第八届先进疗法创新峰会（ATMP2024) | 聚焦细胞与基因治疗产业布局, 聚集”审评监管-产-学-研-医“海内外顶流

文摘 2024-10-30 07:20 辽宁

2024年12月19-20日

中国上海

近年来 ATMP2019-2024 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、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(FAU) 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 第八届先进疗法创新峰会将于2024年12月19-20日在上海康桥万豪酒店召开。

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全球细胞与基因治疗创新峰会2024

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，第八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12月19-20日在上海康桥万豪酒店举办。

部分主要议题：

* CAR-T 细胞免疫治疗产品的注册与商业化

* CRISPR基因编辑疗法与工程化免疫细胞疗法的未来

* 自身免疫性疾病CAR-T 细胞疗法的研发

* 开发新一代的创新细胞免疫疗法: 脑肿瘤, 实体瘤以及恶性血液瘤领域治疗的最新进展 ( CAR-T. TIL, CAR-NK, TCR-T, CAR-M, In Vivo mRNA CAR T etc., )

* 细胞与基因治疗产品监管科学研究中的探索与思考

* AAV基因治疗在神经退行性疾病, 渐冻症, 眼科疾病, 耳聋, 常见病与罕见病治疗领域的突破

* 罕见病治疗的创新途径

* 细胞治疗药物的开发前景,工艺优化,技术创新，质量控制及商业化生产

* 细胞与基因治疗前沿技术创新

细胞免疫与基因治疗

主旨演讲嘉宾 Plenary Speakers

陈星蓉

首席执行官

复星凯特

(复星凯瑞 Fosun Kairos)

演讲主题：Learnings in registration and commercialization as the pioneer in the China CAR-T market

Abstract:

Who is Fosun Kairos?
Fosun Kairos’ successful experience in registration and commercialization of YIKAIDA, the first CAR-T cell therapy in China;
Exploring innovative payment solutions to increase CAR-T accessibility;
Looking forward, how can we release the potential of China CGT market?

华坚

执董总经理

上海医药集团生物治疗技术有限公司

上药生物治疗(香港)有限公司

演讲主题: 大药企背景下的细胞治疗布局

Speaker Bio:

华坚，临床医学专业毕业，早年留学日本获管理硕士学位。回国后深耕医疗行业近20年，涉足生物医药、医疗器械、医学影像设备的研发、生产和销售等多个领域。此外，在香港创业板股权投资和房地产投融资等金融领域亦有十年从业经历，积累了丰富多元的跨行业经验。2018年底入职上海医药组建上药香港细胞治疗研发实验室，2019年组建上海医药上海细胞公司。现任上药生物治疗（香港）有限公司董事长、总经理，上海医药集团生物治疗有限公司执董、总经理，上海市肿瘤细胞治疗技术创新中心执行主任，上药与上海儿童医学中心合建儿童细胞治疗技术中心联合主任。

Mitchell Ho 何苗壮

美国国立卫生研究院 NIH

美国国立癌症研究所 NCI

终身资深研究员

美国肿瘤研究所抗体治疗研究室主任

演讲主题：Nanobody-Based CAR-T Cells Targeting Extracellular and Intracellular Tumor Antigens

Speaker Bio:

Dr. Ho is a Senior Investigator, the Deputy Chief of the Laboratory of Molecular Biology, the Head of the Antibody Therapy Section, and the Director of the Antibody Engineering Program at the National Cancer Institute (NCI), National Institutes of Health (NIH). He received his Ph.D. from the University of Illinois at Urbana-Champaign, where he generated anti-idiotypic antibodies as cocaine antagonists. He completed a postdoctoral fellowship at the NIH, where he engineered immunotoxins targeting CD22 and mesothelin for the treatment of B cell leukemias and mesothelioma. Dr. Ho has pioneered the generation of therapeutic antibodies that target cancer-associated heparan sulfate proteoglycans. A focus of his laboratory work is on the validation of cell surface glypicans such as GPC3, GPC2 and GPC1 as new therapeutic targets in cancer. This area of research ranges from investigation of the fundamental mechanisms by which glypicans regulate Wnt, Yap and other signaling molecules to the design of antibody-based cancer therapeutics. His laboratory also established mammalian cell surface display, developed rabbit monoclonal antibodies, and built shark and camel single domain antibody phage libraries as new tools to advance antibody engineering and drug discovery. The immune therapeutics such as CAR-T cells based on his research are being tested at clinical stages for treating liver cancer, neuroblastoma, mesothelioma and other cancers.

Dr. Ho is was elected to the Board of Directors for the Antibody Society and to the Board of Directors for the Foundation for Advanced Education in the Sciences (FAES). Dr. Ho received many awards including the Asian & Pacific Islander American Organization (APAO) Scientific Achievement Award, Dr. Francisco S. Sy Award for Excellence in Mentorship at HHS, NIH Deputy Director for Intramural Research (DDIR) Innovation Award, and NCI Director’s Innovation Award.

许田

副校长, 遗传学讲席教授

西湖大学

创始人

精缮科技

演讲主题：罕见病治疗的创新途径

梅妮

国家GLP检察员、GCP检察员

学术委员会秘书

药理临床核查部主审审评员

上海药品审评核查中心

演讲主题: 细胞与基因治疗产品监管科学研究中的探索与思考

赵维莅

上海交通大学医学院附属瑞金医院副院长

上海市重中之重临床医学中心主任

上海市血液学研究所所长

Speaker Bio:

Zhao Wei-Li, M.D., PhD, Professor of Hematology, Director of Shanghai Institute of Hematology, Director of Shanghai top priority clinical medical center and Vice President of Shanghai Ruijin Hospital. She is the doctoral supervisor of both Shanghai Jiao Tong University and University Paris VII, foreign member of American Society of Hematology, Vice President of Chinese Association of Hematology, Vice President of Chinese Society of Experimental Hematology, and member of the editorial committee of Pathobiology, Biomarker Research, Chinese Journal of Hematology, Chinese Journal of Leukemia & Lymphoma, and Clinical Hematology Journal. She mainly focuses on the clinical and basic research of hematological malignancies, especially the molecular mechanism and targeted therapy of malignant lymphoma. She innovatively proposed the molecular pathways of apoptosis, angiogenesis and cell differentiation involved in lymphoma progression and the availability of targeted agents for treatment. She has been awarded National Award for Science and Technology Progress and Outstanding Achievement Award of Scientific Research from National Ministry of Education and HUA XIA Award of Medicine and Technology from National Ministry of Science and Technology for her research progress and is now leading projects granted by National High Technology Research and Development Program of China, by National Natural Science Foundation of China and several other events on provincial level. She has been honored New Century Excellent Talents in University by State Ministry of Education, Rising-Star by Shanghai Committee of Science and Technology, Shu Guang Scholar by Shanghai Municipal Educational Committee. She has published over 120 articles including those on leading hematology journals like《CANCER CELL》、《NATURE GENETICS》、《BLOOD》、《MOLECULAR CANCER》、《SIGNAL TRANSDUCT TARGET THER》、《J HEMATOL ONCOL》、《LANCET HAEMATOL》、《CLIN CANCER RES》with total impact factors over 1600.

Stephan Grupp

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

在线演讲主题: Current Advances and Challenges in Engineered Cell Therapy for Leukemia and Red Cell Disorders: from CAR T to CRISPR

Abstract:

• 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

Peter Marks

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

在线演讲主题: 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.

陆英明

联合创始人兼首席执行官

辉大生物

演讲主题: Novel CRISPR-based genome-editing tools rescue fatal diseases and extend lifespan

(基于 CRISPR 的新型基因组编辑工具挽救致命疾病并延长寿命)

Abstract:

Genetic fatal diseases are among the most significant public health challenges, as many lack disease-modifying treatments. The CRISPR system can be used in disease models to edit and manipulate genomes and control gene expression levels precisely, offering the potential to cure diseases as never before. Scientific aspects of employing novel CRISPR technology for gene-editing therapeutic applications will be discussed, focusing on high-fidelity Cas12Max DNA-editing for Duchenne Muscular Dystrophy (DMD) and Cas13 RNA-editing for MECP2 Duplication Syndrome (MDS).

