用于癌症治疗，2017年获批的凯希利珠单抗（Kymriah）便是全球首款CAR-T药物。近年来，CAR-T疗法也逐步进入临床研究，用于治疗各类自身免疫性疾病。 CAR-T疗法治疗自身免疫病的作用机制与治疗癌症相似，区别在于：该疗法不会靶向癌细胞，而是通过基因编辑识别并清除体内过度活化的异常免疫细胞。 目前全球暂无获批用于自身免疫疾病的CAR-T疗法，但凯维纳医疗（Kyverna Therapeutics）的米夫卡赛自体CAR-T细胞（miv-cel）已接近获批。本文梳理了8家核心企业，其CAR-T疗法正处于自身免疫疾病临床试验阶段。目录 1. Kyverna Therapeutics 2. Cabaletta Bio 3. Cartesian Therapeutics 4. Autolus Therapeutics 5. Allogene Therapeutics 6. Quell Therapeutics 7. Sonoma Therapeutics 8. Miltenyi Biomedicine 9. 基因编辑CAR-T：开辟全新治疗路径Kyverna Therapeutics 总部：美国加利福尼亚州 核心候选药物：miv-cel（Ⅲ期临床试验） 僵人综合征是一种神经性自身免疫病，核心症状为肌肉僵硬，患者大多会频繁出现肌肉痉挛，终日伴随疼痛感。约80%的患者会出现行动能力退化，最终无法独立行走。现有治疗手段仅能缓解症状，无法实现治愈。凯维纳医疗研发的CAR-T细胞疗法直击该病发病根源，预计将于今年年底获批上市。 候选药物miv-cel以CD19抗原为靶点，可减少细胞因子释放、清除致病B细胞，同时共刺激CD28靶点，大幅提升治疗效力。其二期临床试验数据结果亮眼：单次给药即可改善患者行动能力、逆转疾病严重程度相关的残疾评分，让患者摆脱僵人综合征的免疫治疗依赖。该研究成果于今年4月在芝加哥举办的美国神经病学学会（AAN）年会上公布。 今年5月，这家初创企业启动了向美国食品药品监督管理局（FDA）的滚动式生物制品许可申请（BLA），预计年底完成全部申报材料提交。滚动式申报允许药企分批次递交审批材料，无需一次性提交全部文件。 目前，该疗法针对重症肌无力的二期临床试验也正在开展。重症肌无力是一种神经-肌肉信号传导受阻引发的自身免疫病，主要表现为肌肉无力、易疲劳，患者自主运动肌肉失控，还可能出现复视、言语及吞咽困难、眼睑下垂等症状。24周治疗随访数据显示，所有患者的重症肌无力日常生活能力评分（MG-ADL）、定量重症肌无力评分（QMG）均持续改善，两项指标分别用于评估病症影响程度与肌肉无力严重程度。公司目前已全面筹备该适应症的三期临床试验。 为保障miv-cel获批后的商业化生产，这家加州企业已与马萨诸塞州的合同研发生产组织（CDMO）易升生物（ElevateBio）达成合作，由其承接药物商业化生产工作。Cabaletta Bio 总部：美国宾夕法尼亚州 核心候选药物：rese-cel（Ⅰ/Ⅱ期临床试验） 卡巴莱塔生物研发的rese-cel是一款全人源CD19 CAR-T疗法，搭载4-1BB共刺激结构域。CD19是主要表达于B细胞表面的蛋白，是调控人体免疫应答的关键分子，也是癌症及自身免疫病治疗的核心靶点。企业表示，rese-cel具备“重置人体免疫系统”的潜力。 目前，rese-cel针对系统性红斑狼疮、肌炎、系统性硬化症、全身性重症肌无力、寻常型天疱疮的Ⅰ/Ⅱ期临床试验正在推进。今年6月，该药物肌炎适应症临床试验取得阳性结果。肌炎由免疫系统错误攻击骨骼肌健康纤维引发，会导致骨骼肌慢性炎症、肌力衰退。 临床试验中，停用常规免疫调节剂的患者里，83%达到主要临床终点，且停药后一年半内疗效持续稳定。其中一名幼年皮肌炎（儿童罕见肌炎亚型）患者实现中度总体改善评分（TIS）应答，疗效持续维持32周。总体改善评分是评估炎症性肌肉疾病治疗效果的核心指标。 与此同时，系统性红斑狼疮适应症临床试验仍在进行中。系统性红斑狼疮患者因免疫系统过度活化，会出现关节、皮肤、肾脏等多器官炎症损伤。试验数据显示，无需预处理化疗的最低剂量rese-cel，可清除两名受试患者中一名的致病B细胞。预处理化疗是CAR-T治疗前的短期化疗方案，用于促进CAR-T细胞在体内增殖活化。 今年早些时候，企业在马萨诸塞州波士顿举办的美国基因与细胞治疗学会（ASGCT）2026年年会上，公布了rese-cel治疗寻常型天疱疮的临床试验数据。寻常型天疱疮是一种自身免疫病，免疫系统会攻击皮肤细胞粘连蛋白，导致皮肤出现疼痛性水疱。数据显示，经预处理化疗联合rese-cel治疗后，50%的患者实现持续6个月的无药物缓解，疗效显著。 目前，卡巴莱塔生物已启动rese-cel商业化筹备工作，与加州合同研发生产企业赛拉瑞斯（Cellares）签订一年期商业化生产合作协议，实现疗法规模化生产。该企业还于今年5月通过公开募资获得1.5亿美元融资。Cartesian Therapeutics 总部：美国马里兰州 核心候选药物：Descartes-08（Ⅲ期临床试验） 美国马里兰州的笛卡尔医疗创新性结合mRNA技术与CAR-T细胞疗法，用于治疗自身免疫疾病。其核心候选药物Descartes-08主攻重症肌无力，目前已进入三期临床试验阶段。 该疗法靶向B细胞成熟抗原（BCMA），依托mRNA技术突破传统DNA型CAR-T的技术局限，无需术前预处理化疗，规避了基因组整合风险与治疗毒性问题，适用于重症肌无力等多种自身免疫病。 二期临床试验数据显示，71%的重症肌无力患者接受Descartes-08治疗后，重症肌无力综合评分（MGC）显著改善，而安慰剂组仅25%的患者出现有效病情缓解。三期临床试验数据预计将于明年年初公布。 目前，该药物针对系统性红斑狼疮、肌炎的二期临床试验也在开展。正在进行的系统性红斑狼疮临床试验中，所有受试患者均达到狼疮低疾病活动状态（LLDAS，红斑狼疮治疗核心疗效标准），三分之二的患者治疗3个月后实现病情完全缓解，且治疗安全性良好。其肌炎适应症的二期临床试验也在同步评估中。 企业另一候选药物Descartes-15一期临床数据表现优异，但已于去年年底暂停研发，目前管线内核心推进品种为Descartes-08。此外，企业两个月前与中国西吉生物达成合作，共同开发体内CAR-T技术平台。今年5月，该公司完成1.5亿美元非稀释性融资。Autolus Therapeutics 总部：英国伦敦 自身免疫病候选药物：obe-cel（Ⅱ期临床试验） 伦敦高校衍生企业奥图勒斯医疗，正推进核心候选药物obe-cel用于系统性红斑狼疮、狼疮性肾炎、多发性硬化症的治疗研发。狼疮性肾炎是系统性红斑狼疮的严重并发症，会造成肾脏严重器质性损伤，目前该疗法正处于狼疮性肾炎二期临床试验阶段。 同时，企业针对经常规治疗无法缓解、难以实现病情缓解的难治性重度系统性红斑狼疮患者，开展obe-cel疗效评估。该疗法靶向狼疮发病的核心致病细胞——CD19阳性B细胞。 