Comirnaty-COVID-19 mRNA vaccine

近日，国际知名学术期刊《Signal Transduction and Targeted Therapy》（IF：81.2）发表了一篇题为"RNA therapeutics: current status and future directions"的重磅综述：系统回顾了RNA疗法从基础发现到临床应用的完整发展历程，全面剖析了反义寡核苷酸、RNA干扰、核酸适配体、信使RNA以及CRISPR/Cas基因编辑五大RNA药物类别的设计原理、优化策略、递送系统和FDA获批进展，并对该领域的未来挑战与突破方向进行了前瞻性展望。 RNA疗法的发展时间线 颠覆传统的治疗范式：RNA药物为何与众不同？ 临床与临床前研究阶段的各类RNA治疗药物概览 与传统小分子药物主要靶向蛋白质不同，RNA疗法通过沃森-克里克碱基互补配对直接在核酸层面调控基因功能，或通过其二级结构特异性结合蛋白质靶标，从而开辟了全新的治疗维度。RNA不仅是蛋白质的编码模板，更是庞大而精密的调控网络的核心成员，包括微小RNA、长链非编码RNA和环状RNA等非编码RNA种类，它们在基因表达调控中发挥着不可或缺的作用。 新冠疫情无疑是RNA疗法发展的里程碑事件。2020年，辉瑞-BioNTech的BNT162b2和莫德纳的mRNA-1273两款mRNA疫苗在紧急使用授权下迅速问世，不仅有效应对了全球大流行，更极大地提振了业界对RNA疗法潜力的信心，标志着数十年跨学科创新成果的集成落地。 五类药物类别齐头并进 近期获FDA批准的RNA药物作用机制概览 反义寡核苷酸：从基因沉默到剪接调控 ASO是最早获得FDA批准的RNA药物类别。自1998年首个ASO药物福米韦森获批以来，目前已有约十余款ASO药物上市。 ASO主要通过两种机制发挥作用：RNase H依赖性ASO通过招募内源性核酸酶RNase H降解靶标mRNA，适用于治疗基因突变导致的毒性获得或病毒RNA感染；而空间位阻型ASO则不依赖RNase H降解，通过调控前体mRNA的可变剪接来实现治疗效果。 诺西那生钠是剪接调控型ASO的经典案例。此外，依特立生、戈洛迪生、维托拉生和卡西梅生等PMO类ASO药物则通过跳跃特定外显子，恢复杜氏肌营养不良症患者的抗肌萎缩蛋白阅读框。 RNA干扰：精准基因沉默的双刃剑 RNAi疗法利用小干扰RNA或微小RNA模拟物，通过Argonaute蛋白介导的RISC复合物实现序列特异性的基因沉默。 2018年，帕提西兰成为首款获批的siRNA药物，用于治疗转甲状腺素蛋白淀粉样变性。 截至目前，共有八款siRNA药物获得FDA批准。值得注意的是，大多数获批siRNA药物都采用了GalNAc偶联技术，实现了高效的肝脏靶向递送，大幅降低了给药剂量和频率。 核酸适配体：化学抗体的崛起 核酸适配体是能够通过三维构象互补性选择性结合靶标的单链寡核苷酸，其功能类似于治疗性单克隆抗体，但具有化学合成、低免疫原性和高热稳定性等独特优势。 目前仅有两款适配体药物获批：2004年的培加他尼钠和2023年的阿伐西卡普塔德聚乙二醇，均用于眼科疾病治疗。 文章指出，先进的筛选技术如细胞SELEX和体内SELEX正在推动适配体靶向复杂生物分子的能力 。 信使RNA：超越疫苗的广阔天地 mRNA疗法不仅限于疫苗应用。在蛋白质替代治疗方面，编码鸟氨酸氨甲酰转移酶的ARCT-810和编码甲基丙二酰辅酶A变位酶的mRNA-3705均已进入临床试验阶段。在肿瘤免疫治疗方面，个体化新抗原mRNA疫苗mRNA-4157联合帕博利珠单抗在黑色素瘤三期临床试验中展现出令人鼓舞的临床结果。 2024年，莫德纳的RSV mRNA疫苗mRNA-1345获批用于60岁以上成人，并在2025年扩展至18至59岁高风险人群，进一步验证了mRNA平台的通用性。 CRISPR/Cas：基因编辑的革命性工具 2023年，首款CRISPR疗法Casgevy获批，标志着基因编辑时代的到来。该系统利用单链引导RNA将Cas核酸酶引导至目标基因组位点，通过双链断裂修复实现基因敲除或敲入。 文章详细介绍了Cas9切口酶在碱基编辑中的应用——通过融合胞苷脱氨酶或腺苷脱氨酶实现C到T或A到G的精准转换，以及先导编辑技术——利用Cas9切口酶与逆转录酶的融合，在不产生双链断裂的情况下完成所有类型的DNA编辑。催化失活的dCas9则被重新用于基因表达调控，通过融合转录激活或抑制结构域，为治疗基因表达失调相关疾病提供了全新策略。 化学修饰与递送系统：RNA疗法的两大支柱 用于稳定RNA类药物的常见化学修饰 文章强调，化学修饰是提升RNA药物稳定性和疗效的关键。 磷酸硫代修饰可显著提高核酸酶抗性和血浆蛋白结合能力；2'-氟、2'-O-甲基和2'-O-甲氧乙基等核糖C2位修饰可增强杂交亲和力和代谢稳定性；锁核酸和约束乙基修饰则进一步提高了熔解温度和靶标亲和力。 在递送系统方面，脂质纳米颗粒已成为mRNA疫苗和部分siRNA药物的标准载体。GalNAc偶联技术则实现了高效的肝脏靶向递送。 此外，细胞外囊泡、聚合物纳米颗粒和抗体偶联等新型递送策略也在积极探索之中。 RNA疗法的常用递送系统 挑战与未来展望 尽管取得了长足进步，RNA疗法仍面临多重挑战。核酸酶降解导致的稳定性问题、靶向递送效率不足、免疫原性反应以及大规模生产成本高昂等问题亟待解决。 此外，miRNA模拟物的脱靶效应、CRISPR编辑的脱靶切割以及长链非编码RNA和环状RNA等新兴RNA类别的治疗开发仍处于早期阶段。RNA疗法已经从一个充满希望的科研概念成长为临床现实，随着我们对RNA生物学理解的不断深化，以及化学、递送和制造技术的持续创新，RNA疗法有望在未来为更多难治性疾病提供革命性的治疗方案。 原文链接： https://www.nature.com/articles/s41392-026-03032-5 【关于投稿】 转化医学网（360zhyx.com）是转化医学核心门户，旨在推动基础研究、临床诊疗和产业的发展，核心内容涵盖组学、检验、免疫、肿瘤、心血管、糖尿病等。如您有最新的研究内容发表，欢迎联系我们进行免费报道（公众号菜单栏-在线客服联系），我们的理念：内容创造价值，转化铸就未来！ 转化医学网（360zhyx.com）发布的文章旨在介绍前沿医学研究进展，不能作为治疗方案使用；如需获得健康指导，请至正规医院就诊。 责任声明：本稿件如有错误之处，敬请联系转化医学网客服进行修改事宜！ 微信号：zhuanhuayixue

2026年12月2-3日 中国北京-昌平       近年来 ATMP2019-2026 汇聚了来自美国宾夕法尼亚大学佩雷尔曼医学院、帕克癌症免疫疗法研究所、纪念斯隆-凯特琳癌症中心、MD 安德森癌症中心、美国费城儿童医院、哈佛大学医学院附属麻省总医院、加州大学圣地亚哥分校、美国国立癌症研究所、美国国立卫生研究院、英国伦敦大学圣乔治医院、新加坡科技研究局、日本京都大学iPS 细胞研究所、美国食品药品监督管理局以及德国埃尔朗根-纽伦堡大学等众多国际先进疗法先驱，中国顶尖学府、科研院所以及知名产业界嘉宾的共同参与。由迪易生命科学主办的第十二届先进疗法创新峰会(ATMP 2026)将于12月2-3日在北京龙城温德姆酒店召开。 会议时间：2026年12月2-3日 会议地点：北京-昌平-龙城温德姆酒店 会议规模：约800人 筹办单位：迪易生命科学 Deliver Life Sciences 部分主要议题： • FMC63与新一代分泌IL-18”装甲” CD-19 CAR-T细胞产品的研究进展 • 生物标志物赋能的“泛血液肿瘤” CAR-T细胞治疗‘精准分层’ • “818” 号令下细胞与基因治疗新技术临床研究与转化应用 • CAR-T 细胞治疗国内外产业发展, 监管科学, 临床转化与未来展望 • In-vivo X体内细胞治疗的研发进展与前沿技术创新 ( mRNA-LNP, 双靶点, 慢病毒, 环状RNA ) • 个性化mRNA肿瘤疫苗, 环状RNA疗法, 个性化新抗原疫苗, AI赋能先进疗法创新 • 细胞免疫治疗产品开发, 前沿技术创新与CMC关键点考量 ( CAR-T, CAR-NK, TCR-T, TIL, Treg ) • 再生医学与干细胞疗法前沿 [  人胚干细胞（hESC）vs间充质干细胞（MSC）vs诱导多能干细胞（iPSC）] • AAV基因治疗罕见病, 眼科疾病与神经退行性疾病以及基因编辑技术创新 • 类器官与器官芯片, 神经科学与脑类器官研究前沿 • 合成生物学与生物制造前沿 • 外泌体-细胞外囊泡与先进疗法前沿 扫码注册 James L. Riley 微生物学教授 宾夕法尼亚大学佩雷尔曼医学院 演讲主题: Fully human CD19 CAR T cell armored to express IL-18 Abstract & Bullet Points:  Patients can develop human anti-mouse immune responses against CD19-specific chimeric antigen receptor (CAR) T cells due to the use of a murine CD19-specific single-chain variable fragment to redirect T cells. We screened a yeast display library to identify an array of fully human CD19 single-chain