We engineered the natural Cas12i variant to develop the high-fidelity CRISPR-Cas12 (hfCas12Max) with high editing efficiency and specificity. Here, we report using a single adeno-associated virus (AAV) carrying hfCas12Max and guided RNA (gRNA) targeting the human dystrophin gene, HG302, for treating DMD. Our in-vivo results of the single-cut DNA-editing strategy support the initiation of MUSCLE clinical study (an open-label, Multidose study to Understand the Safety of Crispr gene-editing therapy and its long-Lasting Effects in DMD patients; NCT06594094). also developed HG204, consisting of CRISPR/hfCas13Y and gRNAs targeting the MECP2 gene, for MDS, a rare and fatal childhood neurodevelopment disorder. One injection of HG204 restores motor function, anxiety behavior, society impairment, and fear of learning, extending lifespan in our in-vivo studies. We plan to initiate the HERO clinical study (a first-in-Human trial to Evaluate the safety and efficacy of a novel crispr Rna-editing therapy in patients with MDS, a rare Orphan disease; NCT06615206) soon.

The HG204 program has been granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the U.S. Food and Drug Administration (FDA) as well as orphan drug by European Medicines Agency. The HG302 program has also been granted ODD and RPDD by the U.S. FDA. Additionally, the abstract of HG302, using a single-cut gene-editing approach which can be applicable for different hotspot of DMD, has been selected for late-breaking presentation at the 29th Annual Congress of the World Muscle Society in Prague, Czechia.

Novel gene-editing tools to overcome the current CRISPR-Cas9 limitations
Dystrophin expression and muscle function restoration with novel CRISPR/Cas12Max single-cut gene-editing therapy of HG302
Development of CRISPR/Cas13 RNA-editing therapy, HG204, to treat fatal neurodevelopment disease

牟晓盾

首席执行官

正序生物

演讲主题: Advancing Clinical Applications of Gene Editing Using Innovative transformer Base Editor (tBE)

Abstract:

The therapeutic application of gene editing technology in the field of cell or gene therapy is growing rapidly. Currently, Exa-cel (Casgevy), a CRISPR/Cas9 gene-edited cell therapy, is approved by US/EU, several programs using in vivo or ex vivo gene editing have entered clinical stage or even BLA for different indications worldwide, but there are safety concerns such as chromosome abnormalities and off-target mutations with current gene editing tools.

Compared with CRISPR/Cas9 and regular CBE, Correctseq’s proprietary transformer Base Editor (tBE) doesn’t cause double strand breaks or undetected off-target mutations, has higher editing efficiency and lower cytotoxicity, making it potentially the best base editor.
CS-101, the first pipeline using tBE to precisely edit human hematopoietic stem cells ex vivo for the treatment of β-hemoglobinopathy, achieved better efficacy and much lower safety risks in pre-clinical studies. It is now at Phase 1 clinical study. Several patients with transfusion-dependent β-thalassemia who have received CS-101 treatment have all achieved transfusion independence and been able to resume a normal life following treatment. It holds great potential to be the best-in-class gene editing treatment for β-thalassemia and sickle cell disease.
Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic diseases including hypercholesterolemia, hypertriglyceridemia, obesity/diabetes, etc. More details of the tBE system and progresses of the pipelines will be presented.

尹鹤群

高级副总裁，研发负责人

Iovance Biotherapeutics

演讲主题：TIL Immunotherapy for Solid Tumors: Present and Future

Abstract:

-Amtagvi (lifileucel) is the first FDA approved TIL immuno cell therapy for a solid tumor

-TIL therapy targets multiple cancer-specific neoantigens in each individual patient, and there are reported studies of clinical benefits across multiple solid tumor types

-Multiple approaches are in development, including inducible membrane-tethered IL12 aim to further enhance efficacy of TIL therapy

Dr. Yin joined Iovance in November 2021. He is a pharmacologist by training with nearly 30 years of discovery and development experience in various therapeutic areas within the biopharmaceutical industry. His prior leadership roles included Vice President of Oncology Research & Early Development at Pfizer, Executive Director at Novartis, Chief Scientific Officer at Fosun Pharma and President of the Innovation Institute at Qilu Pharmaceutical. Dr. Yin has contributed to the development of Amtagvi, Kymriah and Cosentyx, among others. He earned a Ph.D. in Pharmacology from the University of Rochester and conducted post-doctoral research in the Department of Biochemistry and Molecular Pharmacology at the University of California, San Francisco.

沈浩

复星集团科创合伙人

复星医药合伙人

复星凯特生物科技有限公司首席科学官

(复星凯瑞 Fosun Kairos)

美国宾夕法尼亚大学终身教授

演讲主题: Immunotherapy for Cancer Cure and Beyond - A Historical Mission

Speaker Bio:

Dr. Shen has been a professor at the University of Pennsylvania since 1997. He has long been engaged in the study of T cell immunology. His work focuses on the roles of CD4 and CD8 memory T cells, and the potential applications of T cell memory subsets in autoimmune diseases, tumor immunotherapy and infectious diseases. Dr. Shen also delves into the development of stem cell-like memory T cells (Tscm) through epigenetic regulation. This work lays a critical scientific foundation for enhancing the efficacy and persistence of cellular therapies, thereby driving innovations and advancements in the optimization of treatment strategies for cancer and autoimmune diseases.

Dr. Shen’s contributions have been widely recognized in China, earning him prestigious accolades such as "The Yangtze River Scholar" from the Ministry of Education and "Overseas Outstanding Young Scholars" from the National Natural Science Foundation of China. He served as the Director of the Shanghai Institute of Immunology from 2009 to 2012. Additionally, from 2016 to 2018, he was honored with the title of "Highly Cited Scholar in the field of Immunology and Microbiology in China" by Elsevier, further affirming his significant impact in the scientific community.

Dr. Shen joined FOSUNKite in 2022. He is leading R&D to develop cutting-edge technology platforms, build a rich and differentiated product pipeline, and strive to benefit more patients.

何霆

创始人兼首席执行官

艺妙神州

演讲主题: IM96 CAR-T Therapy: Breakthrough in Metastatic Colorectal Cancer

Abstract:

The options for third-line treatments and their outcomes for metastatic colorectal cancer (mCRC) are severely limited; currently, only drugs like regorafenib, fruquintinib, and trifluridine are available, with a clinical response rate of less than 5%. This is particularly true for colorectal cancer with liver metastasis, where these treatments prove largely ineffective.

Guanylyl cyclase 2C (GUCY2C) is highly expressed in over 80% of colorectal cancer (CRC) cases and is restricted to the apical region of intestinal epithelial cells in normal tissue, making it an attractive therapeutic target. We have developed a CAR-T therapy targeting GUCY2C (IM96) and initiated a Phase I study to assess its safety and efficacy (NCT05287165).

This open-label, 3+3 dose escalation study enrolled mCRC patients positive for GCC who had progressed on at least three prior therapies. Enrolled patients first underwent lymphodepletion with fludarabine and cyclophosphamide, followed by a single infusion of IM96. The CAR-T cell doses were 3 × 10^8 cells (DL1), 6 × 10^8 cells (DL2), 12 × 10^8 cells (DL3), and 20 × 10^8 cells (DL4). The primary endpoints were safety and toxicity, while secondary endpoints included efficacy and pharmacokinetic characteristics.

As of March 2024, a total of 26 patients received IM96 treatment, including 14 with liver metastasis. All patients carried the pMMR/MSS genotype. 5 patients were treated at the third-line stage, and 21 received post-third-line therapy.

Across all treated patients, IM96 demonstrated good tolerability, achieving a response rate of 42.9% in the target dose cohort and 40% in those with liver metastases. Notably, the responses were durable.