一期早期临床数据显示，5000万细胞剂量组患者可获得持久治疗应答，1亿细胞剂量组三名患者均出现早期病情改善。试验证实，obe-cel可有效清除靶向致病B细胞，实现免疫系统重置，且治疗安全性良好，未出现任何毒性反应。该研究成果于去年12月在美国血液学会（ASH）年会上发布。 此外，针对多发性硬化症（免疫系统攻击中枢神经系统，引发肢体麻木、重度疲劳），该疗法的一期临床试验已启动，首例患者已于去年10月完成给药。 obe-cel同时布局血液肿瘤适应症。2024年，该英国生物科技企业与拜恩泰科（BioNtech）达成2.5亿美元合作协议，拜恩泰科收购其英国生产基地用于实体瘤疫苗研发，并获得其儿童血液肿瘤细胞疗法的授权选择权。合作包含2亿美元股权收购与5000万美元现金付款，但拜恩泰科已于去年放弃该授权选择权。Allogene Therapeutics 总部：美国加利福尼亚州 自身免疫病候选药物：ALLO-329（Ⅰ期临床试验） 奥罗根医疗的通用型细胞疗法在淋巴瘤残留病灶治疗中表现优异，可使超半数患者病灶清除。这家加州初创企业同时布局自身免疫疾病赛道，核心产品为基因编辑CAR-T疗法ALLO-329。 该药物于2025年获得美国FDA临床试验默示许可，正式启动针对系统性红斑狼疮、狼疮性肾炎、特发性炎症性肌病、系统性硬化症的一期临床试验。 这款CAR-T疗法双靶向B细胞表面的CD19、CD70两大自身免疫病关键致病靶点，依托CRISPR基因编辑技术实现CAR稳定表达，并搭载企业独创的Dagger技术平台，无需术前预处理化疗。 一期临床试验设置两组治疗方案，分别为联合预处理化疗、不联合预处理化疗，重点评估ALLO-329的安全性与初步疗效。 临床前研究证实，该疗法可有效规避免疫排斥、促进CAR-T细胞体内扩增、延缓狼疮疾病进展。目前，ALLO-329已获得美国FDA三项快速通道资格，临床试验数据预计于今年年底公布。今年5月，企业通过公开募资斩获2.004亿美元融资。Quell Therapeutics 总部：英国伦敦 核心候选药物：QEL-005（Ⅰ期临床试验） 英国伦敦的奎尔医疗突破传统CAR-T研发思路，摒弃常规效应T细胞改造模式，专注于改造调节性T细胞（Treg细胞），研发CAR-Treg疗法，适应症覆盖2型糖尿病、各类自身免疫病、炎症性肠病及肝移植术后免疫排斥干预。 企业最成熟的候选药物QEL-005，主打治疗全球5%~10%人口罹患的风湿性自身免疫病，这类疾病主要累及人体关节与肌肉。 区别于传统CAR-T“杀伤致病细胞”的机制，QEL-005采用“温和调控、而非彻底清除”的创新治疗理念，不直接清除B细胞，而是在炎症病灶处特异性激活，抑制促炎免疫细胞活性、调控免疫平衡，从而减轻全身炎症反应。 该疗法去年10月公布优异临床前数据，今年3月正式启动一期临床试验。研究证实，其可有效抑制T细胞异常活化、调控B细胞功能与抗体分泌，同时调节炎症巨噬细胞等多种免疫细胞活性。相关研究成果已于去年芝加哥举办的美国风湿病学会年会上发布。 去年，该英国企业与美国华盛顿州合同研发生产企业AGC生物制药达成合作，依托后者慢病毒载体技术，开展Treg细胞疗法的研发与生产。Sonoma Therapeutics 总部：美国加利福尼亚州 核心候选药物：SBT-77-7101（Ⅰ/Ⅱ期临床试验） 与奎尔医疗研发方向一致，美国加州的索诺玛医疗聚焦CAR改造Treg细胞疗法，通过重塑免疫系统平衡治疗自身免疫疾病，从根源阻断疾病进展，而非单纯控制临床症状。 企业核心候选药物SBT-77-7101，目前处于Ⅰ/Ⅱ期临床试验阶段，用于治疗类风湿关节炎、化脓性汗腺炎。化脓性汗腺炎虽未被归类为自身免疫病，但由免疫系统过度活化、慢性炎症引发，毛囊堵塞后会形成皮下肿块，未及时治疗可扩散蔓延。SBT-77-7101可精准抑制病灶炎症信号，对类风湿关节炎（免疫细胞攻击关节健康组织，引发关节肿痛）同样具备抗炎治疗效果。 该疗法可靶向关节病灶蛋白，不破坏正常细胞，精准抑制关节局部炎症。一期安全性临床试验数据显示，6名受试患者中4人关节肿痛症状缓解50%；治疗4周后，5名患者的类风湿关节炎疾病活动度评分（DAS28-CRP、CDAI）显著下降。试验全程未出现剂量限制性毒性、细胞因子释放综合征（CRS）、免疫效应细胞相关神经毒性综合征（ICANS），治疗安全性优异。 上月，企业与赛拉瑞斯达成合作，启动SBT-77-7101规模化生产。两年前，再生元制药向其投资4500万美元，合作开发Treg细胞疗法，双方共同推进的乳糜泻、溃疡性结肠炎、克罗恩病疗法，目前均处于发现及临床前研发阶段。Miltenyi Biomedicine 总部：德国贝吉施格拉德巴赫 自身免疫病核心候选药物：zamtocabtagene autoleucel（Ⅱ期临床试验） 美天旎生物医学是德国美天旎生物技术的衍生企业，专注于细胞疗法研发。企业核心药物zamtocabtagene autoleucel已提交上市申请，用于治疗侵袭性非霍奇金淋巴瘤——大B细胞淋巴瘤，同时该药物也布局自身免疫疾病适应症。 该CAR-T疗法双靶向B细胞表面CD19、CD20抗原，可精准清除多发性硬化症等自身免疫病中的自身反应性致病B细胞。目前，其针对狼疮性肾炎、系统性红斑狼疮、系统性硬化症、多发性硬化症的一期临床试验正在开展。 企业另一款自身免疫病候选药物zorpocabtagene autoleucel，单靶向CD19抗原，目前处于二期临床试验阶段，用于治疗系统性红斑狼疮、系统性硬化症、皮肌炎、多发性肌炎等慢性炎症性肌肉疾病。 该药物针对系统性硬化症、特发性炎症性肌病、系统性红斑狼疮的篮式临床试验，已全部达成主要及次要临床终点。单次给药治疗24周内，所有患者实现病情缓解，且全程无需依赖类固醇药物治疗。基因编辑CAR-T：开辟全新治疗路径 传统CAR-T疗法需依托病毒载体，在体外完成T细胞CAR基因修饰，再回输患者体内。而新型研发思路聚焦基因编辑技术，通过直接编辑CAR-T细胞基因，大幅提升治疗精准度，同时缩短治疗周期，无需患者长期等待细胞制备与回输。 奥罗根医疗是该赛道代表企业之一，瑞士-美国生物科技企业克瑞斯普治疗（CRISPR Therapeutics）同样布局深远。其核心产品为基于CRISPR/Cas9基因编辑的通用型CAR-T疗法，目前正在开展B细胞源性癌症及自身免疫疾病临床试验。 研究人员表示，基因编辑CAR-T技术有望攻克多种无有效治疗手段的罕见病，包括罕见纤维化疾病。通过基因编程，CAR-T细胞可精准清除心脏、肝脏等器官的致纤维化细胞，阻断器官瘢痕化进程。

