第八届先进疗法创新峰会（ATMP2024)

细胞免疫与基因治疗 

主旨演讲嘉宾 Plenary Speakers

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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?

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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。

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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.

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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

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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

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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.

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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

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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.  

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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

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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.

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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.

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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.

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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.

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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

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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

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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.

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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.

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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.

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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.

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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.

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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.

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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

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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)

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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

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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.

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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.

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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

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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

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张晓雷

联合创始人兼首席执行官

瓴路药业

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李宗海

创始人兼首席执行官

科济生物

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郭晓宁

联合创始人兼首席执行官

毕诺济生物

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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

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李凌松

董事长，首席科学家

瑞吉康生物

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郭炜

联合创始人兼首席科学官神济昌华

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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.

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林卿 

创始人兼首席执行官

凌意生物

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谭青乔

联合创始人兼首席技术官

鼎新基因

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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

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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.

  

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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

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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.

 

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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

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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.

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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.  

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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

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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

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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

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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

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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。

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吕璐璐

首席执行官

合源生物

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刘必佐

首席执行官

西比曼生物

AbelZeta Pharma

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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

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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.