孙敏敏

创始人，董事长兼首席执行官

易慕峰生物

演讲主题: Novel CAR-T Therapy Brings Long-Term Benefits to Solid Tumor Patients

Abstract:

CAR-T therapies have been validated in hematologic malignancies, but not approved for treatment of solid tumors. Immunofoco, a cutting-edge CAR-T company, is dedicated to addressing unmet clinical needs by advancing breakthroughs in CAR-T therapy for treating solid tumors and have adopted firstly the strategy of “curing the solid tumors by treating them as hematologic malignancies”.

Key points as below:

- Pipeline and preliminary clinical progress

- Innovative CAR-T technology platforms

- The prospect of CAR-T industry

张曦

首席科学官

百吉生物

演讲主题: Driving Engineered T Cell Therapy for Solid Tumors

Abstract:

- Current bottlenecks of T cell-based therapies for solid tumor treatment

- Biosyngen’s technology and clinical translation platform

- Biosyngen’s CAR-T/TCR-T/TIL development story

陆金华

联合创始人兼首席科学官

星尘生物

演讲主题：FlexTune Platform: its Development and Application for Solid Tumor Treatment

Abstract:

The conventional CAR T therapy has produced stellar results for blood tumor treatments yet obtained limited success so far for solid tumor treatment. In addition, CAR T in its conventional form suffers drawbacks such as uncontrolled adverse effects, limited indications, high costs of development and manufacturing. At TriArm, we took an integrated approach to develop CAR T therapy for the treatment of diseases ranging from cancer, autoimmune diseases to infectious diseases.

We developed FlexTune platform which offers flexibility and functions similar to T cell engager and antibody-drug-conjugates (ADC) in addition to programmable PD/PKs unique to live cellular therapies. In this presentation we will discuss the development of this technology and its application in solid tumor treatment using CD70 program as an example.

李斌

余㵑学者、特聘教授、科研副所长

上海交通大学医学院 - 上海市免疫学研究所

演讲主题: FOXP3+ regulatory T cell perturbation and the future of Treg-based immunotherapy

Abstract:

Research in our laboratory mainly focuses on understanding molecular mechanism underlying the functional instability, and imbalance of FOXP3+ Regulatory T cells (Treg) and RORγt+ T helper 17 cells (Th17) in inflammatory diseases, as well as their therapeutically modulation, especially by compounds from Chinese Traditional Medicine. By using and developing Treg specifically depletion animal models, as well as these state-of-art cutting-edge technologies in single cell sequencing and immune cell metabolism, we are revealing the functional role of tissue-specific Treg subsets and mechanisms underlying their perturbation in health and disease settings. We expect that our original findings will be translational to biomedical industry and be beneficial to the future of Treg-based immunotherapy.

程尹

工艺和分析方法开发副总裁

沙砾生物

演讲主题: CMC Considerations for TIL Immunotherapy – Manufacturing and Control: Challenges, Headaches and Solutions

Abstract:

Tumor-infiltrating lymphocytes (TILs) therapy is a type of cell-based immunotherapy using the patient’s own immune cells from the microenvironment of the solid tumor to kill tumor cells. Recent clinical studies demonstrated that adopting cell transfer of TIL for advanced solid tumors showed good efficacy, and with the first TIL product lifileucel (Amtagvi) approved by FDA February 2024. This session will cover some of the unique CMC complexity of TIL therapy, and discuss considerations in process design, process control and analytics innovation.

王汉明

副总裁

滨会生物

演讲主题：BS001（OH2） Development and the Unique CMC complexity & Process Development for Oncolytic ViroTherapy

Abstract:

OH2 is a genetically engineered oncolytic herpes simplex virus type 2, which has been granted with Breakthrough Therapy Designation in China, and two Orphan Drug Designations and Fast Track Designation in USA.

Unique CMC complexity and full closed-loop process development for oncolytic virotherapy.
New progress in INDs and clinical trials in unresectable melanoma, advanced glioblastoma, soft tissue sarcoma, biliary track cancer, and metastatic esophageal, colorectal cancer in China / USA.
Enhancing the specificity of oncolytic virus intravenous delivery for a novel oncolytic virus technology pathway.

梅双

首席技术官

英百瑞生物

演讲主题：Conjugated-Antibody allogenic NK cell products for the treatment of tumor and non tumor diseases

Abstract:

Imbioray focuses on the world's first universal NK and CAR-NK technology and drug development, targeting the Chinese and overseas markets, committed to leading the development of high-quality natural killer NK cells worldwide, and building a new pattern of natural medicine health. Layout globally, focusing on researching and developing cutting-edge tumors, autoimmune diseases, chronic diseases, and CNS diseases. Imbioray is the world's first enterprise to achieve large-scale, fully enclosed, automated, and modular production processes for NK cells. We have four core technology platforms that collaborate to accelerate source innovation, including a universal NK platform with independent intellectual property rights, an immune domestication NK cell platform, a virus free vector and gene modified CAR coupling platform, and an antibody guided NK connector platform.

李丹

医学部总监

科弈药业

演讲主题：BCMA/CD19 Dual-targeting CAR T (KQ-2003) in Relapsed/Refractory Multiple Myeloma Patients with Extramedullary Diseases

Abstract:

KQ-2003 is a BCMA/CD19 dual-targeting CAR T-cell therapy product, manufactured by Novatim Immune Therapeutics (Zhejiang) Co., Ltd. Utilizing the latest parallel CAR structure to enhance the cell activation and persistence, the dual CAR structure alleviates the antigen evasion of single target therapy. This Investigator-Initiated study (NCT04714827) in China aims to evaluate the safety, tolerability, and preliminary efficacy of KQ-2003 CAR-T cells in patients (pts) with relapsed/refractory multiple myeloma (RRMM), particularly in those with extramedullary disease (EMD). As of the Jul. 25, 2024 clinical cutoff, 23 pts (52.2% male; median age 64 y (range 52-77) with RRMM received KQ-2003, 60.9% (14/23) of the pts had EMD at baseline, 35.7% (5/14) of EMD pts have no hematological measurable indicators, and the response was evaluated by PET-CT. Hematological overall response rate (ORR) was 100.0% (18/18), with a sCR/CR rate of 88.9% (16/18), VGPR rate of 11.1% (2/18). The PET ORR of EMD pts was 85.7% (12/14), 64.3% (9/14) and 50.0% (7/14) of EMD pts had > 50% and > 75% reduction in the size of soft tissue plasmacytomas. CRS and ICANS were observed in 21/23 (91.3%, grade 3/4 4.3%), and 5/23 (21.7%, grade 3/4 8.7%). KQ-2003 showed prompt, profound and durable response in RRMM with acceptable safety. More importantly, it exerted promising efficacy in pts with EMD, including the clinical intractable EM-E, which merits further clinical investigation. Furthermore, the phase 1/2a KQ-2003-CAR-T study in R/R MM (CTR20233309) and R/R POEMS (CTR20242409) is already ongoing in China.

任江涛

总裁兼首席科学官

北恒生物

演讲主题：Developing Donor-derived CD7-targeting Allogeneic CAR-T cells to Treat CD7+ Malignanc

郭雨刚

联合创始人，首席执行官

莱芒生物

演讲主题: Updated Clinical Results of Metabolically Armored CD19 CAR-T Cells at Extremely Low Doses

Abstract：

• Manageable safety profile in r/r DLBCL/B-ALL

• Promising breakthrough efficacy in all dose groups

• Extremely low dose (0.2 x 10⁵ / kg to 1 x 10⁵ / kg), corresponding to 1% - 5% dose of commercial products (2x10⁶ / kg)

杨林

创始人兼首席执行官

博生吉

演讲主题：The Latest Advances in CAR-T Cell Therapy for T-cell Malignancies

金华君

创始人兼首席执行官

君赛生物

演讲主题: Innovation and Breakthrough of TIL Therapy

Abstract:

- TIL therapy has delivered deep and durable responses for patients suffered with advanced cancer.

- TIL therapy is still confronted with several challenges about production process, treatment regimen, and cost.

- Multiple resolutions are in development, using epigenetic or genetic modification, to overcome the challenges of TIL therapy.