variable fragment binders and performed a series of studies to select the most promising fully human CAR. We observed significant differences in the ability of CARs employing these CD19 binders to be expressed on the cell surface, induce tonic signaling, redirect T-cell function, mediate tumor killing, recognize lower levels of CD19 antigen, and maintain function upon continuous antigen exposure. From this initial analysis, CAR T cells using two binders (42 and 52) were selected for additional studies. Although CAR T cells using both binders controlled tumor growth well in vivo, we advanced a CAR construct using binder 42 for more advanced preclinical testing because of its greater similarity to binders based on the antibody FMC63, which is the murine antibody underlying four FDA-approved CD19-specific CAR T-cell therapies, and ability to robustly respond to tumors expressing lower levels of CD19. We found that this binder uniquely bound CD19 using distinct contact residues than FMC63 and with ∼40-fold lower affinity. CARs using binder 42 were non-inferior to those using the FMC63 binder in a mouse model of acute lymphoblastic leukemia .   Co-expressing IL-18 with this CAR greatly augmented activity and the mechanism of this help will be discussed. Joseph A. Fraietta 微生物学副教授 细胞免疫治疗中心--转化与相关科学负责人 宾夕法尼亚大学佩雷尔曼医学院 演讲主题: CAR T Cells: A Practical Guide to Biology, Safety, and Success Speaker Bio:  Dr. Joseph Fraietta graduated in 2007 from Drexel University with a degree in Bioscience and Biotechnology. His studies continued at Drexel University College of Medicine, where he received a Ph.D. in Microbiology and Immunology in 2012. His graduate work focused on the interrogation of the signaling and molecular requirements for the generation of successful effector and memory T cells and determining why these cells fail to control certain chronic viral infections. As a post-doctoral fellow under the tutelage of Dr. Carl June at the University of Pennsylvania, Dr. Fraietta developed novel approaches for the treatment of cancer through genetic modification of T lymphocytes that contributed to the initiation of multiple clinical trials and U.S. Food and Drug Administration (FDA) approval of the first CAR T cell therapy. In 2015, Dr. Fraietta assumed the directorship of a research laboratory in the first-of-its-kind Center for Advanced Cellular Therapies where his group led initiatives to interrogate CAR T cell infusion products for key biomarkers and mechanisms of potency, with the objective of predicting clinical responses to adoptive cell therapies. He now directs the Solid Tumor Immunotherapy Laboratory in the same center. In 2019, Dr. Fraietta joined the tenure-track faculty as is current in the Perelman School of Medicine Department of Microbiology. His high-impact findings have been published in Nature, Nature Medicine, Nature Immunology and Science Translational Medicine. Dr. Fraietta is the recipient of numerous awards, including a Microbiology and Immunology Distinguished Alumni Award and a 2019 National Clinical Research Award from the Clinical Research Forum, which recognizes the ten most outstanding clinical research accomplishments in the U.S. during the preceding twelve months. Dr. Fraietta was recently invited to Capitol Hill in Washington, DC where he spoke to congressional leaders about the importance of translational research and the need for continued funding. 沈 琳 消化肿瘤内科主任、I期临床病区主任 北京大学肿瘤医院 实体瘤细胞与基因治疗北京市重点实验室主任 演讲主题: 细胞基因治疗在消化道肿瘤中的研究突破与问题 Speaker Bio:  沈琳，北京大学肿瘤医院主任医师、教授、博士生导师，北京学者，消化肿瘤内科主任、I期临床病区主任，实体瘤细胞与基因治疗北京市重点实验室主任。担任CACA肿瘤精准治疗专委会、CSCO胃癌专委会主任委员、中国女医师协会副会长等多个学术职务。 致力于消化道肿瘤精准治疗与转化研究，以及抗肿瘤新药临床研究。带领团队建立消化道肿瘤临床与转化研究创新体系；作为Leading PI，主导百余项国际国内临床研究，促进新药获批适应症16项，推动多个中国原创新药走向国际舞台，成果改变国内外临床实践指南40多项；推动38个药物进入 I - II 期临床研究。以一作/通讯在 Nature、BMJ、JAMA、Nature Medicine等权威期刊发表 SCI 论文 238 篇，应邀为 Nature Medicine 介绍中国肿瘤诊疗研究现状与未来发展，入选全球高被引科学家、全球前 2% 顶尖科学家。撰写 54 部国际国内诊疗规范；获国家专利 18 项，实现科技成果转化金额达1260万。获中华医学科技奖一等奖、中国抗癌协会科技奖一等奖等多项奖项。 