CAR-T 细胞疗法 已在血液恶性肿瘤领域取得显著效果，正在扩展至实体瘤、感染性疾病以及自身免疫疾病的临床试验。 近年来，中外学者利用 CAR-T 细胞疗法成功治疗了包括 系统性红斑狼疮、 特发性炎性肌病、重症肌无力、多发性硬化症 在内的多种 自身免疫疾病 。CAR-T 细胞治疗自身免疫病也入选了 Science 2024 年度十大科学突破。 在产业界， CAR-T 细胞疗法也已进入爆发前夜。2025 年 6 月 30 日， 艾伯维 以 21 亿美元 现金 收购了 CAR-T 细胞疗法开发 公司 Capstan ，该公司目前有 一款 in vivo CAR-T 细胞疗法用于治疗自身免疫疾病，目前处于临床 1 期。而在今年 3 月中旬， 阿斯利康 以 10 亿美元价格收购 CAR-T 细胞疗法开发公司 EsoBiotec 时 ， 就已经给市场带来惊喜。现在，艾伯维给出的翻倍收购价更是让产业界看到了 CAR-T （尤其是 in vivo CAR-T） 更大的发展空间。 CAR-T 细胞疗法在自身免疫疾病中的应用，始于 德国埃尔朗根-纽伦堡大学的 Georg Schett 教授， 2021 年，他领导的团队首次使用 ...

招股书显示，这款瞄准银屑病、强直性脊柱炎等适应证的口服小分子药，凭借"中国首个双机制PDE4抑 制剂"已进入NDA（新药上市申请）优先审评，不过，这个赛道已是硝烟弥漫：前有同类竞品药进入集 采被砍价超过93%，后有37款临床竞品围追堵截，这也为该药品如愿获批上市后的商业化表现增添了不 确定性。 明星的病情能够引发公众对自身免疫疾病的关注，但身处其中的创新药企能否借资本破局，还得靠药效 说话。 核心产品面临激烈竞争 作为一家计划依据港股18A规则上市的生物医药企业，和美药业具有这类公司典型的高投入、高潜力特 征。 当李宇春轮椅出行的画面刺痛公众神经，当周杰伦近期佝偻行走的视频引爆热搜，"强直性脊柱炎"这个 医学名词在一夜之间"火"了。 公开资料显示，中国有约400万名强直性脊柱炎患者。实际上，在自身免疫疾病方面，中国有超过8000 万位患者面临无药根治的困境。 《每日经济新闻》记者注意到，近日，一家深耕该领域的药企赣州和美药业股份有限公司（以下简称和 美药业）正式向港交所递交上市申请，携核心药物Mufemilast冲刺18A生物科技IPO（首次公开募股）。 招股书显示，和美药业尚未获得收益，存在显著的"资金饥渴 ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 自身免疫疾病 是由过度活跃的免疫反应导致组织损伤而引发的。目前已有许多疗法通过靶向细胞因子或 B 细胞来治疗自身免疫疾病 （例如，利用 CAR-T 细胞 靶向耗竭 B 细胞来治疗系统性红斑狼疮等自身免疫疾病） ，然而，T 细胞在诸如 1 型糖尿病、肝炎、多发性硬化症和类风湿性关节炎等许多疾病中也发挥着核心 作用。 T 细胞通过细胞毒性、细胞因子释放以及通过辅助性 T 细胞引导其他免疫细胞等方式参与疾病的发生发展。然而，广泛的 T 细胞抑制可能会导致免疫缺陷、癌症 以及机会性感染等风险。 因此，我们需要开发一种新疗法——能够选择性靶向自身反应性或致病性 T 细胞，同时保留有益的 T 细胞功能。 2025 年 6 月 30 日，纽约大学格罗斯曼医学院 王俊 教授 团队联合中国科学院生物物理研究所 娄继忠 研究员 团队、浙江大学医学院 陈伟 教授 团队 （ Jasper Du 、 陈辉 、 由佳 为共同第一作者 ） ，在国际顶尖学术期刊 Cell 发表了题为： Proximity between LAG-3 and the T Cell Receptor Guides Sup ...