刘华

创始人兼首席科学官

星华生物

演讲主题: Allogeneic Universal Ready-to-Use Tumor-Infiltrating Lymphocyte (TIL)

Abstract:

Solid tumors account for more than 90% of all cancers. Treating solid tumors using TILs can produce reproducible, even sustained anti-tumor responses. The advantages of using TIL for anti-tumor adoptive cellular therapy are: multi-target and polyclonal that partially solves the problem of tumor heterogeneity; has mild side effects; with natural chemokine receptors; strong tumor infiltration and rapid homing, and rich in memory T cells.

At present, all TIL cell therapies are autologous immune cells that have many limitations. Xinghua Biotherapeutics is a global pioneer in allogeneic TIL cell therapy. Allogeneic UR-TIL technology greatly expands the scope of clinical applications. Using our tumor organoid production (TOP) platform, and UR-TIL production technology, we are forging a robust immune cell therapy pipeline to combat multiple cancers. Powerful components of TIL manufactured from doner’s PBMC with a purity of CD8+ T cells greater than 95% of the population. Unlimited quantity can be produced as a universal “off-the-shelf” TIL product.

Our method has been evaluated as safe in animal toxicity studies including mice and dogs. Quantitative analysis of GVHD-related protein factors showed that these cytokines were all at low levels after administration of UR-TIL. Allogeneic UR-TIL has been used for solid tumors and hematological malignancies.

Clinical applications of this UR-TIL indicate an excellent clinical safety profile. No CRS or GVHD are observed. Clinical application of UR-TILs is also effective, such as improving life quality, increasing survival time, and inducing tumor regression or disappearance associated with decreased or normalization of tumor biomarkers.

These results indicated that UR-TIL could GVL or GVT therapeutic effects but avoid GVHD toxicity. For cancer patients receiving our UR-TIL, lymphodepleting before TIL infusion is unnecessary; only a low dose of interleukin-2 is needed after TIL infusion. These unique characteristics of our UR-TIL overcome the limitations of autologous TIL-based ACT.

Using our TOP platform and UR-TIL production technology, TIL is manufactured from doner’s PBMC with a purity of CD8+ T cells greater than 95%. Unlimited quantity can be produced as a universal “off-the-shelf” TIL product.
Allogeneic UR-TIL has been used for solid tumors and hematological malignancies. Results of clinical applications of this UR-TIL indicate an excellent clinical safety profile. No CRS or GVHD nor autoimmune diseases were observed.
UR-TILs is also effective, such as improving life quality, increasing survival time, and inducing tumor regression or disappearance associated with decreased or normalization of tumor biomarkers.
These results indicated that UR-TIL could GVL or GVT therapeutic effects but avoid GVHD toxicity. These unique characteristics of our UR-TIL overcome the limitations of autologous TIL-based ACT.

聂小千

高晓飞教授实验室

西湖生物

演讲主题：Harnessing the Power of Red Blood Cells for Treating Cancers

方宏亮

首席研发官

晨泰医药

演讲主题：Leading the Innovation of Developing Next Generation Universal CD-7 CAR-T Cell Therapy against Hematological Malignancies -- WU-CART-007 Development Story

张晓雷

联合创始人兼首席执行官

瓴路药业

李宗海

创始人兼首席执行官

科济生物

郭晓宁

联合创始人兼首席执行官

毕诺济生物

陈功

暨南大学粤港澳中枢神经再生研究院大脑修复中心主任

科学创始人兼首席科学家

神曦生物

演讲主题：大脑原位神经再生技术与临床转化

李凌松

董事长，首席科学家

瑞吉康生物

郭炜

联合创始人兼首席科学官神济昌华

胡新德

联合创始人兼基因治疗设计副总裁

鲸奇生物

演讲主题：Advancements in AAV-based drug development for innovative treatments of Parkinson's disease

Abstract:

Parkinson's disease ranks as the second most prevalent neurodegenerative disorder globally, affecting approximately 10 million individuals worldwide, with around 4 million of those cases in China. The primary pathological hallmark of Parkinson's disease is the degeneration of dopaminergic neurons within the substantia nigra of the midbrain. Despite existing treatments, there remains a significant gap in addressing the therapeutic needs for this condition. In the early stages, medications such as levodopa can provide symptomatic relief, but their efficacy tends to diminish as the disease advances. Moreover, these drugs do not address the fundamental issue of replenishing the lost dopaminergic neurons.

Hence, there is an urgent need for a therapeutic intervention that can stimulate the regeneration of dopaminergic neurons, thereby reversing the disease's progression. Our research has successfully harnessed cell reprogramming techniques to regenerate dopaminergic neurons in vivo, offering a novel therapeutic strategy for the treatment of Parkinson's disease. This breakthrough has the potential to revolutionize the management of mid to late-stage Parkinson's disease, providing hope for patients where traditional treatments have fallen short.

林卿

创始人兼首席执行官

凌意生物

谭青乔

联合创始人兼首席技术官

鼎新基因

金夷

首席执行官

精缮生物

里程碑 - 细胞治疗在自身免疫疾病与炎症领域的突破

主旨演讲嘉宾 Plenary Speakers

Georg A. Schett

德国埃尔朗根-纽伦堡大学

风湿病学和免疫学系主任(FAU)

在线演讲主题 : CAR T Cell Therapy in Autoimmune Diseases

Abstract:

• Principles of CAR T cell therapy and rationale for SLE treatment

• Efficiency and safety in Lupus

• Research agenda

• Understand current unmet needs in treatment of autoimmune disease

• Discuss the potential of CAR T cell therapy in autoimmune diseases

• Present the key datasets on CAR T cell therapy in autoimmune disease

Georg Schett is Professor of Internal Medicine and since 2006 head of the Department of Medicine 3 - Rheumatology and Immunology at Uniklinikum Erlangen, Friedrich-Alexander-Universität Erlangen-Nürnberg in Germany.

Professor Schett graduated from the University of Innsbruck (Austria) in 1994. After his dissertation from medical school, he worked as scientist at the Institute of BioMedical Aging Research of the Austrian Academy of Science in Innsbruck. Two years later, he joined the Department of Medicine at the University of Vienna, where he completed his postgraduate training in Internal Medicine and subsequently in Rheumatology. In 2003 he was promoted to professor of Internal Medicine. Before accepting his position as the chair of the Department of Internal Medicine 3 in Erlangen, he worked as a scientist in the United States of America for one year.

Georg Schett’s scientific work includes a broad spectrum of clinical and immunological issues, particularly the molecular basics of immune-inflammatory diseases. Initially, he investigated the immunology of atherosclerosis and focused on antibody-mediated endothelial cell damage. His research work led to the understanding of the phenomenon of LE-cells in 2007. He was awarded the renowned START Award in 2002 and established a research group for arthritis in Vienna. In 2008, he initiated in collaboration with colleagues the priority program IMMUNOBONE in Germany, funded by the German Research Foundation (DFG). IMMUNOBONE aims to elucidate the interactions between the skeletal and the immune systems. Since 2015, Prof. Schett has led the DFG collaborative research centre 1181 “Checkpoints for Resolution of Inflammation” in Erlangen. Additionally, he is spokesperson of the project METARTHROS, funded by the Federal Ministry of Education and Research, which investigates the impact of the metabolism on arthritis. In 2019, he received funding for the ERC-Synergy grant “4D+ nanoSCOPE Advancing osteoporosis medicine by observing bone microstructure and remodelling using a four-dimensional nanoscope” of which he is spokesperson. 4D nanoSCOPE aims to develop tools and techniques to permit time-resolved imaging and characterization of bone in three spatial dimensions (both in vitro and in vivo), thereby permitting monitoring of bone remodelling and revolutionizing the understanding of bone morphology and its function.

In 2021, Prof. Schett was appointed Vice President for Research at the Friedrich Alexander University of Erlangen-Nuremberg and became a Leopoldina member of the German National Academy of Sciences.