George Eastwood 执行主任 Emily Whitehead 基金会 演讲主题: Empowering Patients, Delivering Access Speaker Bio:  George Eastwood is the Executive Director of the Emily Whitehead Foundation, an organization created by the Whitehead family after their daughter Emily became the first pediatric patient to receive CAR-T cell therapy. Under his leadership, the foundation is focused on expanding access to advanced therapies, influencing policy to remove barriers to care, and supporting patients and families throughout their treatment journey, work that builds on its original mission of funding groundbreaking research for less-toxic cancer treatments. A mission-driven innovator in cell and gene therapy, George has spent much of his career creating products, tools, and services to accelerate the development and delivery of cell-based therapeutics. As an early employee at HemaCare, he partnered with pioneering CAR-T companies like Kite and Novartis to help bring their therapies from development to commercialization. He later held leadership roles in the start-up space, including Vice President of Business Development and Partnerships at Kytopen and co-founder of Excellos, a cell and gene therapy company spun out of the San Diego Blood Bank. In addition to his role at the Emily Whitehead Foundation, George serves on the Board of Directors for the Alliance for Regenerative Medicine, where he works alongside global leaders to advance policies, standards, and innovations that ensure more patients can benefit from next-generation therapies. Daniel Getts 创始人兼首席执行官 CREATE Medicines 演讲主题: Barriers and Strategies for Expanding Access to CAR T-Cell Therapy Speaker Bio:  Daniel Getts is a global leader in RNA therapeutics and immune engineering. He is the founder and CEO of CREATE Medicines, a clinical-stage biotech pioneering in vivo mRNA immune cell programming. Throughout his career, Getts has founded or co-founded several biotechnology companies, including Cour Pharmaceuticals, the field leader in immune tolerance, striking major partnerships with Takeda ($420M) and Genentech ($940M). Collectively, his ventures have generated more than $2 billion USD in value and advanced 11 novel therapies into clinical trials across cancer, autoimmune disease, and transplant rejection. Born and raised in a working-class family in southern Sydney, Getts earned his PhD in Medicine from the University of Sydney and his MBA from Western Michigan University. He was a research professor at Northwestern University, where he co-invented multiple immune modulation platforms. Dr. Getts serves as an advisor to governments and health agencies in the US, Europe, and Asia, and has spoken at international forums including the World Economic Forum, World Government Summit, and the BIO International Convention. He participates in international policy and industry think tanks shaping policy in biotechnology, health security, and advanced manufacturing. Getts holds over 200 patents and has published extensively in top peer-reviewed journals, including Nature Biotechnology and Science Translational Medicine. 刘 诚 创始人兼首席执行官 优瑞科生物 演讲主题: From Liver Cancer to Broad Solid Tumor Applications: ARTEMIS CAR-T Clinical Validations and Opportunities Across Cancer Types and Combination Strategies Speaker Bio:  刘博士是优瑞科生物技术公司（“优瑞科”）（一家专注于创新型细胞基础癌症免疫疗法的公司）的创始人、首席执行官兼董事，药明巨诺（港交所代码：2126）公司董事会主席，Estrella Immunopharma（一家在纳斯达克上市的公司（纳斯达克：ESLA））的首席执行官兼董事，以及InvisiShield Technologies Ltd.的董事。刘博士是生物技术领域享誉全球的杰出科学家，以T细胞工程和免疫治疗方面的开创性研究闻名，专注于开发颠覆性的创新癌症疗法。在创立优瑞科之前，刘博士曾在Chiron Corporation（现已被诺华收购）担任抗体药物研发的首席科学家。凭借在该领域逾二十年的专业经验，刘博士拥有超过300多项专利和已公开的专利申请。刘博士在全球CAR-T专利排名中位居前列，且在CAR-T疗法领域占据着突出位置。根据《自然·生物技术》杂志（2020年第38卷）统计，他在全球发明家中排名第14位，拥有169项专利。 刘博士始终站在全球医学科学的前沿，致力于推进癌症治疗技术的创新与发展。他不仅专注于实验室研究成果，还积极推动这些技术的临床转化，以造福更多患者。他的工作不仅改变了癌症治疗的格局，还为全球医学科学进步做出了重大贡献。他发明了ARTEMIS T细胞工程平台，这是一项颠覆性的T细胞癌症治疗前沿技术。此外，他还是多个首创类临床阶段抗癌药物的发明者，针对各种肿瘤靶点，包括针对治疗癌症骨转移的CSF1、针对多发性骨髓瘤的BCMA以及针对肝癌的AFP及GPC3等药物。刘博士于2007年就其改善人类健康方面的贡献获得美国国会特别嘉许（Special US Congressional Recognition）。刘博士为《Biosimilars of Monoclonal Antibodies：A Practical Guide to Manufacturing, Preclinical, and Clinical Development》一书的编辑。