Resecabtagene Autoleucel

免预处理，一月几乎完全清除致病细胞，CAR-T治疗自免疾病结果亮眼 Cabaletta Bio近日公布其RESET-PV临床试验的初步数据。该试验旨在评估其在研自体CAR-T细胞疗法rese-cel（resecabtagene autoleucel，原名CABA-201）在无需预处理的情况下治疗寻常型天疱疮（PV）的效果。结果显示，接受1×10⁶个细胞/公斤剂量rese-cel的三名可评估患者，在输注后一个月，其B细胞几乎完全清除，两名患者实现自身抗体的快速下降和几乎完全的临床症状缓解；截至数据截止，三名患者均已停用免疫调节药物，并全部停用或正在减量使用糖皮质激素。研究同时观察到，在无预处理条件下，三名患者体内的CAR-T细胞扩增水平与其他RESET试验中30余名接受预处理患者的扩增情况相似。 这些初步数据支持在当前剂量下继续探索rese-cel无需预处理的治疗方案，并评估该方案在其他自身免疫疾病中的应用潜力。Cabaletta Bio指出，截至2025年9月30日，其在肌炎、红斑狼疮、硬皮病和重症肌无力等适应症的RESET系列1/2期成年患者队列已全部入组。Rese-cel是一种在研的自体CAR-T细胞疗法，采用全人源CD19结合域与4-1BB共刺激结构域设计，旨在通过一次按体重计算的单次输注，暂时但深度清除CD19阳性细胞，以重置免疫系统，实现无需长期治疗即可获得持久的临床缓解。 近80%癌症患者2年后仍存活，靶向免疫疗法2b期临床结果积极 OS Therapies今日宣布，其进行中的2b期临床试验获得积极结果。该试验旨在评估现货型免疫治疗候选药物OST-HER2在病灶完全切除的肺转移性骨肉瘤患者中，用于预防或延缓复发的疗效。分析显示，与历史对照组的40%相比，OST-HER2治疗组在可评估的36例患者中有75%（27例；另有5例失访）自最近一次肺部切除术起算的2年时仍存活，差异具统计学意义（p<0.0001）。分层分析进一步显示，达到12个月无事件生存（EFS）的患者，其2年总生存率为100%。 OST-HER2是一种创新免疫疗法，通过使用生物工程改造的单核细胞增生李斯特菌（Listeria monocytogenes）作为载体表达HER2蛋白，诱发对HER2阳性癌细胞的强效免疫反应。作为现货型方案，该疗法旨在清除表达HER2的原发肿瘤细胞并预防转移，从而推迟疾病复发；同时可作为单药使用，或与已获批疗法联合，以期提升患者的总生存率。 15亿美元布局体内细胞疗法！百时美施贵宝达成收购协议 百时美施贵宝（Bristol Myers Squibb）与Orbital Therapeutics今日宣布达成协议，百时美施贵宝将以15亿美元收购Orbital。Orbital致力于开创下一代RNA药物，在体内对免疫系统进行重编程，以满足不同疾病潜在生物学及其复杂性对疗法精准性、可控性与灵活性的要求。本次交易还包含其专有RNA平台，该平台整合环状与线性RNA工程化改造、先进的脂质纳米颗粒（LNP）递送及AI驱动设计，旨在实现持久、可编程的RNA疗法，以契合更广泛疾病谱的差异化需求。 根据协议，百时美施贵宝将获得Orbital旗下管线，其中主打的RNA免疫候选疗法OTX-201目前处于支持IND的临床前研究阶段。该候选疗法由优化的环状RNA构成，可在体内表达靶向CD19的钱和抗原受体，并通过定向LNP递送。与体外制备的CAR-T相比，此体内生成路径由患者自身充当生产工厂，有望显著降低治疗负担并提升可及性。通过本次收购，百时美施贵宝将把OTX-201与Orbital的平台能力纳入研发体系，强化其在RNA药物与细胞免疫治疗交叉领域的布局。 参考资料： [1] Cabaletta Bio Presents First Rese-cel Data with No Preconditioning Demonstrating Biologic Activity and Early Clinical Responses at the 2025 ESGCT Annual Congress. Retrieved October 10, 2025 from https://www.cabalettabio.com/investors/news-events/press-releases/detail/135/cabaletta-bio-presents-first-rese-cel-data-with-no [2] OS Therapies Announces Statistically Significant Positive Final 2-Year Overall Survival Data from Phase 2b Trial of OST-HER2 in the Prevention or Delay of Recurrent, Fully-Resected, Pulmonary Metastatic Osteosarcoma. Retrieved October 10, 2025 from https://ir.ostherapies.com/news-events/press-releases/detail/85/os-therapies-announces-statistically-significant-positive [3] Bristol Myers Squibb Strengthens and Diversifies Cell Therapy Portfolio with Acquisition of Orbital Therapeutics. Retrieved October 10, 2025 from https://news.bms.com/news/details/2025/Bristol-Myers-Squibb-Strengthens-and-Diversifies-Cell-Therapy-Portfolio-with-Acquisition-of-Orbital-Therapeutics/default.aspx 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

The B cells of a third patient were also depleted after treatment, though not completely, Cabaletta Bio said. \n Cabaletta Bio’s CAR-T cell therapy has eliminated the overreactive B cells of two patients with an autoimmune disease without preconditioning chemotherapy, pleasing investors and analysts while providing hope of a less toxic autoimmune treatment.The two patients are enrolled in one of Cabaletta’s six RESET phase 1/2 trials testing resecabtagene autoleucel (rese-cel) in a suite of autoimmune diseases. This trial, RESET-PV, is studying