Professor Schett’s scientific work has been honored with several awards, including the Carol-Nachman Prize from Wiesbaden. In March 2023, Prof. Schett received the 2023 “Funding Prize in the Gottfried Wilhelm Leibniz Programme” awarded by the DFG. He has published over 1000 peer-reviewed papers.

陈竹

中国科学技术大学附属第一医院（安徽省立医院）风湿免疫科行政主任

德国埃尔朗根-纽伦堡大学医学博士中国科学技术大学特任研究员安徽省教育厅领军人才特聘教授

演讲主题：CAR-T疗效持久性的关键和挑战；自体CAR-T降低成本的可行性

徐沪济

免疫与炎症全国重点实验室副主任

海军军医大学长征医院大内科主任和风湿免疫科主任

清华大学医学院教授

清华-北大生命科学联合中心临床研究员

演讲主题: Allogeneic CAR-T Cell Therapy in Refractory Autoimmune Diseases

Abstract:

Allogeneic chimeric antigen receptor (CAR)-T cells hold great promises for expanding the accessibility of CAR-T therapy, whereas risks of allograft rejection have hampered its application. Here we genetically engineered healthy donor-derived, CD19-targeting CAR-T cells using CRISPR-Cas9 to address the issue of immune rejection and treated autoimmune diseases with these cells. The infused cells persisted for over three months, achieving complete B cell depletion within 2 weeks of treatment. During the six-month follow-up, we observed deep remission without cytokine release syndrome or other serious adverse events in all cases, primarily shown by the significant improvement in the clinical response index scores for the three diseases we studied respectively and supported by the observations of reversal of inflammation and fibrosis. Our results demonstrate high safety and promising immune modulatory effect of the off-the-shelf CAR-T cells in treating severe refractory autoimmune diseases.

刘明耀

华东师范大学生命医学研究所所长

上海市调控生物学重点实验室主任

邦耀生物董事长

钱浩

联合创始人兼首席执行官

恩瑞恺诺

演讲主题：KN5501 CD19 CAR-NK Cell Therapy to SLE Patients

Abstract:

Our ongoing clinical trial (NCT 06010472) has completed the KN5501 CAR-NK treatment of 20 SLE patients. The 1st patient has reached to 12 months post infusion and the median follow-up duration of 20 patients treated is 6 months. The received data demonstrates the efficacy, safety and persistency of KN5501 CD19 CAR-NK cell therapy to relapsed and refractory SLE patients.

The data demonstrated：

1. The efficacy of KN5501 CD19 CAR-NK, specifically designed for autoimmunity, to SLE patient:

- the complete deletion of B cells and reset of B cell immune system with Naïve B cell reconstitution,

- overall improvements of clinical disease activity, including reduced dsDNA autoantibody, reduced urine protein excretion, hydrarthrosis

disappearance, normalization of autoantibodies and complements etc. and SLEDAI-2K decrease

- observed CR in LLDAS and DORIS assessments

2. No CRS, ICANS, infections or AEs were observed during the infusion/12-month follow-up, which demonstrated a supper safety

characteristic of KN5501 CAR-NK therapy in autoimmunity.

3. The patient of 'disease-free' post infusion for up to 12-month so far indicated a potential of KN5501 drug persistency.

As a breakthrough therapy, KN5501 CD19 CAR-NK cell therapy demonstrated a great potential that can help many patients suffering autoimmune diseases.

郑颂国

上海交大讲席教授

细胞和基因治疗研究院院长

国家重点实验室核心成员

松江科协主席

演讲主题: T Regulatory Cell Therapy in Inflammatory Diseases

Speaker Bio:

Dr. Zheng earned his MD from the Anhui Medical University, MS from Fudan University Shanghai Medical School in China and PhD from the University of Orleans in France. He completed his postdoctoral training at the University of California, Los Angeles and the University of Southern California in United States. Two years later, he joined the Department of Medicine at the University of Southern California as an Assistant Professor and Associate Professor until 2013. He then was promoted as a full professor and director of Rheumatology Research Center at Penn State University Medical School. In 2018, he was appointed as a Ronald L. Whisler Chair and Professor of Medicine, and Chair of Rheumatology Research Center in the Department of Internal Medicine at the Ohio State University. He has returned to China to serve as a Chair Professor of Medicine and Dean at the Shanghai Jiao Tong University since 2023.

Song Guo Zheng’s scientific work includes a broad spectrum of clinical and immunological issues, particularly the therapy basics of immuno-inflammatory diseases. He initially discovered TGF-β induced Tregs in 2002. His research led to the understanding of Treg differentiation and broadened the immunotherapies to various autoimmune and inflammatory diseases. Additionally, Dr. Zheng has been also focusing on the instability and dysfunction Treg cells under inflammatory conditions, for which he discovered that all-trans retinoic acid maintains and enhances natural Treg function even under inflammatory condition. He has significantly contributed to understanding of molecular mechanisms thereby Treg cells result in the pathogenic conversion, eventually promoting immunotolerance in immune systems and immunotherapy for patients with autoimmune and inflammatory diseases.

Dr. Zheng’s work has been well funded by NIH multiple R01, R33, R43, R61, and P30, STAR, ACR, Wright Foundation, Arthritis Foundation in US, as well as China NSFC and National key research and development project from the Department of Science and Technology and others.

He was awarded the renowned Freda Newton Memorial Scholar Award from Arthritis National Research Foundation in 2006, James R. Klinenberg M.D. Award (most outstanding immunologist in Southern California) from Arthritis Foundation in 2007 and ACR Investigator Award from American College of Rheumatology in 2008. He was then awarded the renowned NIH STAR Award in 2016, Outstanding Investigator Award in Pennsylvania (2017), the Edmund Dubois Award (most outstanding rheumatologist in the Asia-Pacific region, 2019), the Outstanding Clinical Immunologist in the big bay area (2019), the Outstanding Scientist Award from ScienceFather (2023) and the Hospital President Award in Shanghai (2023).

He is an elected member of the Henry Kunkel Society and an ANFR fellow.

王立群

创始人，董事长兼首席执行官

星奕昂生物

演讲主题：Developing Next Generation Allogenic iPSC-Derived CAR-NK Cell Therapies for Cancer and Autoimmune Diseases

陈杰

首席医学官

驯鹿生物

演讲主题：CAR T在中枢神经系统自免疾病的临床进展

张鸿声

复旦大学生命科学学院兼职教授

雅科生物, 创始人

演讲主题：Advancing CAR T-Cell Therapy in a New Frontier-Autoimmune Diseases

杨黎明

董事长兼首席科学官

瑞顺生物

演讲主题：全球创新非基因编辑现货通用型DNT细胞疗法的研究进展

肖泽秀

研发中心负责人

虹信生物

演讲主题：虹信生物细胞靶向递送平台（EnC-LNPs）的研究与产业化进展

Speaker Bio:

肖泽秀, 医学博士,毕业于中山大学中山医学院，师从著名免疫学家陈列平教授，博士后师从调节性T细胞权威郑颂国教授，曾任中山大学附属第一医院特聘副研究员，基于共刺激分子的调节作用，在自身免疫疾病转化治疗应用研究10余年，研究成果以第一或者通讯作者发表论文13篇。现任深圳虹信生物科技有限公司董事、研发中心负责人。

吕璐璐

首席执行官

合源生物

刘必佐

首席执行官

西比曼生物

AbelZeta Pharma

沈连军

高级副总裁兼研发负责人

亘喜生物阿斯利康集团成员

钟岚

同济大学教授, 博士生导师

上海市东方医院南院消化内科主任

演讲主题: Advancing Towards the Future: Mesenchymal Stem Cell Therapy as an emerging Treatment for Crohn's Disease

Abstract:

Crohn's disease (CD) is a primary type of inflammatory bowel disease (IBD), predominantly affecting young adults, who face lifelong recurrence and an increased risk of cancer. Clinical manifestations include abdominal pain, diarrhea, abdominal masses, fever, weight loss, and in severe cases, intestinal stenosis, obstruction, intra-abdominal abscesses, fistulas, perianal lesions, as well as extraintestinal complications involving joints, skin, eyes, and oral mucosa (Figure 1), significantly impacting quality of life. In China, the incidence of CD is rapidly increasing, having doubled or tripled over the past 30 years, making it a common gastrointestinal disorder, with a similar rising trend observed globally. Current standard therapies for Crohn's disease (CD) include glucocorticoids, immunomodulators, and biologics. Glucocorticoids can effectively induce remission in CD, but approximately half of patients become steroid-dependent or require surgery. Monoclonal antibodies targeting tumor necrosis factor-alpha, interleukin 12/23, leukocyte adhesion molecules, as well as small molecules like Janus kinases (JAK) inhibitors and Sphingosine 1-phosphate (S1P) receptor modulators represent significant milestones in CD treatment. Despite their superior efficacy over traditional therapies and improved prognosis, overall response rates remain only 32-45%.

