3D类脑器官, 器官芯片, 疾病模型与细胞治疗 | 全球再生医学研发创新与类器官研究峰会 2024年5月9-10日·北京
ATMP 系列峰会持续专注全球细胞与基因治疗领域，始终坚持从全球视角出发，促进来源于中国的高质量研发创新, 快速推进先进治疗产品的研发与商业化进程，探索全球合作新模式。近年来 ATMP2019-2023 汇聚了来自宾夕法尼亚大学、纪念斯隆-凯特琳癌症中心、MD 安德森癌症中心、帕克癌症免疫疗法研究所、美国费城儿童医院、加州大学、美国国立癌症研究所、美国国立卫生研究院、新加坡科技研究局、日本京都大学iPS 细胞研究所等众多国际先进疗法先驱，及中国顶尖学府、科研院所及知名产业界嘉宾的共同参与。由迪易生命科学主办的 ATMP2024 第七届先进疗法创新峰会与全球再生医学研发创新与类器官研究峰会将于2024年5月9日-10日在北京龙城温德姆酒店召开。
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类器官在疾病建模，抗癌药物筛选，药物毒理检测，还有基因和细胞疗法的应用并做出了重大贡献。类器官将用于模拟更复杂的器官，模拟器官内相互作用，并探索致病机制。为促进探索神经发育、神经表观遗传、3D类脑器官、诱导性多能干细胞、神经和精神类疾病模型、肿瘤类器官在免疫治疗中的应用，类器官模型在长寿医学领域的研究, 视网膜类器官疾病模型，单细胞组学等众多研究方向；探讨合作新模式。第七届先进疗法创新峰会 – 全球再生医学研发创新与类器官研究峰会将于2024年5月9-10日在北京举办。主要议题：
– 3D人脑类器官-神经科学与脑科学的最新研究进展
– 人脑类器官在神经发育疾病中的研究
– 精准治疗时代 – 肿瘤类器官在免疫治疗中的应用
– 3D细胞培养与类器官；器官芯片助力疾病建模 – 再生医学- 干细胞与脏器类器官研究 ( 脑科学、神经科学、眼科-视网膜、呼吸道-肺成体、肝胆、肿瘤与器官芯片）
– 干细胞技术与类器官模型在长寿医学领域的研究进展及应用前景
– 视网膜类器官疾病模型与视网膜再生医学
– 神经退行性疾病的干细胞疗法
– 类器官芯片，生物传感器与疾病模型
著名华人神经科学家美国国家医学院院士美国宾夕法尼亚大学佩雷尔曼医学院演讲主题: Therapeutic Application of Human 3D Brain Organoids: Opportunities and ChallengesAbstract: Brain organoids are 3D tissue cultures that resemble cell type diversity, tissue architecture and developmental trajectory of the native human brain tissues. Rapid advances in the stem cell technologies have led to human pluripotent stem cell-derived brain organoids that mimic the development and properties of different regions of the developing human brain. In parallel, brain organoids have been generated from patient surgical tissues, such as glioblastoma, that can maintain inter- and intra-tumor heterogeneity as well as the tumor microenvironment. I will review recent development of brain organoid technologies and provide examples for therapeutic applications of these human stem cell-derived brain organoids, such as applications during the past two global pandemics (Zika virus and SARS-Cov2). I will also discuss technologies of tumor organoids and their applications in the personized medicine. Finally, I will discuss challenges ahead.Abstract: Biosensor is a powerful, label-free technique allowing us to perform analysis of molecular interactions in real-time. SPR spectroscopy can address questions such as specificity of an interaction, dissociation and association rate constants; binding kinetics, binding affinity, and concentrations of selected molecules present in a sample of interest. In this work, we report the novel SPR based cell/organoid integrated sensing platforms that allow us to real-time monitor cell and 3D tissue activities upon various of stimulations. Using the novel set up, we measured and compared the binding affinity of vascular endothelial growth factor (VEGF) to vascular endothelial growth factor receptor (VEGFR) and VEGF to bevacizumab. Results have shown that bevacizumab binds VEGF with a higher association rate and affinity compared to VEGFR. Further, this platform has been employed to mimic the in vivo condition of the VEGF–VEGFR angiogenic switch. Competitive binding to VEGF between VEGFR and bevacizumab was monitored in real-time using this platform. The present invention provides surface plasmon resonance (SPR) based sensing systems and methods for rapid, sensitive, and real-time analysis of analyte secretion from living cells. In one embodiment, the SPR based sensing device of the present invention comprises at least one cell culture module for culturing living cells, wherein the cell culture module is configured so that analytes secreted from the living cells can be released onto a SPR sensing surface. The SPR based sensing system can perform a real-time analysis of one or more analytes secreted from the living cells by including a coating on the SPR sensing surface. In addition, we have successfully demonstrated the use of surface plasmon resonance (SPR) technology to characterize the contractility of 3D cardiac tissues in response to Blebbistatin and ATP drug exposure in real time.