Phase 3 AURORA trial of Descartes-08 in myasthenia gravis (MG) continues to advance; data expected in 1Q27, biologics license application (BLA) filing planned for mid-2027 Phase 2 TRITON trial of Descartes-08 in myositis on track with data from subset of patients expected in 1H27 Phase 1/2 HELIOS pediatric trial of Descartes-08 in juvenile dermatomyositis (JDM) continues to progress with data expected in 1H27 Phase 1 in vivo trial in MG patients, in partnership with WestGene BioPharma (WestGene), expected to initiate in 2H26; clinical data expected in 1H27 ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Approximately $149.3 million cash, cash equivalents and restricted cash as of June 30, 2026 , expected to support planned operations into 2028 FREDERICK, Md., Aug. 06, 2026 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (“we”, the “Company” or “Cartesian”), a late clinical-stage biotechnology company pioneering cell therapy for autoimmune diseases, today reported financial results for the second quarter ended June 30, 2026, and outlined recent business updates. “This quarter was marked by significant progress as we secured a strategic partnership to explore an in vivo platform and executed a non-dilutive financing, enhancing our pipeline and extending cash runway. As we prepare for four expected clinical readouts over the next twelve months, including from our Phase 3 AURORA trial in patients with MG in the first quarter of 2027, these agreements further strengthen our emerging pipeline and financial position,” said Carsten Brunn, Ph.D., President and Chief Executive Officer of Cartesian. “Our partnership with WestGene gives us an efficient, accelerated path to extend our payloads into in vivo delivery, with in-human clinical data expected in the first half of next year. While our top priority remains executing on our Phase 3 AURORA trial, our WestGene partnership is intended to create future optionality for Cartesian across MG and other autoimmune indications, with the potential to further enhance cell therapy delivery and shift the treatment paradigm. As we advance toward this next phase of growth, an extended cash runway into 2028, supported by up to $150 million of non-dilutive financing through a credit facility with K2 HealthVentures (“K2HV”), allows us to continue investing in precommercial readiness activities in parallel with clinical execution. We look forward to a robust set of near-term milestones ahead, each bringing us closer to addressing the significant unmet need for deep and durable treatments in autoimmune diseases.” Pipeline Progress and Anticipated Milestones Phase 3 AURORA Trial of Descartes-08 in Participants with MG; Data Expected in 1Q27, with BLA Planned for Mid-2027. The randomized, double-blind, placebo-controlled Phase 3 AURORA trial is designed to assess Descartes-08, Cartesian’s autologous anti-B cell maturation antigen (BCMA) chimeric antigen receptor T-cell therapy (CAR-T) versus placebo (1:1 randomization) administered as six once-weekly outpatient infusions without