Mesenchymal stem cells (MSCs) have become a focal point in research for various diseases due to their regenerative, anti-inflammatory, and immunomodulatory properties. This pesentation will review existing animal experiment results demonstrating its ability to alleviate symptoms of CD colitis, analyze clinical studies showing its effectiveness in treating CD-associated fistulas, and further integrate our own research findings indicating its potential in treating refractory CD. It represents a third option for CD treatment beyond medication and surgery.

同期举办

第八届先进疗法创新峰会2024再生医学-干细胞-细胞外囊泡-外泌体-类器官研发与产业化创新Extracellular Vesicles and Advanced TherapiesStem Cell, Human Organoids and Regenerative Medicines

细胞治疗的发展，不仅为患者带来了新的治疗希望，也为医药行业带来了新的发展机遇。细胞与基因治疗是当今医疗领域最具热度和潜力的发展方向之一，细胞治疗产业也成为各地抢先布局规划的重地。第八届先进疗法创新峰会2024 将于12月19-20日在上海康桥万豪酒店举办。

主要议题：

* 国家干细胞转化资源库和教育部干细胞资源库建设

* 干细胞治疗糖尿病最新研究进展与突破

* 干细胞外泌体在阿尔兹海默症治疗中的研究进展

* 干细胞治疗炎症性肠病研究进展

* 干细胞与退行性骨病研究进展

* 干细胞治疗产品：从中-美双报到NDA

* 胚胎发育与干细胞研究; 胚胎干细胞自我更新和分化

* 干细胞来源的细胞外囊泡的临床应用

* 工程化载药细胞外囊泡 - 细胞外囊泡递送系统技术创新

* 工程化外泌体新药研发的前景与挑战

* 外泌体生物学与肿瘤精准医学

* 3D人脑类器官-神经科学与脑科学的最新研究进展

* 促进肺泡再生, 治疗肺纤维化的新方法

* 视网膜类器官疾病模型与视网膜再生医学

* 肿瘤类器官在癌症研究中的革命性作用

* 类器官芯片在生物医学中的研究进展

* 斑马鱼与类器官在科研中的应用进展

主旨演讲嘉宾 Plenary Speakers

张灏

暨南大学医学院教授

肿瘤精准医学和病理研究所所长

教育部肿瘤分子生物学重点实验室副主任

康九红

同济大学生命科学与技术学院党委书记，特聘教授

国家干细胞转化资源库负责人

上海市信号转导与疾病研究重点实验室副主任

演讲主题: Cell Therapy and National Stem Cell Translational Resource Center Construction

Abstract:

Cell therapy has become an important therapy besides drugs and surgery, and is becoming a hope to many refractory diseases, leading the revolution of medical methods. Stem cell therapy is one of the most attractive cell therapies, which utilizes the multiple differentiation potential of pluripotent stem cells and the paracrine function of stem cells and their differentiated cells, aiming to treat diseases such as degenerative diseases and organ terminal lesions, and has made increasing progress. However, due to the heterogeneity of stem cells, the comparability of research results on the same type of stem cells from different sources is relatively poor, which also leads to significant differences in the therapeutic effects of the same type of stem cells on the same disease. These seriously hinder the research and clinical application of stem cells. To this end, China has established the National Stem Cell Resource Center and the National Stem Cell Translational Resource Center, with the aim of researching and establishing unified stem cell standards and sources, expanding stem cell sharing, researching and promoting universal stem cell technologies, and improving the level of national stem cell research and application. Here, I will share with you the situation of stem cell therapy and the construction of the National Stem Cell Translational Resource Center.

廖顺意

高级研发主管瑞普晨创科技

演讲主题：Unlock the Power of iPSC-Derived Islets for Diabetes Therapy

李维达

同济大学生命科学与技术学院教授

同济大学附属东方医院再生医学研究所研究员

同济大学教育部干性与命运编辑前沿中心PI

演讲主题:Advanced Cell Therapy for Diabetes Using Optimized Human Islet Organoids Derived from Pluripotent Stem Cells

Abstract:

The deficiency of islet β cells is an important pathological factor in diabetes, and achieving the regeneration and protection of these cells is a crucial aspect of diabetes treatment. While clinical cases have demonstrated the potential of human pluripotent stem cell-derived pancreatic β-cells (SC-β cells), this novel cell therapy faces several challenges. Differentiated β-cells fail to respond effectively to blood glucose fluctuations and struggle to adapt to the pathological microenvironment characterized by hyperglycemia and hyperlipidemia in diabetic patients, leading to potential secondary defects. By combining genome editing and small molecule compound treatments, we developed an efficient in vitro differentiation system to generate more mature islet organoids with a structure similar to human primary islets. Furthermore, we have optimized these islet organoids to improve their adaptability to the pathological microenvironment in vivo. This new technology addresses the low survival rate of functional islet organoids after transplantation, achieving better therapeutic outcomes. Our research provides novel insights and therapeutic targets for diabetes treatment.

左为

同济大学医学院/附属东方医院长聘教授

同济大学干细胞研究中心副主任

国家重点研发计划“干细胞及转化研究”首席科学家

上海张江超级器官研发中心（教育部工程研究中心转化基地）主任

浙江省江南干细胞研究院院长

Speaker Bio:

同济大学医学院/附属东方医院长聘教授，同济大学干细胞研究中心副主任，国家重点研发计划“干细胞及转化研究”首席科学家，中国细胞生物学学会细胞与基因治疗分会副会长、党组书记，上海张江超级器官研发中心（教育部工程研究中心转化基地）主任，浙江省江南干细胞研究院院长，上海市青年联合会常务委员（教育界别）。先后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，浙江省高层次人才、江苏省双创专家、江西省双千计划专家等人才计划。担任国自然优青基金、国自然国际合作基金、国家生物药技术创新中心“揭榜挂帅”等项目负责人。兼任呼吸疾病国家重点实验室“南山特聘”教授，国家卫健委移植与免疫重点实验室特聘教授，国家卫健委尘肺病重点实验室特聘教授，中国细胞生物学学会校企双创委员会委员，Gene Therapy、Clinical Precision Medicine等SCI期刊编委等学术职务。

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与功能增强研究。提出器官再生是其发育过程重演的创新理论，开创了R-Clone药品级前体细胞分离扩增技术，负责三项国家卫健委干细胞临床研究备案项目，主导完成世界上首个肺前体细胞移植临床试验。论文成果发表在Nature、Science Translational Medicine、AJRCCM、EMBO Mol Med等期刊上, 单篇通讯作者论文被引超1700次。开发的全球首创肺前体细胞1类新药REGEND001获国家药监局批件进入II期临床，目前已成功完成上百例患者受损肺脏的再生修复治疗。成果入选2018年“中国医药生物技术年度十大进展”，入选2023年欧洲呼吸学会(ERS)发布的全球年度三大重要进展， 2024年团队慢阻肺细胞治疗成果登上《科学》官网首页。个人2018年获国家刊物官方评选为“中国年度十大创新先锋”, 2021年入选中国“科学家创业先锋”榜单Top5, 2023年入选树兰基金会“生命科学新力量年度人物”。

罗剑

同济大学医学院长聘/特聘教授,博导

同济大学附属养志康复医院（上海市阳光康复中心）副院长

上海市残联智能康复辅助器具与技术重点实验室常务副主任

同济大学附属养志康复医院-国际华人骨研学会转化医学联合研究中心主任

演讲主题: Advanced Therapies for Skeletal Diseases

Abstract:

G-protein-coupled receptors (GPCRs or GPRs) are integral membrane proteins involved in the transmission of signals from the extracellular environment to the cytoplasm. A variety of external stimuli, including neurotransmitters, hormones, phospholipids and growth factors, can activate GPCRs. Therefore, GPCRs and their signal transduction pathways represent important specific targets for a variety of physiological functions and therapeutic approaches, ranging from the control of blood pressure, allergic response, kidney function, hormonal disorders, neurological diseases, to bone formation and remodeling. We did GPCRs screening on osteoclastogenesis, we found multiple GPCRs have key roles in osteoclast differentiation. We have reported that leucine-rich repeat-containing G-protein-coupled receptor 4 (LGR4, also called GPR48) is another receptor for RANKL.