rese-cel in patients with pemphigus vulgaris, a rare autoimmune disease that causes blisters on the skin, throat, genitals and elsewhere.The B cells of a third patient were also depleted after treatment, though not completely, Cabaletta said. All three patients saw substantial improvements on the Pemphigus Disease Area Index, a measure of disease severity, in the month after rese-cel infusion. Rese-cel is an autologous treatment, meaning a patient’s T cells are extracted and modified in a lab to target B cells before being reinfused into the patient.Rese-cel was generally well-tolerated, Cabaletta said in an Oct. 9 release. One patient developed a fever from grade 1 cytokine release syndrome, and another needed a course of steroids after a disease flare that arose when immunomodulators were discontinued. Besides being off all immunomodulators, the three patients are also tapering glucocorticoids.Notably, Cabaletta reported no instances of immune effector cell-associated neurotoxicity syndrome (ICANS), a dangerous side effect that a patient in a different RESET trial developed after rese-cel treatment earlier this year. Philadelphia-based Cabaletta shared the new findings in a late-breaking presentation at the 2025 European Society of Gene & Cell Therapy conference in Sevilla, Spain.The biotech now plans to expand enrollment in the RESET-PV trial and then potentially up the dosage, while also pursuing regimens with no preconditioning in other RESET trials, Cabaletta Chief Medical Officer David Chang, M.D., said in the release.“These data provide preliminary evidence that a single infusion of rese-cel without preconditioning can achieve complete B cell depletion and meaningful early clinical responses with a simplified regimen that can expand access to patients who may desire a treatment option without preconditioning,” Chang said.Cabaletta’s stock price jumped from $2.40 per share to $3.25 after the data debut, with shares sitting at $2.71 as of 10 a.m. ET on Oct. 10.“The ability to eliminate preconditioning would be a colossal differentiator and would significantly decrease the treatment burden for patients,” William Blair analyst Sami Corwin, Ph.D., wrote in an Oct. 10 note. While uncertainties like “the small cohort size, limited follow-up duration, absence of tissue-level B cell data to confirm depletion in lymph nodes and lack of data demonstrating class switching in repopulated B cells” exist, “the data are the most impressive early evidence to date for using CAR-T without cytotoxic preconditioning.”The stock bump stands in contrast to Cabaletta\'s April announcement, when a patient in the biotech’s systemic sclerosis RESET trial suffered from grade 3 ICANS nine days after treatment. The patient experienced a “transient period of confusion,” the company said at the time, with the complication clearing up after treatment with dexamethasone. Cabaletta’s stock dipped 15% as a result, though analysts were less concerned.“We believe the event could have been prevented,” William Blair analysts wrote in an April 1 note, agreeing with Cabaletta’s position that the adverse event was caused by a failure to follow proper trial protocol.