刘博士于1988年7月取得中国北京大学生物学及遗传学的学士学位，于1996年5月取得美国加州大学伯克利分校分子细胞生物学博士学位。 Peggy Sotiropoulou 首席科学官 T-knife Therapeutics 演讲主题: Supercharged T cell therapies to combat resistance and enhance durability in Solid Tumors Abstract & Bullet Points:  • Designing multi-armored T cell therapies to enhance durability of responses in solid tumors. • Evaluating T cell therapy efficacy using advanced solid tumor models. •Understanding the specific challenges of solid tumors for in vivo CAR-T therapy. • Balancing armored CAR and TCR designs to optimize long-term efficacy while minimizing safety risks . 黄纲雄 创始人兼首席执行官 拓新天成 演讲主题: B7H3 靶向CAR-T 治疗恶性肿瘤 Speaker Bio:  2005在MD安德森癌症中心博士后工作期间，开始应用转座子技术进行CAR-T细胞和NK细胞治疗肿瘤的研究 2018年开始在国内研究开发了创新性的CAR-T细胞用于治疗多种实体瘤, 获得美国FDA/中国NMPA批准，进入注册临床试验。 汪 文 创始人兼首席执行官 天宜康医药 演讲主题: Strategy of Design and Develop Bi-specific CAR T-cell Therapy Abstract & Bullet Points:  1.Unmet medical needs of single targeting CAR-T therapy 2.Bi-CARs might be effective way to solve unmet medical needs 3.Optimization of Bi-CAR constructs 1)Ligand-binding domain 2)Linker domain 3)Spacer domain 4)Transmembrane domain 5)Cytoplasmic domain 4.CD19/CD20 4th generation fully human fast CAR-T 5.CD7/BCMA LNP-KO allogeneic CAR-T 6.LVV based dual targeting 4th generation fully human in vivo CAR-T 7.DNA-LNP based dual targeting 4th generation fully human in vivo CAR-T 齐菲菲 联合创始人兼首席技术官 艺妙生物 演讲主题: Comparability and the management of manufacturing changes for cellular therapy products Abstract & Bullet Points:  Manufacturing changes are inevitable throughout the lifecycle of cellular therapy products, driven by process optimization, scale-up requirements, and raw material evolution. Unlike traditional biologics, cellular therapies are complex, living drugs whose critical quality attributes are deeply intertwined with their manufacturing process. Ensuring product safety, purity, and potency before and after a change requires a robust regulatory framework and rigorous comparability exercises. Common drivers and types of manufacturing changes in cellular therapy are examined, alongside an analysis of evolving global regulatory expectations and the strategic implementation of non-clinical, analytical, and clinical comparability protocols to maintain product continuity and patient safety. 左 为 创始人、董事会主席 吉美瑞生 演讲主题: 肾脏干细胞新药研究进展与临床转化 Speaker Bio: 左 为，同济大学医学院长聘教授，吉美瑞生 (Regend Therapeutics Limited) 创始人、董事会主席，中国细胞生物学学会细胞与基因治疗分会副主任委员、党组书记，国家重点研发计划“干细胞及转化研究”首席科学家，上海市青年联合会常务委员，浙江省江南干细胞研究院院长。入选国家教育部长江学者奖励计划及中组部高层次人才计划青年项目，获国自然重点项目和国自然优秀青年基金资助。先后荣获2026年联合国WIPO知识产权全球奖、2025年山东省科技进步一等奖，以及“中国年度十大创新先锋”、“中国生命科学新力量年度人物”等荣誉称号。 带领团队深入开展基于成体干细胞的肺、肾等重要脏器的再生修复、功能增强与青春恢复研究。主导完成世界上首个肺干细胞移植再生肺脏临床研究。论文成果发表在Nature、Science Translational Medicine、Nature Communications、Science Advances、Cell Reports Med、eLife、EMBO Mol Med、AJRCCM等国际期刊上, 单篇通讯论文被引超2000次。领导开发了全球首创肺干细胞1类新药Arieautotemcel (艾瑞奥泰赛)和肾干细胞新药REGEND003，获3项国家药监局临床批件及1项美国FDA孤儿药认证，并推进肺脏再生医学产品进入三期临床阶段。研究成果先后入选“中国医药生物技术年度十大进展”和欧洲呼吸学会(ERS)发布的全球年度三大重要进展，登上《科学》期刊官网首页。2025年成果获批海南博鳌首批干细胞治疗技术应用，累计成功实现数百例患者的器官再生修复治疗。 周国庆 首席执行官 荣瑞医药 演讲主题: In Vivo CAR-Tumor, New Frontier of Cell Therapy against Cancer Abstract & Bullet Points:  Cancer is a major disaster in human society. We use genetically edited Vesicular Stomatitis Virus (VSV) as a precise tumor-targeting delivery vector to deliver chimeric antigen receptors (CAR), such as anti-CD3, into the cytoplasm of tumor cells for expression. Through intracellular transport, they are anchored on the outer membrane of tumor cells, binding to ligands outside the T cell membrane, such as CD3, to activate T cells to attack and kill tumor cells. This is the basic principle of In Vivo Chimeric Antigen Receptor Tumor Cell Therapy, namely In Vivo CAR-Tumor. It reprograms cancer cells into "drug factories", transforming cancer tissues in situ into immune-sensitive activation sites, recruiting and activating the body's own T cells to attack and achieve precise and continuous killing of cancer cells. This VSV-based In Vivo CAR-Tumor technology enables tumors to "commit suicide", effectively solving the core problems of limited efficacy and high cost in traditional CAR-T treatment of solid tumors, revolutionizing immune cell therapy and pioneering a brand-new field of In Vivo CAR-Tumor, providing new hope for cancer patients worldwide. 