汪泱

上海交通大学医学院附属第六人民医院

骨科研究所副所长

演讲主题: Identification of Specific Markers for Human Stem Cell-derived Small Extracellular Vesicles

Abstract:

Pluripotent stem cell-derived small extracellular vesicles (PSC-sEVs) have demonstrated great clinical translational potential in multiple aging-related degenerative diseases. Characterizing the PSC-sEVs is crucial for their clinical applications. However, the specific marker pattern of PSC-sEVs remains unknown. This study aimed to identify the potential specific markers of iPSC-sEVs and ESCs-sEVs. Firstly, we performed proteomic analysis of iPSC-sEVs and ESC-sEVs and found that a group of pluripotency-related proteins were enriched in PSC-sEVs. Further Western Blot verification showed that several proteins including PODXL, OCT4, Dnmt3a and LIN28A were specifically expressed in PSC-sEVs but not non-PSC-sEVs. Next, to facilitate the characterization of PSC-sEVs at single particle resolution, we analysed the surface markers for PSC-sEVs using NanoFCM. PODXL, the only surface protein among the proteins validated above, together with three mostly used specific surface antigens on PSC (SSEA4, Tra-1-60 and Tra-1-81) were selected for NanoFCM analysis. Among them, SSEA4 is a kind of glycolipid while Tra-1-60 and Tra-1-81 are carbohydrate epitopes. By NanoFCM analysis, we showed that PODXL and SSEA4 were enriched in PSC-sEVs with high abundance and high positive rates, but not in non-PSC-sEVs. At last, we verified these results with density gradient ultracentrifugation to exclude the possible contaminated protein particles. In conclusion, our results demonstrated that PODXL, OCT4, Dnmt3a and LIN28A detected by Western Blot analysis and PODXL and SSEA4 detected by NanoFCM can be used for PSC-sEVs identification. These findings would be an important milestone for the clinical application of sEVs derived from pluripotent stem cells.

王刚

上海交通大学医学院附属仁济医院神经科科主任

AD/PD MDT 门诊创建人

演讲主题: Clinical safety and efficacy of allogenic human adipose mesenchymal stromal cells-derived exosomes in patients with mild to moderate Alzheimer's disease: A phase I/II clinical trial

Abstract:

Background: There had been no effective treatments for slowing or reversing Alzheimer’s disease (AD) until now. Our team has established the potential of allogenic human adipose-derived MSCs Exosomes (ahaMSC-Exos) from healthy volunteers for the intranasal treatment of AD mice. After intranasal administration with a dose of 30 μg ahaMSC-Exos per mice, ahaMSC-Exos can be rapidly transported to various brain regions through the olfactory system involving olfactory sensory neurons. In APP/PS1 mice, ahaMSC-Exos displayed neuroprotective effects, increased neurogenesis, and enhanced spatial memory. The ahaMSC-Exos also appeared to decrease Ab deposition and inhibit microglial activation. Importantly, ahaMSC-Exos did not induce morphological alterations in key organs, indicating the safety of the intranasal administration.

Aims: The first 3 arms, drug-intervention, phase I/II clinical trial (Clinical Trial NCT04388982) was conducted to explore the safety and the efficacy of allogenic human adipose MSCs-Exo (ahaMSCs-Exos) in patients with mild to moderate AD.

Methods: The eligible subjects were assigned to 1 of 3 dosage groups, intranasally administrated with ahaMSCs-Exos twice a week for 12 weeks, and underwent follow-up visits at week 16, 24, 36, and 48.

Results: No adverse events were reported. In the medium-dose arm, ADAS-cog scores decreased by 2.33 (1.19) and MoCA-B scores increased by 2.38 (0.58) at week 12 compared to baseline levels, indicating improved cognitive function. Moreover, the ADAS-cog scores in the medium-dose arm decreased continuously by 3.98 points until week 36. There were no significant differences in altered amyloid or tau deposition among the 3 arms, but hippocampal volume shrank less in the medium-dose arm to some extent.

Conclusions: Intranasal administration of ahaMSCs-Exos was safe and well tolerated, and a dose of at least 4×108 particles could be selected for further clinical trials.

Bullet Points of Discussion: Recent advances of MSCs or exosomes in treatment of AD (administered by other modalities).

朱朝

纽伦捷生物

创始人

董事长兼首席执行官

演讲主题: In Situ Trans-differentiation, an Innovative Gene Therapy for Neurological Disorders

李翔

创始人、CEO&CSO

士泽生物

演讲主题: R&D, trial development & industrialization of clinical-grade iPS-derived cell therapy for Parkinson’s disease

Speaker Bio:

李翔博士专注iPS衍生细胞创新药的科学研发及产业化十五年：先后师从iPS衍生细胞领域多位世界级科学家，以第一作者或通讯作者在Cell Stem Cell等领域内顶级国际期刊发表论文十余篇，引用率千余次，研究成果入选Cell Stem Cell杂志封面论文、Cell Stem Cell杂志年度最佳论文等。之后，李翔博士先后在美国帕金森病基金会、美国iPS衍生细胞治疗产业界及国内上市药企全职任职关键研发岗位及高级管理岗位。

自全职归国创立士泽生物后，李翔博士获评了姑苏领军人才、苏州工业园区科技领军人才、上海市春申金字塔杰出人才，担任中国药品监督管理研究会细胞与基因治疗产品监管研究专业委员会委员、中国药学会专业委员会药物安评专委会委员、中国干细胞产业联盟副理事长等；获评2023脑科学与类智能科创新青年30人、2023科创先锋人物、2023中国生物医药U40菁英榜、2022年东方英才计划青年项目、2022年度生物技术行业影响力之星（BioCon Awards）、2022年中国CGT年度影响力企业领袖、全球华人精英Power100、2021中国杰出青年创新30人等领域榜单。

在李翔博士的带领下，士泽生物获批我国首个iPS衍生细胞治疗神经系统疾病的国家级备案临床并完成中国首例iPS衍生细胞药移植治疗帕金森病；士泽生物开发的全球首创iPS衍生细胞治疗渐冻症新药获FDA授予全球孤儿药资格。已完成由启明创投、礼来亚洲基金、红杉中国等领投的多轮数亿元融资。

张芸

联席首席执行官

茵冠生物

演讲主题: Research progress and challenges of mesenchymal stem cell therapy for ischemic stroke

陈丽娟

代理首席执行官兼首席运营官

跃赛生物

演讲主题：USA pre-IND Submission of hPSC Product - Experience Sharing

Abstract:

Growing number of CNS clinical trials using stem cell therapies；

R&D pipeline focus on CNS indications；

Cell therapy product GMP implementation.

