ATMP 系列峰會持續專注全球細胞與基因治療領域，始終堅持從全球視角出發，促進來源于中國的高質量研發創新,  快速推進先進治療產品的研發與商業化進程，探索全球合作新模式。近年來 ATMP2019-2023 匯聚了來自賓夕法尼亞大學、紀念斯隆-凱特琳癌癥中心、MD 安德森癌癥中心、帕克癌癥免疫療法研究所、美國費城兒童醫院、加州大學、美國國立癌癥研究所、美國國立衛生研究院、新加坡科技研究局、日本京都大學iPS 細胞研究所等眾多國際先進療法先驅，及中國頂尖學府、科研院所及知名產業界嘉賓的共同參與。由迪易生命科學主辦的 ATMP2024 第七屆先進療法創新峰會與全球再生醫學研發創新與類器官研究峰會將于2024年5月9日-10日在北京龍城溫德姆酒店召開。    
類器官在疾病建模，抗癌藥物篩選，藥物毒理檢測，還有基因和細胞療法的應用并做出了重大貢獻。類器官將用于模擬更復雜的器官，模擬器官內相互作用，并探索致病機制。為促進探索神經發育、神經表觀遺傳、3D類腦器官、誘導性多能干細胞、神經和精神類疾病模型、腫瘤類器官在免疫治療中的應用，類器官模型在長壽醫學領域的研究, 視網膜類器官疾病模型，單細胞組學等眾多研究方向；探討合作新模式。第七屆先進療法創新峰會 - 全球再生醫學研發創新與類器官研究峰會將于2024年5月9-10日在北京舉辦。
 
主要議題：
- 3D人腦類器官-神經科學與腦科學的最新研究進展
- 人腦類器官在神經發育疾病中的研究
- 精準治療時代 - 腫瘤類器官在免疫治療中的應用
- 3D細胞培養與類器官；器官芯片助力疾病建模 - 再生醫學- 干細胞與臟器類器官研究 ( 腦科學、神經科學、眼科-視網膜、呼吸道-肺成體、肝膽、腫瘤與器官芯片）
- 干細胞技術與類器官模型在長壽醫學領域的研究進展及應用前景
- 視網膜類器官疾病模型與視網膜再生醫學
- 神經退行性疾病的干細胞療法 
- 類器官芯片，生物傳感器與疾病模型
 
主旨演講嘉賓 Plenary Speakers
 
宋洪軍

著名華人神經科學家
美國國家醫學院院士
美國賓夕法尼亞大學
佩雷爾曼醫學院
再生醫學研究所
演講主題: Therapeutic Application of Human 3D Brain Organoids: Opportunities and Challenges
Abstract: 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.
 
李晨鐘 

香港中文大學
校長講席教授
演講主題: 生物傳感器和器官/類器官芯片的一體化
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.
 
金子兵
首都醫科大學教授
附屬北京同仁醫院
北京市眼科研究所

演講主題: Human Retinal Organoids for Disease Modeling & Regeneration
Abstract: 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.
 
劉興國
中國科學院廣州生物醫藥
與健康研究院研究員
中國科學院再生生物學
重點實驗室研究員

演講主題: Stem Cell and Organoid Model for Aging and Diseases
Abstract: 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.
 
劉鵬
清華大學醫學院
生物醫學工程系研究員，博導
昌平國家實驗室
新發突發傳染病部領銜科學家
 
演講主題: 腫瘤類器官在免疫治療中的應用
Application of Tumor Organoids in Immunotherapy 
Abstract: 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.
 
向陽飛
上海科技大學
助理教授，研究員
博士生導師

演講主題: Development and Application of Human Neural Organoids
Abstract: 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.
 
艾曉妮
北京大學藥學院副研究員
國家重點研發計劃首席青年科學家
 
演講主題: 基于器官芯片的藥物評價新方法
Organ-on-a-chip for Drug Discovery 
Abstract: 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.
 
王凱
北京大學基礎醫學院
生理學與病理生理學系
研究員，博導
血管穩態與重構
全國重點實驗室PI

演講主題: Injectable Vascular Organoids for Treating Ischemic Diseases
Abstract: 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.
 
冷泠
中國醫學科學院
北京協和醫院教授，博導
疑難重癥與罕見病
全國重點實驗室獨立PI
 
演講主題: Construction of Complex Skin Organoids and their Application in Diseases
Abstract: 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.

那潔
清華大學
醫學院副教授

演講主題: 干細胞來源的脈絡膜內皮細胞移植治療眼脈絡膜缺血 - 類器官研究最新進展與再生醫學 
Human Pluripotent stem Cells Derived Endothelial Cells Repair Choroidal Ischemia-New Advance in Organoid Research and Regenerative Medicine
Abstract: Choroidal atrophy is closely related to the development of age-related macular degeneration (AMD), retinitis pigmentosa, and pathological myopia. Studies suggested that choroidal endothelial cells (CECs) that form the choriocapillaris vessels are the first cells lost in choroidal atrophy. We found that endothelial cells derived from human pluripotent stem cells (hPSC-ECs) expressed CECs-specific markers and can integrate into choriocapillaris. scRNA-seq studies showed that hPSC-ECs upregulated angiogenesis and immune-modulatory and neural protective genes after interacting with ex vivo ischemic choroid. In a rat model of choroidal ischemia (CI), transplantation of hPSC-ECs into the suprachoroidal space increased choroid thickness and vasculature density. Close-up examination showed that engrafted hPSC-ECs integrated with all layers of choroidal vessels and lasted 90 days. Remarkably, EC transplantation improved the visual function of CI rats. Our work demonstrated that hPSC-ECs could repair choroidal ischemia, which may lead to a new therapy to alleviate choroidal atrophy implicated in dry AMD, pathological myopia, and other ocular diseases.
 