三生国健 20250627 摘要 2024 年公司研发投入达 5.4 亿元，因产品临床推进加速，现金储备 33 亿元，资金稳健。聚焦自免领域，管线数量增至 22 个，重点布局高发 病率及临床解决方案不足的自免疾病，目标未来五年内每年至少一到两 个产品提交 NDA 申报。 2025 年是数据读出大年，8 个产品将进入下一阶段，包括 2 个产品推 进到二期临床，5 个产品的相关适应症推进到三期临床，以及 1 个产品 推进到 NDA 状态，保障每年有一到两个产品提交 NDA 申报。 白介素 17A 产品（608）银屑病适应症已提交 NDA，强直性脊柱炎和 放射性阴性脊柱炎适应症将于 2025 年推进到三期临床。有效性方面， 核心指标改善超 90%，且持续缓解率高；安全性方面，Ada 发生率低于 2%。 伊赛普面临招标和指标压力，预计今年三款已上市产品仍将实现个位数 增长。白介素-17 产品预计 2025 年获批，计划 2024 年 Q4 组建销售团 队，竞争优势在于疗效、低不良反应率和 Q8W 给药方案。 Q&A 2024 年公司整体业绩表现如何，主要增长来源和利润构成是怎样的？ 2024 年公司营业收入达到 11 ...

Core Viewpoint - The rising public awareness of autoimmune diseases, particularly ankylosing spondylitis, has led to increased interest in the pharmaceutical company Ganzhou Hemei Pharmaceutical Co., Ltd. as it seeks to go public with its core drug Mufemilast targeting psoriasis and ankylosing spondylitis [1][6]. Company Overview - Ganzhou Hemei Pharmaceutical is preparing for an IPO under the Hong Kong Stock Exchange's 18A biotechnology rules, focusing on high investment and potential returns typical of biopharmaceutical companies [2]. - The company has not yet generated revenue and relies heavily on government subsidies and continuous financing to sustain operations [2][11]. Product Pipeline - The company has seven clinical-stage small molecule candidates aimed at addressing unmet medical needs in autoimmune diseases and oncology [2]. - Mufemilast is a dual-action PDE4 inhibitor that has entered the NDA priority review process, targeting multiple indications including psoriasis and ankylosing spondylitis [2][3]. Market Potential - The market for psoriasis drugs in China has seen rapid growth, with a compound annual growth rate (CAGR) of 30.4% from 2019 to 2023, reaching a market size of 13.9 billion yuan in 2023, projected to soar to 89.4 billion yuan by 2032 [3]. - The autoimmune disease drug market in China has expanded from $2.5 billion in 2019 to $4 billion in 2023, with expectations to reach $26.3 billion by 2032, reflecting a CAGR of 23.3% [6]. Competitive Landscape - The competitive environment for Mufemilast is intense, with existing competitors having undergone significant price reductions due to national procurement policies, with price drops exceeding 93% for some products [5]. - There are currently five approved small molecule targeted therapies for psoriasis in China, with 37 more in clinical development, indicating a crowded market for Mufemilast [5][8]. Financial Status - The company reported significant losses of 156 million yuan in 2023 and 123 million yuan in 2024, with minimal other income [11]. - The company has undergone multiple rounds of financing since 2021, raising over 500 million yuan to support its operations, with a post-investment valuation of 3.9 billion yuan [11]. Leadership - The founder and chairman, Zhang Hesheng, has over 20 years of experience in biopharmaceuticals and holds a significant voting power in the company [12].

编译丨王聪 编辑丨王多鱼 排版丨水成文 促炎细胞因子是炎症性疾病的关键介质，在转录和转录后等多个层面受到严格调控，以避免不必要的炎症反应并维持体内平衡。促炎细胞因子的转录调控在很大 程度上受先天免疫信号通路和转录因子协同激活的调节，其中，翻译后修饰和表观遗传修饰发挥着关键作用。此外，RNA 结合蛋白 （RBP） 通过 RNA 剪接、修 饰、核输出、翻译和降解，在先天免疫反应和炎症的转录后调控中发挥关键作用。然而，多功能 RBP 介导特定促炎细胞因子翻译调控的机制仍需进一步研究，以 全面了解免疫系统的稳态和功能障碍。 泛素 对蛋白质的修饰在稳态调节和免疫反应中发挥着关键作用，包括炎症性疾病。已有数种 E3 泛素连接酶被报道在调节先天免疫反应和炎症方面发挥关键作 用，其机制是通过直接结合并修饰其底物来实现的。然而，对于含有 RNA 结合基序的 E3 泛素连接酶 （E3-RBP） ，它们是否能够结合特定的 mRNA 并在 mRNA-蛋白复合物中发挥 E3 泛素连接酶活性以调控炎症反应，仍需进一步阐明。 2025 年 6 月 16 日 ， 中国医学科学院系统医学研究院 / 苏州系统医学研究所 及 海军军医大学免疫与炎 ...