preconditioning chemotherapy in approximately 100 patients with acetylcholine receptor autoantibody positive (AChR Ab+) MG. The primary endpoint will assess the proportion of Descartes-08 participants with an improvement in MG Activities of Daily Living (MG-ADL) score of three points or more at Month 4 compared to placebo. Announced New Strategic Licensing Agreement with WestGene Intended to Accelerate the Development of In Vivo CAR-T Platform in Autoimmune Diseases; Phase 1 Data Expected in 1H27. Cartesian has partnered with WestGene to conduct a Phase 1 dose-escalation trial of the mRNA used in Descartes-08 delivered via WestGene’s proprietary targeted lipid nanoparticles (tLNPs) in patients with MG. Intravenous infusions will be administered across multiple dose levels using a Bayesian Optimal Interval (BOIN) adaptive design with a comprehensive translational assessment package including clinical response measures. The program represents a novel in vivo approach to BCMA-directed T-cell engineering that, if successful, could eliminate the need for ex vivo manufacturing. Cartesian is also planning to advance multiple internally developed next-generation anti-BCMA CAR constructs and a BCMA-directed T-cell engager (TCE) as part of its expanding mRNA payload portfolio. The WestGene partnership is designed to provide an efficient framework to move additional Cartesian payloads into human trials, extending the platform’s potential to generate clinical proof-of-concept data across multiple programs in several disease states. Under the terms of the agreement, WestGene received an upfront payment and is eligible to receive potential development and commercial based milestone payments. This clinical trial is expected to initiate in the second half of 2026 with in-human data expected in the first half of 2027. Phase 2 TRITON Trial in Myositis Remains on Track with Data from Subset of Patients Expected in 1H27 to Inform Path Forward to Pivotal Trial. The randomized, double-blind, placebo-controlled Phase 2 TRITON trial in myositis is designed to assess Descartes-08 versus placebo (1:1 randomization) administered as six weekly outpatient infusions without preconditioning chemotherapy in patients with moderate to severe multi-refractory dermatomyositis and antisynthetase syndrome. The primary endpoint is to assess the safety and efficacy of Descartes-08 compared to placebo added to standard of care in participants with myositis. The Company plans to evaluate a subset of patients from the trial to determine the potential path to a pivotal trial in myositis. Phase 1/2 HELIOS Pediatric Trial of Descartes-08 in Autoimmune Diseases, Including JDM, Continues to Progress with Data Expected in 1H27. Enrollment remains ongoing in the Phase 1/2 HELIOS pediatric trial of Descartes-08 in children and young adults with autoimmune diseases, including JDM. JDM is a rare pediatric autoimmune disorder