演讲主题: Human Retinal Organoids for Disease Modeling & RegenerationAbstract: Together with the rapid advancement of retinal organoid technology, human induced pluripotent stem cell have enabled us to generated patient-specific retina tissue. In this talk, I will introduce the retinal organoid differentiation, disease modeling, and transplantation.中国科学院广州生物医药与健康研究院研究员中国科学院再生生物学重点实验室研究员演讲主题: Stem Cell and Organoid Model for Aging and DiseasesAbstract: Aging in mammals is accompanied by an imbalance of intestinal homeostasis and accumulation of mitochondrial DNA (mtDNA)mutations.However, little is known about how accumulated mtDNA mutations modulate intestinal homeostasis. We observe the accumulation of mtDNA mutations in the small intestine of aged male mice, suggesting an association with physiological intestinal aging. Using polymerase gamma (POLG)mutatormice and wild-type mice, we generate male mice with progressive mtDNA mutation burdens. Investigation utilizing organoid technology and in vivo intestinal stem cell labeling reveals decreased colony formation efficiency of intestinal crypts and LGR5-expressing intestinal stem cells in response to a threshold mtDNA mutation burden. Mechanistically, increased mtDNA mutation burden exacerbates the aging phenotype of the small intestine through ATF5 dependent mitochondrial unfolded protein response (UPRmt) activation. This aging phenotype is reversed by supplementation with the NAD+ precursor, NMN. Thus, we uncover a NAD+ dependent UPRmt triggered by mtDNA mutations that regulates the intestinal aging.清华大学医学院生物医学工程系研究员，博导昌平国家实验室新发突发传染病部领衔科学家演讲主题: 肿瘤类器官在免疫治疗中的应用Application of Tumor Organoids in ImmunotherapyAbstract: Immune checkpoint blockade (ICB) opens the new era of cancer treatment, yet the heterogeneous nature of immune cells and their diverse spatial distributions demand novel techniques to decipher the local tumor immune microenvironment (TIME) to expand the patient groups benefiting from ICB. Here we generate primary lung cancer organoids (pLCOs) by isolating the tumor cell clusters, including the infiltrating immune cells, from dissected lung cancer samples. A FascRNA-seq platform allowing both the phenotypic evaluation and the scRNA-seq of all the single cells in an organoid was developed to dissect the TIME in individual pLCOs. Our analysis on 171 individual pLCOs derived from 7 patients revealed that pLCOs retained the fundamental features as well as the intra-tumor heterogeneity of local TIME in the parenchyma of parental tumor tissues, providing a series of models with the same genetic background but various TIME. Linking the single cell transcriptome data of individual pLCOs with their responses to ICB allowed us to confirm the central role of CD8+ Ts in ICB induced antitumor immunity, to identify the potential tumor-reactive T cells with a set of 10 genes, and to unravel the factors regulating T cell activity.