郭 磊 创始人兼首席科学官 普略医学/北京百替生物 演讲主题: Beyond Viruses and LNPs: AI-Designed Synthetic Transfer Vehicles (STVs) for Programmable In Vivo Immune Cell Engineering Abstract & Bullet Points:  • From “CAR delivery” to programmable in vivo immune-cell engineering: precision entry, transient versus durable programming, functional enhancement and disease-specific design. • STV as a third delivery paradigm: AI-designed synthetic protein assemblies, programmable tropism and modular RNA cargo delivery beyond conventional viral vectors and LNPs. • Translational opportunity for in vivo CAR/TCE engineering: combining targeted RNA delivery with CAR/TCR payloads, CRISPR-based functional editing and multi-component immune programming. • Development roadmap: payload–carrier co-design, cell-type specificity, biodistribution, repeat dosing, safety, manufacturability and clinically meaningful disease models as key gates from platform concept to therapeutic product. 王书航 廊坊院区GCP中心 副主任 中国医学科学院肿瘤医院 演讲主题: Clinical Translation of In Vivo CAR-T Therapy: Progress and Perspectives Abstract & Bullet Points:  In vivo CAR-T aims to generate CAR-expressing immune cells directly in patients by delivering genetic payloads in situ, potentially bypassing leukapheresis, ex vivo manufacturing, and reinfusion required for conventional autologous CAR-T. This presentation will review current technological and clinical development trends, focusing on key translational issues from delivery platform and target/indication selection to early-phase trial design and risk management, and will discuss opportunities, challenges, and future development paths in oncology and other disease settings. - Translational features of major platforms: targeted LNP/mRNA and viral-vector approaches, with their respective strengths and limitations. - Target and indication selection: differentiated considerations for hematologic malignancies, solid tumors, and immune-mediated diseases. - Clinical development: in vivo transduction efficiency and cell specificity, dose exploration, biodistribution/persistence, pharmacodynamic biomarkers, and long-term follow-up. - Safety and risk management: cytokine-related toxicities, unintended cell transduction, off-target risks, immunogenicity, and long-term risks associated with integrating vectors. - CMC and regulatory considerations: evolving quality-control and clinical-evaluation frameworks as the modality shifts from an ex vivo cell product toward an in vivo gene/cell therapy. - Future directions: controllable/reversible expression, repeat dosing, combination strategies, and improved accessibility 梁霏霏 首席技术官 华夏英泰 演讲主题: 从工程化细胞到工程化体内：STAR-T in vivo的设计逻辑与临床转化 Speaker Bio:  华夏英泰首席技术官。曾任博雅辑因研发高级副总裁，主导多项异体CAR-T在血液瘤、实体瘤与自免适应症至IIT；此前任传奇生物细胞工艺开发负责人，参与多项血液瘤及实体瘤自体CAR-T管线的IIT，其中2项后获美国IND批准；早期于法国Svar Life Science工作，巴黎十一大/CNRS细胞学、肿瘤学博士。 张亚晶 肿瘤与免疫创新医学中心 主任 北京高博博仁医院 演讲主题: New Regimens and Novel Strategies for CAR-T Cell Therapy in Hematologic Malignancies and Autoimmune Diseases Speaker Bio:  主要研究方向： 细胞与免疫治疗在肿瘤及自身免疫性等疾病中的临床应用与转化研究。 科研成果： 以第一负责人主持国际合作项目、国家自然科学基金和省部级课题等7 项； 以第一作者在《Blood》、《Leukemia》、《JEM》、《STTT》等杂志发表多篇SCI论文，累积影响因子150余分； 获得《JEM》2023年度优秀论文奖； 南粤科技创新优秀论文一等奖 解放军总医院科技进步二等奖 学术任职： 中国研究型医院学会 生物治疗专业委员会 常务委员 中国癌症基金会 血液肿瘤康复核心专家 中国人体健康科技促进会中枢淋巴瘤多学科诊疗专业委员会  常务委员 中国医药教育协会 基础与临床研究促进工作委员会 常务委员 中国医药生物技术协会皮肤软组织修复与重建技术分会第三届委员会 常务委员 中国临床肿瘤学会(CSCO)骨髓瘤专家委员会 委员 《JCO-Blood中文版细胞与免疫治疗》专刊编委会 委员 中国人体健康科技促进会 细胞免疫治疗专业委员会 委员 中国血液病专科联盟 淋巴瘤/骨髓瘤自体移植与细胞治疗协作组 成员 中国医学科学院 血液病医院 研究员 北京抗癌协会淋巴血液肿瘤专业委员会 常务委员 天津市抗癌协会血液肿瘤专业委员会第三届委员会委员 宋相容 董事长, 总经理, 科学创始人 威斯津生物 演讲主题: From Cancer Vaccines to In Vivo CAR-T: Expanding the Therapeutic Potential of mRNA-LNP Technologies Abstract & Bullet Points:  • Innovation in mRNA and LNP technologies • Development of mRNA cancer vaccines •Targeted LNP delivery for in vivo CAR-T •Expanding applications of mRNA-LNP therapeutics 孙静玮 副总裁兼研发负责人 沙砾生物 演讲主题: From TIL Therapy to Neoantigen Vaccines: Ushering in a New Era of Precision Immunotherapy Abstract & Bullet Points:  • NEOvigator® identifies functional, clinically relevant neoantigens. • APC-targeted LNPs enable potent, durable immunity with minimal liver off-targeting. •GT601 shows promising clinical activity and favorable safety in ongoing trials. 