第六届先进治疗产品创新峰会（ATMP2023）

部分已确认嘉宾

刘明耀

邦耀生物

董事长

杨林

博生吉生物

董事长

何霆

艺妙神州

首席执行官

刘雅容

沙砾生物

首席执行官

张曦

百吉生物

首席科学官

张柯

星汉德生物

首席科学官

孙敏敏

易慕峰

首席执行官

黄可

济因生物

首席执行官

潘国华

华夏英泰

首席执行官

胡荣宽

星锐医药

首席执行官

刘新星

鼎新基因

总经理

何晓文

原启生物

首席科学官

任江涛

北恒生物

首席科学官

张雷

星奕昂生物

研发副总裁

     更多嘉宾持续邀请中...

2023部分已确认嘉宾及议题

何苗壮

美国国立卫生研究院

美国国立癌症研究所

教授

终身资深研究员

美国医学和生物工程院会士

演讲主题: Glypicans as Emerging CAR-T Therapeutic Targets in Solid Tumors

Abstract: The emergence of CAR-T cell therapy has provided renewed hope for many patients with B-cell malignancies. However, while CAR-T cells can safely target and destroy lymphoma and leukemia cells without harming other organs in the body, researchers have struggled to identify tumor-specific proteins that can be used in CAR-T cell therapy to target solid cancers without harming healthy organs in the process. In the past 15 years, we have investigated a family of such proteins called glypicans. Glypican-3 (GPC3) and GPC2 are highly expressed in hepatocellular carcinoma and neuroblastoma. More recently, we began to validate GPC1 as a target of CAR-T cell therapy in pancreatic cancer. The update about characterizing these glypicans as CAR-T targets in solid tumors will be presented. The engineering of more potent CAR-T cells using nanobody and protein engineering technologies will also be discussed.

 
田志刚

中国工程院院士

欧洲科学院院士

中国科技大学学术委员会副主任

免疫学研究所所长

医学中心主任

恩凯赛药

创始人兼董事长

演讲主题: SynNK Cells: Beyond CAR-T Cells

Abstract: Natural killer (NK) cells are the first line against tumor with inherited innate receptors with an array of activating and inhibiting surface molecules to recognize the ligands on tumor and finally to activate NK cells for eliminating tumor. Comparing with “CAR”, an artificial receptor using antibody targeting associated antigen on tumor surface, NK receptors (NKR) and their ligands (NKR-Ls) are more valuable always-standing storehouse of surface targets for NKR as tumor-sensors or NKR-Ls as CARs. Though CAR-T become practical in clinic settings, NKR-NK cells, as effector cells, need pay more attention since advantages of NK cells over T cells, such as NK cells express many naturally-holding anti-tumor receptors in addition to modified CAR/NKR. Importantly, we could not stay at the stage of modification of NK cells only with tumor-sensor CAR or NKR, we need make a more intelligent NK cells with multiple functions to combat complicated “sly” tumor. So, synthetic immunology becomes an ideal tool to produce synthetic NK cells (SynNK) with Logic Gates to precise discriminate tumor from normal tissue, and Basic Circuit to prevent exhaustion, ageing and rejection, and to improve tumor-infiltrating and in situ amplification of NK cells, and so on.

 
回爱民

惠正奇医药 

董事长兼CEO

复星荣誉全球合伙人
中国生物医药产业创新与转化联盟副会长

演讲主题: mRNA: COVID-19 Vaccine and Future

Abstract: mRNA technology has gained popularity over the last decade as a versatile tool for developing novel therapeutics. The recent success of coronavirus disease (COVID-19) mRNA vaccine has unlocked the potential of mRNA technology as a powerful therapeutic platform. Two mRNA vaccines, BNT162b2 (BioNTech) and mRNA-1273 (Moderna TX), have acquired authorization from FDA and other authorities across the world that are currently being used to prevent COVID-19. mRNA vaccines have good efficacy and safety as demonstrated in the various phase III trials and real world studies. Knowledge gained from these trials and versatile therapeutic potential of the mRNA can be applied for the development of vaccine for the infectious diseases, cancer treatment and therapeutics for other diseases. In this presentation, we focus on succesful development experiences of BNT162b2 mRNA vaccine against COVID-19 and an overview on the therapeutic aspect of mRNA technology for other therapeutic areas in future.

 
沈海法

斯微生物 

联合创始人

首席技术官

演讲主题: Application of New Technologies in mRNA-based cancer therapeutics

 
许田

复星医药首席

科学顾问

复星领智董事长

西湖大学副校长

演讲主题: The Challenge and Opportunity of Gene and Cell Therapy

Abstract: Gene and cell therapy holds great promise for the treatment of a wide range of human diseases from cancer to rare genetic disorders. Recent progress has significantly advanced our ability to overcome the challenges including safety and delivery vehicle. The efforts at GeCell Therapeutics using modified piggyBac transposon and NK cells for cancer therapy and gene therapy of skin disease will be discussed.