演讲主题: Development and Application of Human Neural OrganoidsAbstract: Neural organoids are in vitro three-dimensional models that mimic the human brain or other structures of the nervous system. Beginning with stem cells, neural organoids are formed through unguided or guided neural differentiation under three-dimensional suspension culture conditions, relying on cell self-organization. In the past decade of research, we have focused on guided differentiation to construct various human brain region-specific organoids. Furthermore, by integrating multiple brain regions or cell lineages, we have explored the development of more complex human brain organoid technologies, providing new models for studying brain development, function, diseases, and drug effects in the context of human genetic backgrounds in vitro. As a cutting-edge technology, neural organoids still face various technical challenges that need to be overcome. This talk will introduce our efforts in the refined construction of human neural organoids, including how to build organoids that possess characteristics of human brain nuclei.演讲主题: 基于器官芯片的药物评价新方法Organ-on-a-chip for Drug DiscoveryAbstract: The high cost and low success rate of drug development are major challenges, and there are significant differences between traditional preclinical biological models and the human physiopathology. Organ-on-a-chip technology as a novel model offers advantages for drug discovery, such as high biomimicry, low sample consumption, and high efficiency. We established microfluidic chip platforms for single-organ and multi-organ cultures. Based on these platforms, we developed over 20 highly biomimetic organ-on-a-chip models. These models have been applied in drug discovery and mechanism research. A notable achievement includes the pioneering use of tumor-on-a-chip models for the clinical application of cell therapy drug and dual-specificity antibody drug in China, marking a groundbreaking advancement in the field.演讲主题: Injectable Vascular Organoids for Treating Ischemic DiseasesAbstract: Critical limb ischemia (CLI) is a severe obstruction of the arteries which markedly reduces blood flow to the limbs and has progressed to the point of severe pain, ulcer and even amputation. Therapeutic angiogenesis using implanted vascular cells has been widely investigated to treat the CLI, however, the therapeutic outcome is quite mixed. Since the vasculogenesis potential and paracrine effect of the transplanted vascular cells are the two major driving forces for enhancing the local neovascularization, we hypothesized that stem cell derived vascular organoids (VO) could be the ideal cell sources.演讲主题: Construction of Complex Skin Organoids and their Application in DiseasesAbstract: Difficult and rare diseases have greatly hindered basic research and clinical diagnosis and treatment due to their small patient base, difficulty in medication, and unclear causes of onset. Xeroderma pigmentosum (XP) is a rare disease with birth defects. XP patients usually die before the age of 10, and there is currently no cure. We utilized the previously established skin organoid model to construct a human-induced pluripotent stem cell (hiPSC) derived XP skin organoid model from XP patients and a PDX model derived from XP organoid transplant mice. By combining multidimensional omics techniques such as single-cell transcriptome and spatial proteomics, we discovered a potential small molecule drug that can serve as a preventive measure against XP tumors after surgery.演讲主题: Development of Adult Stem Cell Products: from Autologous to Allergenic– Advances of adult stem cell therapy products worldwide– Key considerations in developing autologous and allogenic stem cell products
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Optimized cell source: Perinatal tissue vs adult tissue vs ESC/iPSC CMC issues
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License-out vs in-house development