marked by pathognomonic skin rash and muscle inflammation affecting multiple organ systems. The U.S. Food and Drug Administration (FDA) previously granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of JDM. Corporate Update Cash Runway Extended into 2028 with Up to $150 Million of Non-Dilutive Financing from K2HV Secured. Under the Company’s credit facility with K2HV, the first $50 million term loan was funded upon signing of the agreement in May 2026. The second $25 million term loan is expected to be available to be drawn between January 1, 2027 and December 1, 2027, subject to the Company’s achievement of specified clinical and financing milestones and the third $25 million term loan is expected to be available to be drawn between January 1, 2028 and June 1, 2028, subject to the Company’s achievement of specified approval and sales milestones. An additional $50 million tranche is available for draw at the Company’s option subject to K2HV’s discretion. The Company now anticipates current cash resources to support planned operations into 2028, including through four expected clinical data readouts and accelerated investment in precommercial activities. Second Quarter 2026 Financial Results Cash, cash equivalents and restricted cash as of June 30, 2026 was $149.3 million, inclusive of the initial $50 million tranche of non-dilutive financing from K2HV and $19.3 million raised year to date after commissions and expenses through the Company’s at the market (ATM) offering program. The Company’s current cash resources on hand are expected to support planned operations, including completion of the ongoing Phase 3 AURORA trial, into 2028. Research and development expenses were $20.4 million for the three months ended June 30, 2026, compared to $14.9 million for the three months ended June 30, 2025. The increase was primarily a result of increased expenses associated with the ongoing Phase 3 AURORA trial, partially offset by a decrease in stock-based compensation expenses and a decrease in expenses for early stage programs. General and administrative expenses were $8.7 million for the three months ended June 30, 2026, compared to $7.2 million for the three months ended June 30, 2025. The increase was primarily the result of higher professional and consulting fees. Net income was $15.8 million, or $0.47 net income per share allocable to common stockholders (basic), for the three months ended June 30, 2026, compared to net income of $15.9 million, or $0.51 net income per share allocable to common stockholders (basic), for the three months ended June 30, 2025. About Descartes-08 Descartes-08, Cartesian’s lead cell therapy candidate, is an autologous CAR-T product targeting BCMA in clinical development for generalized MG and myositis, specifically dermatomyositis and antisynthetase syndrome. In contrast to conventional DNA-based CAR T-cell therapies, Cartesian’s CAR-T administration