回爱民 创始人, 董事长兼首席执行官 惠正奇医药 演讲主题: mRNA个性化肿瘤疫苗的全球突破对中国产业的战略启示 Speaker Bio:  回爱民博士是惠正奇医药的创始人，董事长兼CEO；呼吸疾病全国重点实验室产业教授，中国生物医药产业链创新转化联合体副理事长。 回博士是前复星医药执行总裁，科学委员会主任，全球研发总裁兼CMO，复星全球合伙人。曾任赛诺菲全球副总裁，上海市干细胞治疗重点实验室主任，中国药促会肿瘤委员会副主任,第5届中国肿瘤大会(CACA)学术委员会副主任。从事分子生物学，肿瘤临床诊疗，转化医学及药物/疫苗研发30年，直接领导了Ixazomib，Isatuximab等多个小分子及生物抗癌药的全球研发及上市，并主导了伊莎唑咪，阿发曲珀帕等多款创新药在中国的研发，上市。2013年首创“全球三期临床中国延展性试验”的临床注册模式，开辟了全球三期临床数据直接在中国注册上市的先河，大大缩短了创新药物在中国的上市时间。2017-2022年主导了复星医药研发转型，深化与国际化，实现公司历史上多个零的突破。2020年初新冠大流行伊始，与德国BioNTech合作，深度参与了人类历史上第一款mRNA产品（mRNA新冠疫苗）的研发，并主导了该疫苗（复必泰）在大中华区的研发及港澳台上市，带动了我国mRNA产业发展。曾获中国第四届转化医学-国际合作奖，国际消化道癌症青年科学家奖等国内外奖20余。 在国际顶级期刊包括新英格兰医学杂志，自然医学，柳叶刀，细胞癌症等发表论文100余篇。主编，参编中、英、日文专著8部。 栗世铀 联合创始人兼首席技术官 启辰生生物 演讲主题: Survivin-Targeted mRNA Platform for Therapeutic Cancer Vaccines Abstract & Bullet Points:  Survivin (BIRC5) is a shared tumor antigen broadly expressed across multiple malignancies and has emerged as a clinically investigated target for therapeutic cancer vaccination. Internationally, Survivin-targeted vaccines, including the peptide-based SurVaxM, have progressed to a fully enrolled Phase 2b SURVIVE trial in newly diagnosed glioblastoma (GBM), which reached its data cutoff on July 15, 2026, with statistical analysis underway. In China, Survivin mRNA-loaded dendritic cell (DC) vaccines have advanced to Phase II clinical evaluation in GBM, further supporting the translational potential of Survivin-targeted immunotherapy. mRNA-based antigen delivery may enable full-length Survivin expression and broader epitope generation through MHC-I and MHC-II pathways, potentially reducing HLA restrictions and supporting the development of therapeutic vaccines for GBM and other solid tumors, including postoperative MRD, recurrence prevention, and combination immunotherapy. 杨海涛 创始人兼首席执行官 瑞美奥纳 演讲主题: Development of AI-Powered Large RNA Models and Tumor mRNA Vaccines Abstract & Bullet Points:  AI-powered large RNA foundation models have greatly promoted the rational design and clinical translation of personalized tumor mRNA vaccines, overcoming the defects of traditional vaccine development. - We innovatively replaced the traditional Transformer backbone with a high-performance Mamba architecture, effectively enhancing the feature extraction ability, computational efficiency and neoantigen prediction accuracy of our oncology-oriented AI RNA model.- Focusing on refractory breast cancer and pancreatic cancer, our team develops targeted mRNA vaccine candidates for precise tumor immunotherapy. - To further optimize model performance and adapt to complex clinical tumor heterogeneity, we plan to collect spatial omics data from a cohort of 1000 cancer patients. Integrating high-resolution spatial transcriptomics and tumor microenvironment profiles enables the model to accurately screen high-immunogenicity neoantigens and optimize mRNA sequence design. 戴东升 创始人, 董事长兼总经理 优环生物 演讲主题: New CircRNA Platforms & New Drugs Development Abstract & Bullet Points: -- Platform IP · Linear Scalable Process · GMP Two independent platforms: Scar and scarless, supporting coding and non‑coding RNA, with linearly‑scalable manufacturing up to the kilogram scale based on affinity chromatography -- Multi‑batch · Multi‑site · Successful GMP‑grade Manufacturing Across 3 projects and 2 manufacturing sites, we completed 4 engineering batches and 3 GMP batches. All batches achieved successful process validation and quality release, validating the platform’s robust industrialization capability -- Industry & Academia‑wide Technical Recognition The TIE platform, successfully licensed‑out to an MNC for T‑cell therapeutics, maintains deep collaborations with dozens of top‑tier industrial and academic teams domestically and internationally --  Professional · Specialized · Comprehensive Team Dr.  