張宇
中源藥業
首席執行官

演講主題: Development of Adult Stem Cell Products: from Autologous to Allergenic
Abstract: 
- Advances of adult stem cell therapy products worldwide- Key considerations in developing autologous and allogenic stem cell products
Optimized cell source: Perinatal tissue vs adult tissue vs ESC/iPSC CMC issues
IIT study vs IND trial
Indication selecting
License-out vs in-house development
- Vcanbiopharma-20-year’s experiences: from HSC to MSC to iPSC
 
盛健
神曦生物
首席執行官
 
演講主題：In-situ Neuro-regenerative Gene Therapy: from Bench to Bedside
Abstract: 
1. Background information of In-situ neuro-regenerative gene therapy2. NeuExcell cutting-edge platform and pipeline progress: bench to bedside3. Team introduction and partnership
 
郭瑋
神濟昌華
聯合創始人兼首席科學官
 
演講主題: AAV-Based Gene Therapy Strategies for Neurological Diseases

周靜敏
鯨奇生物
聯合創始人兼首席執行官

演講主題: The Progress on Genemagic’s Key Pipeline – Parkinson’s Disease
Abstract: Parkinson's disease is a progressive disorder that affects the nervous system. Unfortunately, the current medicines only reduce the disease symptoms, none of them could cure, stop, or even slow down the rapid progression. A novel method to reverse Parkinson’s disease by regeneration of dopaminergic neurons has been developed.
This presentation includes: 1 NHP long term (3 years) safety and efficacy data on neuron regeneration, 2 the efficacy study of neuron regeneration at CRO, and 3 observation of direct neuron conversion from astrocyte in vivo by two-photon.
   
2023年12月，美國食品藥品監督管理局（FDA）宣布，批準CRISPR/Cas9基因編輯療法Casgevy（通用名exagamglogene autotemcel, 簡稱exa-cel）上市，用于治療鐮狀細胞�。⊿CD）�；�因編輯是一種新興的、能夠比較精確地對生物體基因組特定目標基因進行修飾的基因工程技術。Casgevy是FDA批準的第一種新型基因組編輯療法, 標志著基因治療領域的創新進步。
 
隨著全球范圍內學術、醫療、資本與產業的積極參與，ATMP相關在研產品數量呈現爆發式增長。與此同時，該領域也面臨著法規監管、工藝開發、商業化生產、出海合作、患者可及性等諸多問題與挑戰。為促進來源于中國的高質量研發創新，快速推進先進治療產品的研發與商業化進程，探索合作新模式，第七屆先進療法創新峰會 - 全球細胞與基因治療創新峰會將于2024年 5月9-10日在北京舉辦。
 
部分主要議題：
- 基因編輯療法與工程化免疫細胞療法的未來
- 開發新一代安全且有效的細胞與基因治療產品的研發與先進技術創新
- 新技術-新靶點: 實體瘤創新細胞免疫療法 與 mRNA 體內編輯 In-Vivo CAR-T
- 創新細胞免疫療法在惡性血液瘤以及實體瘤領域治療最新進展
- CAR-T, CAR-M, TIL, CAR-NK, TCR-T 與自身免疫性疾病CAR-T 細胞療法的研發
- 基因治療在神經退行性疾病、眼科疾病、耳聾、常見病與罕見病治療領域的突破
 
主旨演講嘉賓 Plenary Speakers

Stephan Grupp
世界著名癌癥免疫治療專家、CAR-T先驅
美國賓夕法尼亞大學教授
美國費城兒童醫院
Emily Whitehead 主治醫生
 
演講主題: 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
 
Mitchell Ho 何苗壯
美國醫學與生物工程院
院士
美國國立衛生研究院
美國國立癌癥研究所

演講主題: Nanobodies as Emerging Antibody and Cell Therapeutics for Cancer and Viral Infections 
Abstract: 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.
 
Peter Marks
美國國家醫學院院士
美國食品藥品監督管理局
生物評估和研究中心
 
演講主題: 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.  
 
王立群
星奕昂生物科技
創始人，董事長
兼首席執行官
 
演講主題: Allogenic Cell Therapy: To Be or Not to Be
 
林欣

清華大學醫學院教授
基礎醫學系主任
華夏英泰創始人
 
謝興旺

可瑞生物創始人
董事長兼首席執行官

何霆

藝妙神州
創始人兼首席執行官
 
孫艷

上海細胞治療集團
北京細胞治療集團
集團首席運營官
 
劉明耀

邦耀生物
董事長兼首席科學家
 
汪文

馴鹿生物
執行總裁

孫敏敏

易慕峰創始人
董事長兼首席執行官

任江濤

北恒生物
聯合創始人兼首席科學官

楊林

博生吉創始人
董事長兼首席執行官
 
張同存

波睿達創始人
董事長兼首席執行官
 
劉華

星華生物
董事長兼首席科學家
 
況嬌

森朗生物
首席醫學官
 
王漢明

濱會生物
首席醫學官
 
王永增

合源生物
首席技術官

肖嘯

信念醫藥
董事長兼首席科學家

楊麗萍

中因科技
董事長兼首席科學家北京大學第三醫院眼科研究員

董文吉

中吉智藥
創始人、董事長

董飚

至善唯新
董事長