– Vcanbiopharma-20-year’s experiences: from HSC to MSC to iPSC演讲主题:In-situ Neuro-regenerative Gene Therapy: from Bench to Bedside1. Background information of In-situ neuro-regenerative gene therapy2. NeuExcell cutting-edge platform and pipeline progress: bench to bedside3. Team introduction and partnership演讲主题: AAV-Based Gene Therapy Strategies for Neurological Diseases2023年12月，美国食品药品监督管理局（FDA）宣布，批准CRISPR/Cas9基因编辑疗法Casgevy（通用名exagamglogene autotemcel, 简称exa-cel）上市，用于治疗镰状细胞病（SCD）。基因编辑是一种新兴的、能够比较精确地对生物体基因组特定目标基因进行修饰的基因工程技术。Casgevy是FDA批准的第一种新型基因组编辑疗法, 标志着基因治疗领域的创新进步。随着全球范围内学术、医疗、资本与产业的积极参与，ATMP相关在研产品数量呈现爆发式增长。与此同时，该领域也面临着法规监管、工艺开发、商业化生产、出海合作、患者可及性等诸多问题与挑战。为促进来源于中国的高质量研发创新，快速推进先进治疗产品的研发与商业化进程，探索合作新模式，第七届先进疗法创新峰会 – 全球细胞与基因治疗创新峰会将于2024年 5月9-10日在北京举办。部分主要议题：
– 基因编辑疗法与工程化免疫细胞疗法的未来
– 开发新一代安全且有效的细胞与基因治疗产品的研发与先进技术创新
– 新技术-新靶点: 实体瘤创新细胞免疫疗法 与 mRNA 体内编辑 In-Vivo CAR-T
– 创新细胞免疫疗法在恶性血液瘤以及实体瘤领域治疗最新进展
– CAR-T, CAR-M, TIL, CAR-NK, TCR-T 与自身免疫性疾病CAR-T 细胞疗法的研发
– 基因治疗在神经退行性疾病、眼科疾病、耳聋、常见病与罕见病治疗领域的突破
世界著名癌症免疫治疗专家、CAR-T先驱美国宾夕法尼亚大学教授美国费城儿童医院Emily Whitehead 主治医生演讲主题: Current Advances and Challenges in Engineered Cell Therapy for Leukemia and Red Cell Disorders: from CAR T to CRISPR– Current status of CAR T therapy for relapsed/refractory ALL- Updates in CAR T toxicity management- Alternative targets beyond CD19- Engineered stem cell therapies for thalassemia and sickle cell disease
– Approval of Casgevy in the US for red cell disorders
美国医学与生物工程院院士美国国立卫生研究院美国国立癌症研究所演讲主题: Nanobodies as Emerging Antibody and Cell Therapeutics for Cancer and Viral InfectionsAbstract: The emergence of nanobody technology has provided new hope for antibody and cell based drug development. Our laboratory at the U.S. National Cancer Institute at the NIH has constructed large nanobody phage display libraries derived from the VHH and VNAR single domains of dromedary camels and nurse sharks, respectively. In my talk, I will present recent examples of CAR-T cells based on our nanobodies targeting glypican-1 (GPC1) and B7-H3 (CD276) for the treatment of pancreatic cancer and pediatric cancers. We have also identified nanobodies capable of neutralizing SARS-CoV-2 and the Lassa virus. I will also discuss the structure and functional features of these nanobodies and CAR-T cells as innovative antibody and cell therapeutics.美国国家医学院院士美国食品药品监督管理局生物评估和研究中心演讲主题: The Future of Human Genome Editing: A Regulatory Perspective (Online Presentation)Abstract: Genome editing offers tremendous promise for the treatment of disease. The US FDA understands that we need to re-evaluate and modernize our approach to the unique challenges of genome editing while also ensuring the resulting therapies are both safe and effective. The FDA is taking steps to facilitate more efficient genome editing product development. For example, the FDA will encourage the use of biomarkers as surrogate endpoints to help facilitate the accelerated approval of gene therapies for serious or life-threatening conditions, such as lysosomal storage disorders and neurodegenerative diseases affecting very small numbers of individuals. FDA is also running a pilot program to attempt to further accelerate the pace of development of therapeutics for very small populations with very high medical need. This pilot for rare pediatric genetic diseases will allow ongoing informal interactions during development of the product. Finally, while countries around the world have their own regulatory authorities, there are not uniform global quality safety standard for the evaluation and regulation of cell and gene therapy products. The FDA supports work toward global regulatory convergence and, ultimately, global harmonization of regulations for these products.演讲主题: Allogenic Cell Therapy: To Be or Not to Be
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