Dai:  30+ years full‑cycle biopharma veteran, collaborating with USTC distinguished young scholar Prof. Shan on biopharmaceutical R&D 胡荣宽 创始人兼首席执行官 星锐生物 圆桌讨论: In-vivo CAR-T：从技术突破到产业规模化发展思考 Speaker Bio: 胡荣宽博士，星锐医药创始人、首席执行官，正高级工程师，中国科大生物医学工程学院、苏州大学药学院产业教授。长期从事RNA药物和疫苗的开发，领导了多个RNA项目进入临床阶段，申请专利和发表SCI论文60余篇。获得江苏省“双创人才”、江苏省333高层次人才、苏州市“姑苏创新创业领军人才”等称号。他在中国科大获得博士学位，中欧国际工商学院EMBA，并曾于德克萨斯大学西南医学中心从事博士后研究。 苗振伟 创始人兼首席执行官 英百瑞生物 演讲主题: New Paradigms and New Opportunities of Off the Shelf Universal NK Cells in Clinical Tumor Therapy Abstract & Bullet Points:  Traditional autologous cell therapies are restricted by long manufacturing cycles, high cost and poor scalability, which hinders their wide clinical access for cancer patients. As allogeneic, off the shelf products, universal NK cell therapies bring a transformative new paradigm for tumor immunotherapy, featuring HLA matching free administration, GMP based mass production, instant availability and favorable safety profile. With unique MHC unrestricted anti tumor killing capacity, NK cells can overcome major bottlenecks of T cell based immunotherapy against solid tumors and show strong synergistic potential with chemo therapy, targeted agents and immune checkpoint inhibitors. Based on Imbioray’s R&D pipeline progress including ACC NK and tiNK immune primed NK cell programs, this presentation discusses key technical strategies, clinical translation challenges, industrialization pathways and promising clinical opportunities of off the shelf NK therapies, paving the way for next generation accessible cancer immunotherapies. 刘明宇 研发负责人 百吉生物 演讲主题: Innovation strategies in TIL Therapy against Solid Tumor Abstract & Bullet Points:  • Limitation and challenges for solid tumor T cell therapies; • BioSyngen’s strategy for T cell therapy development on solid tumors; •BioSyngen’s TIL platform and preclinical development; •Phase I clinical data of BST02 liver cancer TIL. 璩 良 研究员、博士生导师 复旦大学基础医学院 演讲主题: Nucleoside-modified circRNA technology and Chemically-synthesized RNA Vaccine Speaker Bio:  璩良, 复旦大学研究员、博士生导师，致力于RNA医学技术开发，解决现有技术的脱靶、稳定性低及免疫原性高等瓶颈问题，取得了系列成果。①其开发了RNA编辑技术LEAPER及其迭代版LEAPER2.0（Nature Biotechnology 2019 第一作者；2022 共一排二），摆脱了对CRISPR-Cas核酸酶的依赖，解决了此前RNA编辑技术的脱靶问题，入选国家十三五科创成就展，已进入临床（Cell 2026）。②其在国际上率先报道circRNA疫苗技术（Cell 2022第一作者）以及基于circRNA的现货型in vivo CAR疗法（Cell Rep Med 2025最后通讯；bioRxiv 2025最后通讯），突破了线性mRNA稳定性低的技术瓶颈，解决了传统CAR-T疗法成本极高、依赖化疗清淋等临床痛点。③其在国际上率先报道碱基修饰circRNA技术，消除了circRNA在体内的天然免疫刺激反应，拓展了circRNA的疫苗外应用新场景（Nature Biomedical Engineering 2026最后通讯）；进一步开发了可翻译蛋白的全化学合成RNA寡核苷酸技术—PEO（PNAS 2026最后通讯），实现RNA疫苗生产范式从生物制造到化学合成的革新，将制备周期由3个月压缩至1天，为个体化癌症疫苗和新发突发传染病的快速应对提供了新技术。璩良博士申请PCT发明专利12项、两项技术进入临床试验；入选国家高层次青年人才计划、上海东方英才计划、上海启明星计划、上海晨光计划、国家博新计划、吴瑞奖等。 徐 伟 首席科学官 剂泰科技 演讲主题: in vivo TCE in Oncology and Autoimmunity Speaker Bio:  徐伟博士是一位在免疫、肿瘤、细胞治疗等多个领域具有深厚学术背景的科学家。他的学术生涯始于荷兰莱顿大学医学中心，并于2007年获得博士学位。随后，他加入贝勒免疫学研究所担任博士后研究员，进一步积累了宝贵的科研经验。在罗氏创新中心（瑞士）工作期间，他领导了4条管线进入临床阶段，展现了出色的研发管理能力。2018年至2022年，徐博士在信达生物（苏州）担任副总裁兼新药生物与转化医学负责人，帮助公司建立了细胞治疗和转化医学等团队，推动公司研发团队从Me too药物到FIC和BIC药物的能力转变。随后，他加入Numab Therapeutics公司（瑞士）担任首席科学官，负责制定公司的整体研发及管线战略。 张亭亭 BD负责人 博生吉 演讲主题: Latest Advances in Lentivirus‑Mediated In Vivo CAR‑T Abstract & Bullet Points:  • In vivo CAR研发的核心挑战 • 全球慢病毒 In vivo CAR-T 研发格局 •风险与挑战 马丽佳 领衔科学家 昌平实验室 演讲主题: Site-specific in vivo CAR integration Abstract & Bullet Points:  • AI-guided capsid evolution identified an AAV6 variant with enhanced T-cell targeting and reduced liver tropism • A single injection achieved >70% CAR+ T cells in vivo at 6 weeks in an hCD34+ mouse model. •AAV vector accumulation was reduced by >2 orders of magnitude in both mouse and NHP models. •Site-specific CAR integration supports safe and durable in vivo CAR-T generation. 李建强 创始人, 首席执行官兼首席科学官 金要生物 圆桌讨论： “ 818 ”号令下细胞免疫治疗新技术临床研究与转化应用 Speaker Bio:       先后于协和医科大学、德国维尔茨堡获得免疫学硕士和博士学位，长期就职于美国西雅图福瑞德•哈金森（Fred Hutchinson）肿瘤研究中心，从事T细胞及造血干细胞治疗技术的开发和临床转化。      