is designed to not require preconditioning chemotherapy, can be administered in the outpatient setting, and does not carry the risk of genomic integration associated with cancerous transformation. Descartes-08 has been granted Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation by the U.S. Food and Drug Administration for the treatment of MG, and Rare Pediatric Disease Designation for the treatment of JDM. About Cartesian Therapeutics Cartesian Therapeutics is a late clinical-stage company pioneering cell therapy for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is a CAR-T in Phase 3 clinical development for patients with generalized myasthenia gravis, Phase 2 clinical development in myositis, specifically dermatomyositis and antisynthetase syndrome, and in Phase 1/2 clinical development for pediatric autoimmune diseases, including juvenile dermatomyositis. For more information, please visit or follow the Company on LinkedIn or X. Forward Looking Statements Any statements in this press release about the future expectations, plans and prospects of the Company, including without limitation, statements regarding the Company’s expected cash resources and cash runway, the availability and use of funds under the Company’s credit facility with K2HV, statements regarding the partnership and strategic licensing agreement between the Company and WestGene and the ability of the Company and WestGene to develop therapies, treat disease, extend mRNA payloads into in vivo delivery, the integration and complementary nature of the Company’s product candidates and WestGene’s lipid nanoparticles platform, the speed and efficiency of the development pathway for collaboration products between the Company and WestGene, the ability of the Company and its partnership with WestGene to generate clinical proof-of-concept data across multiple programs in several disease states, the ability of the Company and its partnership with WestGene to advance multiple internally developed next-generation anti-BCMA CAR constructs and a BCMA-directed TCE and the speed at which these constructs and TCE are developed, the ability of the Company’s product candidates to be administered in an outpatient setting or without the need for preconditioning lymphodepleting chemotherapy, the potential of Descartes-08, or any of the Company’s other product candidates to treat MG, juvenile MG, myositis, JDM, or any other disease, the anticipated timing or the outcome of ongoing and planned clinical trials, studies and data readouts, including the ongoing Phase 3 AURORA trial of Descartes-08 in MG, the ongoing Phase 2 TRITON trial of Descartes-08 in myositis, and the ongoing Phase 1/2 HELIOS pediatric trial of Descartes-08 in autoimmune diseases, including JDM, the anticipated timing or the outcome of the FDA’s review of the Company’s regulatory filings, including the number of trials that may be necessary in order