朱芳芳

创始人兼首席执行官

血霁生物

演讲主题: 人工“造血”：从干细胞定向分化成血小板的产业化

张宇

首席执行官

中源药业

演讲主题：干细胞治疗产品：从IND到NDA

韩之波

总经理

天津昂赛细胞基因工程有限公司

汉氏联合集团

赵立波

联合创始人兼首席技术官

恩泽康泰

演讲主题: Development and Reflection on the Key Technologies of Drug Delivery Using Engineered Extracellular Vesicles

Abstract:

Extracellular vesicles (EVs) are a cluster of nano-sized vesicles of different sizes, cargo, and surface markers that are secreted into the extracellular environment through a variety of mechanisms. Due to their ability to mediate the delivery of biomolecules, as well as the regulation of the phenotype of recipient cells, EVs are considered as a novel non-viral drug carrier and received a widespread attention. In this presentation, we first provide a brief overview of the development history of EVs’ base therapy. Next, we introduce the advantages and the key technical challenges of EVs as drug delivery system. And finally, based on our own research findings, we discuss the development process and downstream application of EVs engineering technology.

- Technical challenges of EVs as drug delivery system

- Development of EVs engineering platform (Echosome®)

- Massive production of engineered membrane vesicles through cell dehydration

- Future prospective

周国瑛

首席执行官

亦诺微医药

演讲主题：工程化外泌体新药研发的前景与挑战

李杰

副研究员

中国科学院脑科学与智能技术卓越创新中心

（神经科学研究所）刘真教授课题组

演讲主题 : Primate Embryonic Development, Stem Cells, and Embory Model Construction

Abstract:

The gold standard for demonstrating naive pluripotency in mammalian pluripotent stem cells is the generation of chimeric animals through early embryo complementation with homologous cells. While several human naive ES in vitro culture systems have been reported, ethical and resource constraints prevent the verification of in vivo developmental totipotency in these systems. Non-human primates share reproductive and embryonic developmental characteristics more akin to humans, making them ideal models for studying human reproductive and embryonic development. Therefore, we systematically tested various culture conditions to establish naive embryonic stem cells from monkeys and optimized procedures for chimeric embryo culture. This approach resulted in the generation of both an aborted fetus and a live chimeric monkey with a high donor cell contribution. A rigorous characterization pipeline demonstrated that donor cells efficiently incorporated into various tissues, including the gonads and placenta, of the chimeric monkeys, achieving up to 90% efficiency. Additionally, we constructed cynomolgus monkey blastoids using naive monkey ESCs resembling blastocysts in morphology and transcriptomics and further confirmed their developmental potential of developing into gastrula-like structures by in vitro culture and triggering pregnancies with gestation sacs by in vivo transplantation. Such functional experiments are unfeasible with human stem cell-derived blastoids due to ethical constraints and resource limitations. Our research have major implications for studying primate ES naive pluripotency and genetic engineering and provide an important model for studying primate embryonic development in vitro, holding significant potential for investigating diseases related to abnormal embryonic development and for advancing stem cell therapy.

宋洪军

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

再生医学研究所

在线演讲主题: 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.

孟丹

复旦大学基础医学院生理与病理生理学系

党支部书记，副主任，教授，博士生导师

演讲主题: The Role of Transcription Factor BACH1 in Cardiovascular Diseases

Abstract:

Cardiovascular disease (CVD) is the leading cause of mortality globally. The transcription factor BTB and CNC homology1 (BACH1) has been linked to coronary artery disease risk by human genome-wide association studies. However, the role of BACH1 in CVD remains unclear. Our preceding studies have shed light on its multifaceted roles in cardiovascular pathologies. Notably, our findings indicate that BACH1 acts as a mechano-sensor in endothelial cells (ECs), where it is implicated in the dysfunction of ECs and the subsequent progression of atherosclerosis. Furthermore, BACH1 is an essential regulator of vascular smooth muscle cell phenotype switching and neointima formation after artery injury in adult mice. Moreover, in the cardiomyocytes, BACH1's regulation of AT1R expression and the Ca2+/CaMKII signaling pathway positions it as a key factor in the pathogenesis of cardiac hypertrophy. Expanding on its role in CVD, our studies have also demonstrated that the endothelial specifical BACH1 deficiency attenuated hypertension, suggesting its potential as a therapeutic target for blood pressure regulation. Additionally, our findings indicated that BACH1 is involved in pulmonary arterial hypertension, further highlighting its negative regulatory impact on vascular remodeling and disease progression. Moreover, beyond its role in CVD, BACH1 is also associated with metabolic disorders. Our present results revealed that hepatic BACH1 deletion improves hyperglycemia and enhance insulin sensitivity in diabetic male mice, underscoring its critical role in modulating insulin signaling and maintaining glucose homeostasis. Given the crucial role of BACH1 in pathological processes, we employed a virtual screening strategy to identify the specific inhibitor of BACH1. After cellular and disease-level validation, we have identified BI033 as a novel BACH1 inhibitor, which shows promise for treatment of cardiovascular diseases. Collectively, our findings present BACH1 as a promising therapeutic target in both cardiovascular and metabolic disorders.

Keywords: BACH1, cardiovascular diseases, metabolic disorders, inhibitor

韩欣欣

礼升生物创始人

北京大学生命科学华东院

礼升器官再生X实验室

演讲主题: Fast Organoid Culture and Efficient Drug Response with Self Immune Microenvironment and Blood Vessels from Patients

快速培养自带免疫微环境和血管的患者类器官与高效类器官药物响应机理探索

Abstract:

This report will introduce the established tumor organoid systems with unique simplicity and self-contained immune microenvironment, including intrahepatic cholangiocarcinoma, colorectal cancer, liver metastasis of colorectal cancer, ovarian cancer, glioma and skin cancer organoids. The aim is to provide a new research platform for exploring individual precision treatment strategies and drug response mechanisms for cancer patients. By utilizing single-cell sequencing technology, we will analyze the consistency between these tumor organoids and patient tissues. Meanwhile, drug sensitivity screenings will be conducted on tumor organoids to reveal the individual differences in drug response among cancer patients. Furthermore, we will further investigate the correlation between individual differences in tumor tissues and tumor organoids at the single-cell level and drug sensitivity, providing insights for personalized treatment and drug development.

席莹

助理教授研究员博士生导师

上海科技大学生命科学与技术学院

演讲主题：CasRx-based Wnt Activation Promotes Alveolar Regeneration while Ameliorating Pulmonary Fibrosis

Abstract:

Wnt/β-catenin signaling is an attractive target for regenerative medicine. A powerful driver of stem cell activity and hencetissue regeneration, Wnt signaling can promote fibroblast proliferation and activation, leading to fibrosis, while prolongedWnt signaling is potentially carcinogenic. Thus, to harness its therapeutic potential, the activation of Wnt signaling mustbe transient, reversible and tissue-specific. In the lung, Wnt signaling is essential for alveolar stem cell activity andalveolar regeneration, which is impaired in lung fibrosis. Activation of Wnt/β-catenin signaling in lung epithelium mayhave anti-fibrotic effects. Here, we used intratracheal AAV injection to selectively deliver CasRx into lung epithelium,where it reversibly activates Wnt signaling by simultaneously degrading mRNAs encoding negative regulators of Wnt/β-catenin signaling. Interestingly, CasRx mediated Wnt activation specifically in lung epithelium not only promotes alveolartype II cell (AT2) proliferation and alveolar regeneration, but also inhibits lung fibrosis resulted from bleomycin-inducedinjury, relevant in both preventive and therapeutic settings. Our study offers an attractive strategy for treating pulmonaryfibrosis, with general implications for regenerative medicine.

往届回顾

往期回顾

ATMP 2019

ATMP 2020

ATMP 2021

ATMP 2022

ATMP 2023

Carl June

美国宾夕法尼亚大学

帕克癌症免疫疗法研究所主任

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

林欣

清华大学医学院基础医学系主任

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

E-mail: michelle.wang@deliver-consulting.com

END

http://mp.weixin.qq.com/s?__biz=MzI1NTYyOTg1MA==&mid=2247495206&idx=3&sn=4e335da9173e97952deb2831c49acd65

细胞知聊

细胞治疗的时代已经来临，“细胞知聊”致力于向大众普及细胞治疗的相关知识，分享最新资讯，以及前沿科学。