2016年归国后先后创建森朗生物和金要生物，致力于新一代CAR-T药物的研发，国际上首创自然选择模式制备CD7 CAR-T（NS7CAR），主导开发的CAR-T类新药“Senl_B19”和“SENL101”已获得国家药监局新药临床试验批件；累计主持省市级研发项目8项，在Nature Medicine等杂志以第一和通讯作者发表学术论文15篇，以第一发明人拥有多项国际发明专利。      目前还担任河北医科大学客座教授/研究生导师、河北省高层次创业人才、石家庄市政府特殊津贴专家等。 夏珏妤 临床开发副总裁 毕诺济生物 演讲主题: A New Generation of CAR-Treg Therapy for Autoimmune Diseases: The Development Story of BEN301 Abstract & Bullet Points:  BEN301 represents a pioneering next-generation CAR-Treg cell therapy developed by bennnubio (Shanghai) Biotechnology for the treatment of autoimmune diseases. Preclinical studies demonstrated that BEN301 effectively suppresses aberrant T-cell and B-cell activation in SLE models, significantly reduces total IgG secretion and SLE-specific autoantibodies—particularly anti-double-stranded DNA antibodies—without observable treatment-related toxicity. The therapeutic rationale leverages the fundamental defect in regulatory T-cell (Treg) number and function observed in autoimmune disease patients, positioning CAR-Treg therapy as a potentially disease-modifying strategy applicable across a broad spectrum of autoimmune conditions. 冯 义 首席医学官 合源生物 圆桌讨论： “ 818 ”号令下细胞免疫治疗新技术临床研究与转化应用 Speaker Bio:  毕业于北京协和医学院，曾在跨国药企（罗氏、诺华等）和创新型药企（合源、瓴路）一直从事新药研发工作，目前就职于合源生物科技有限公司任首席医学官，负责职能部门包括临床医学、转化医学、药物警戒、质量保证和医学撰写等。专注于肿瘤，心血管和代谢性疾病，自身免疫性疾病，肾脏内科，神经精神疾病等；在血液肿瘤和实体肿瘤的新药研发方面具有丰富的经验。负责中国，亚太和全球关键性注册临床试验。研发产品包括小分子药物，大分子药物，抗体偶联药物（ADC）、免疫治疗药物、双抗和细胞治疗产品（Car-T）等。 陈新颖 高级副总裁 新合生物 圆桌讨论：mRNA 个性化肿瘤新生抗原疫苗的开发与产业化 Speaker Bio:  现任新合生物高级副总裁（SVP），全面负责公司生产、商务拓展（BD）及知识产权与专利相关工作。 其博士毕业于香港大学李嘉诚医学院，在生物医药研发、产业化落地、商务合作及全球知识产权布局等领域拥有深厚专业背景与丰富经验，主导推动公司创新药管线推进、生产体系建设及全球化知识产权战略。 方永聪 助理教授, 特别研究员 清华大学机械工程系 演讲主题: Biomimetic Fabrication of Cardiac Organoids via 3D Bioprinting Abstract & Bullet Points:  Cardiac organoid technology has important applications in disease research, drug screening and regenerative repair. However, current systems face critical bottlenecks, including limited size, batch-to-batch variability and insufficient functional biomimicry. Based on key cardiac developmental pathways, we established a small-molecule induction system to direct stem cells toward multiple cardiac lineages. We developed high-throughput droplet-ejection bioprinting for scalable cardiac organoid fabrication and precise assembly, enabling microvascularized organoid assemblies. We further developed hierarchical embedded bioprinting to construct centimeter-scale cardiac chamber models with three-dimensional perfusable vascular networks, reproducing helically ventricle models and supporting stable beating in vitro for more than one month. In parallel, a multimodal dynamic bioreactor was developed to recapitulate mechanical, electrical and fluidic cues during cardiac development and enhance functional maturation. • Directed cardiac multilineage differentiation through developmental pathway-inspired small-molecule induction. • High-throughput droplet-ejection bioprinting for scalable organoid fabrication and precise assembly. • Microvascularized cardiac organoid and centimeter-scale ventricle models with perfusable vascular networks. • Helically myocardial fiber-bundle architecture with stable in vitro beating for more than one month. • Multimodal mechanical, electrical and fluidic conditioning to enhance cardiac tissue maturation. 往届回顾  往期回顾  ATMP 2019 ATMP 2020 ATMP 2021 ATMP 2022 ATMP 2023 ATMP 2023 ATMP 2024 ATMP 2024 Carl June 美国宾夕法尼亚大学 帕克癌症免疫疗法研究所主任 Georg Schett德国埃尔朗根-纽伦堡大学风湿病学和免疫学系主任 Renier Brentjens 美国纪念斯隆-凯特琳癌症中心细胞治疗主任 Mickey Koh 圣乔治医学院干细胞移植科主任 Bruce Levine 美国宾夕法尼亚大学佩雷尔曼医学院荣誉教授 Michael Milone 美国宾夕法尼亚大学佩雷尔曼医学院 Eric Smith 美国纪念斯隆-凯特琳癌症中心 Dan Kaufman 加州大学圣地亚哥健康中心医学部细胞治疗项目主任 Rafal Krol 日本京都大学iPS 细胞研究所研发部首席研究员 Steve Oh 新加坡A*STAR BTI研究所干细胞研究室主任 Round Table 1 Round Table 2 Round Table 3 Round Table 4 会议报名与合作联系 （会议演讲与合作） （会议参会与展位） 【会议演讲, 支持与战略合作】 Kevin Tan 谭先生Tel: (86) 13641961545 E-mail: Kevin.tan@deliver-consulting.com 【会议参会与展位合作】 David Xu 徐先生Tel: (86) 13776293901 E-mail: david.xu@deliver-consulting.com 【会议媒体合作】 Michelle Wang 王小姐Tel: (86 21) 5269-8916  END