Core Viewpoint - Huadong Medicine's subsidiary, Zhejiang Zhongmei Huadong Pharmaceutical, received approval for a Phase III clinical trial of 0.3% roflumilast foam (ZORYVE) for seborrheic dermatitis patients, indicating a significant advancement in treatment options for this condition [1][2]. Group 1: Product Development and Clinical Trials - The approved clinical trial targets local treatment for patients aged 9 and above with seborrheic dermatitis, utilizing roflumilast, a PDE4 inhibitor that reduces inflammation [2]. - Roflumilast foam has shown significant efficacy, with nearly 80% of patients achieving complete or near-complete clearance of symptoms by week 8, while maintaining good safety and tolerability [2]. - The product is designed for ease of use, with a once-daily application and no long-term usage restrictions compared to traditional topical corticosteroids [2]. Group 2: Market Potential and Demand - There is a substantial unmet clinical need for effective treatments for seborrheic dermatitis, as existing therapies often have limited efficacy and significant side effects [2]. - The Chinese psoriasis drug market is projected to reach $3.255 billion by 2025, while the global atopic dermatitis treatment market is expected to reach $24.5 billion by 2032, highlighting the market potential for roflumilast [4]. Group 3: Company Strategy and Innovation - Huadong Medicine is focusing on autoimmune diseases, with a comprehensive pipeline that includes over 20 innovative products targeting various conditions, including psoriasis and atopic dermatitis [5]. - The company has established an R&D platform for topical formulations and has built three production lines for external preparations, indicating a strong commitment to innovation in this area [8]. - Huadong Medicine aims to enhance the accessibility of innovative drugs and provide differentiated treatment options, positioning itself for long-term competitive advantage in the autoimmune sector [9].

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 4 月 29 日，西班牙拉巴斯大学医院的研究人员在 Cell 子刊 Med 上发表了题为： CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease 的案例 报道。 该研究使用 CD19 CAR-T 细胞治疗了 1 例 MDA5+ 皮肌炎合并快速进展性间质性肺病的患儿，显示出了有 效性和安全性，治疗至今 11 个月内，患者的病情逐渐好转并实现免疫抑制剂停药缓解。 在这项最新研究中，研究团队介绍了第二代 CD19 CAR-T 细胞疗法首次用于一名患有严重 MDA5+ DM- RPILD 的儿童患者的情况。 在 CAR-T 细胞注射前 34 天 （-34 天） ，常规治疗使 MDA5+ DM-RPILD 患者病情稳定。由于在 B 细胞再 生障碍的情况下 CAR-T 细胞扩增，怀疑深层组织中存在可能作为靶细胞的 CD19+ B 淋巴细胞。未出现发热 或细胞因子释放综合征/细胞 ...

Core Viewpoint - Sanofi's strategic shift towards focusing on the autoimmune sector has led to significant revenue and profit growth, despite challenges in the competitive landscape and pricing pressures from centralized procurement policies [1][4][7]. Financial Performance - In 2024, Sanofi achieved revenue of 1.194 billion yuan, a year-on-year increase of 17.70%, and a net profit attributable to shareholders of 705 million yuan, up 139.15% [1]. - The company reported a non-recurring net profit of 246 million yuan, reflecting an 18.99% increase year-on-year [1]. - Cash flow from operating activities showed a net inflow of 281 million yuan, a decrease of 32.48% compared to the previous year [2]. Product Portfolio and Market Position - Sanofi currently has three therapeutic antibody drugs on the market, with Yisaipu being the core product, alongside Jianipai and Saiputing [3]. - The market share of Yisaipu has declined from 40.14% in 2016 to 27.00% in 2022 due to increased competition [3]. - The company has faced pricing pressures due to the expansion of centralized procurement policies, with Yisaipu's average price decreasing as it entered over 20 provincial procurement programs in 2024 [3][4]. Strategic Focus and Business Model - Sanofi has divested its oncology pipeline to concentrate on the autoimmune sector, which is seen as a strategic move to enhance its core competencies and improve financial performance [5][6][7]. - The company has signed multiple agreements to transfer oncology and ophthalmology projects to its affiliate, Shenyang Sanofi, to reduce R&D costs and focus on autoimmune drug development [6][7]. - The autoimmune disease market is projected to reach $6.1 billion by 2025, with a compound annual growth rate (CAGR) of over 20% [9]. Industry Trends and Opportunities - The autoimmune disease market is characterized by a growing patient population and a significant unmet clinical need, with only about 30 treatment options currently available globally [8][9]. - The market for biological agents in autoimmune diseases is expected to grow from 12 billion dollars in 2022 to 138 billion dollars by 2030, indicating a robust opportunity for innovation and development in this sector [9].