to obtain marketing approval, the Company’s ability to conduct its clinical trials and preclinical studies, the timing or making of any regulatory filings, the anticipated timing or outcome of selection of developmental product candidates, the ability of the Company to enter into and maintain potential collaborations or partnerships, the novelty of treatment paradigms that the Company is able to develop, the potential of any therapies developed by the Company to fulfill unmet medical needs, and enrollment in the Company’s clinical trials and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial and whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human subjects, the unproven approach of the Company’s technology, potential delays in enrollment of patients, undesirable side effects of the Company’s product candidates, political uncertainty, the Company’s reliance on third parties to conduct its clinical trials, the Company’s inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the Company’s recurring losses from operations and negative cash flows, substantial fluctuation in the price of the Company’s common stock, risks related to geopolitical conflicts, pandemics, and macroeconomic impacts, and other important factors discussed in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and subsequently filed Quarterly Reports on Form 10-Q, and in other filings that the Company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the Company’s views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any intention to update any forward-looking statements included in this press release, except as required by law. Cartesian Therapeutics, Inc. and Subsidiaries Consolidated Balance Sheets (Amounts in thousands, except share data and par value) June 30, December 31, 2026 2025 Assets Current assets: Cash and cash equivalents $ 147,606 $ 125,139 Accounts receivable 196 1,115 Prepaid expenses and other current assets 5,813 3,022 Total current assets 153,615 129,276 Property and equipment, net 11,311 12,185 Right-of-use assets, net 5,124 5,601 In-process research and development asset 93,900 93,900 Goodwill 48,163 48,163 Long-term restricted cash 1,735 1,735 Long-term prepaid expenses and other assets 4,780 5,551 Total assets $ 318,628 $ 296,411 Liabilities and stockholders’ deficit Current liabilities: Accounts payable $ 1,046 $ 1,288 Accrued expenses and other current liabilities 15,142 9,498 Lease liabilities 4,197 4,151 Warrant liability 165 — Total current liabilities 20,550 14,937 Lease liabilities, net of current portion 6,805 8,525 Warrant liability, net of current portion — 141 Long-term debt, net 52,851 — Contingent value rights liability 356,700 392,100 Deferred tax liabilities, net 6,948 6,948 Total liabilities 443,854 422,651 Stockholders’ deficit: Series A Preferred Stock, $0.0001 par value; 112,164.533 and 134,904.563 shares authorized as of June 30, 2026 and December 31, 2025, respectively; 98,050.372 and 120,790.402 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively — — Series B Preferred Stock, $0.0001 par value; 437,927 shares authorized as of June 30, 2026 and December 31, 2025; 437,927 shares issued and outstanding as of June 30, 2026 and December 31, 2025 — — Preferred stock, $0.0001 par value; 9,449,908.467 and 9,427,168.437 shares authorized as of June 30, 2026 and December 31, 2025, respectively; no shares issued and outstanding as of June 30, 2026 and December 31, 2025 — — Common stock, $0.0001 par value; 350,000,000 shares authorized as of June 30, 2026 and December 31, 2025; 30,037,962 and 26,011,106 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively 3 3 Additional paid-in capital 725,165 700,706 Accumulated deficit (845,786 ) (822,373 ) Accumulated other comprehensive loss (4,608 ) (4,576 ) Total stockholders’ deficit (125,226 ) (126,240 ) Total liabilities and stockholders’ deficit $ 318,628 $ 296,411 Cartesian Therapeutics, Inc. and Subsidiaries Consolidated Statements of Operations and Comprehensive Loss (Amounts in thousands, except share and per share data) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Revenues: Collaboration and license $ — $ — $ — $ 400 Grant — 298 78 998 Total revenues — 298 78 1,398 Operating expenses: Research and development 20,431 14,869 39,894 29,543 General and administrative 8,724 7,240 15,838 15,555 Total operating expenses 29,155 22,109 55,732 45,098 Operating loss (29,155 ) (21,811 ) (55,654 ) (43,700 ) Other income (expense): Interest income 1,103 1,748 2,129 3,763 Interest expense (852 ) — (852 ) — (Loss) gain on change in fair value of warrant liability (118 ) 654 (24 ) 2,472 Loss on change in fair value of embedded derivative (4,535 ) — (4,535 ) — Gain on change in fair value of contingent value rights liability 49,200 35,300 35,400 35,646 Other income (expense), net 126 (5 ) 123 (5 ) Total other income, net 44,924 37,697 32,241 41,876 Net income (loss) $ 15,769 $ 15,886 $ (23,413 ) $ (1,824 ) Other comprehensive (loss) income: Foreign currency translation adjustment (25 ) 12 (32 ) 44 Total comprehensive income (loss) $ 15,744 $ 15,898 $ (23,445 ) $ (1,780 ) Net income (loss) $ 15,769 $ 15,886 (23,413 ) (1,824 ) Less: Undistributed earnings allocable to participating securities (2,049 ) (2,628 ) — — Net income (loss) allocable to shares of common stock - basic and diluted $ 13,720 $ 13,258 $ (23,413 ) $ (1,824 ) Net income (loss) per share allocable to common stockholders: Basic $ 0.47 $ 0.51 $ (0.83 ) $ (0.07 ) Diluted $ 0.46 $ 0.50 $ (0.83 ) $ (0.07 ) Weighted-average common shares outstanding: Basic 29,467,130 25,980,262 28,142,256 25,941,670 Diluted 29,860,880 26,447,251 28,142,256 25,941,670 Investor Contact Megan LeDuc Associate Director of Investor Relations megan.leduc@cartesiantx.com Media Contact David Rosen Argot Partners david.rosen@argotpartners.com