 
施霖宇

辉大基因联合创始人

兼首席科学官

中科院神经科学研究所副高级研究员

演讲主题: Development of Gene editing Tools for Treating Rare Diseases 

 
陈功 

暨南大学大脑修复

中心主任 

NeuExcell 神曦集团

创始人, 首席科学家

演讲主题: 运用神经再生型基因疗法治疗退行性疾病

 
周静敏

鲸奇生物

联合创始人

兼首席执行官

演讲主题: Treatment of neurodegenerative diseases with AAV and its CMC

Abstract: In situ cell reprogramming offers the hope of a regenerative therapeutic approach to numerous neurodegenerative diseases. Using AAVs to deliver reprogramming factors to glial cells can result in the transdifferentiation of these cells into neurons and the replenishment of specific neuronal cell populations lost in a given disorder. In a chemically induced non-human primate model of PD, treatment with AAV-GM101 has led to improved motor behavior, increased dopamine concentration in the CSF, and enhanced dopamine signal within the striatum as measured by PET-CT. To achieve such significant improvement, the quality of AAV is critical, we’ll also discuss the chemistry, manufacturing and controls (CMC) of AAV.

 
郭晓宁

纽福斯首席医学官 

演讲主题待更新

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赵新平

中因科技

首席科学官

演讲主题: Gene Therapy for BCD- 眼科基因治疗药物的研发方向

Abstract: This presentation briefly describes the history and current status of gene therapy with particular focus on ophthalmology and introduces the overall strategy of Chinagene for developing gene therapy drugs for ocular diseases. One of the Chinagene’s pipelines, ZVS101e is designed to treat BCD patients and has shown excellent safety and efficacy profile in both preclinical studies and clinical trials. The presentation discusses the current trend and future direction of gene therapy. A wave of consolidation and MA is anticipated in the gene therapy industry. Possible solutions to address challenges in R&D of gene therapy drugs are also reviewed and proposed.

 
王海峰

无锡科金生物

创始人, 首席执行官

演讲主题: Development of innovative Ex Vivo Gene Editing gene cell therapy technology

 
马一介

北海康成

全球商务拓展和战略

合作主管

高级总监

演讲主题: R&D and Business Strategy for Translational Innovation in Gene Therapy – A CANbridge’s Perspectives

Abstract: One of the major trends in modern medicine is the increasing role of industry partnerships in driving innovation. Academia has traditionally been the main driving force of breakthroughs and innovations in medical sciences in the past. However, due to the open-ended, explorative nature of academic research, the lack of commercial perspectives, and the complex, capital-heavy process of drug development, academia alone cannot meet the growing demand for better healthcare solutions. Therefore, industry partnerships have emerged as a way to drive innovation in modern medicine by providing access to capital, expertise, infrastructure, and markets that are essential for translating research into bedside applications to benefit patients. CANbridge is an innovation-driven biopharma committed to developing transformative therapies for patients. We will share our story and result-oriented strategy of gene therapy innovation and discuss challenges along the way.

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王永忠

锐正基因

董事长, 首席执行官

演讲主题: 开发全球Best-in-class 的体内基因编辑产品

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牟晓盾

正序生物

首席执行官

演讲主题: 应用创新型变形式碱基编辑技术 (tBE) 开发同类最好 (Best-in-Class) 基因编辑药物  

演讲摘要: 基因编辑技术在细胞和基因治疗领域的应用发展迅速，目前全球范围内有几项通过in vivo或ex vivo编辑方式针对不同的适应症的治疗性应用已经进入临床研究甚至BLA阶段，然而他们目前使用的基因编辑工具仍然存在导致染色体异常、脱靶等安全风险。

- 正序生物自主研发的创新型变形式碱基编辑器tBE（transformer Base Editor）可实现较高的安全控制，与CRISPR/Cas9 和传统的碱基编辑器相比，tBE不会造成DNA双链断裂，具有更高的靶向编辑效率而且不会造成脱靶突变，具有更低的细胞毒性，这些安全性和有效性上的优势使其有望成为同类最好的碱基编辑工具。

- 正序生物首条管线CS-101针对β-地中海贫血症和镰刀型贫血症，以tBE技术通过ex vivo的方式精准编辑患者造血干细胞，在临床前研究实验中实现了高效靶向编辑效率的同时，也消除了脱靶突变的风险。CS-101即将进入临床阶段，有很大潜力成为治疗β-地中海贫血症和镰状细胞病的best-in-class基因编辑药物。

- tBE系统也适用于AAV、LNP等体内递送方式。采用经商业验证过的LNP或AAV将tBE系统递送至小鼠肝脏中，实现了高效的靶向编辑效率，同时无脱靶突变。此次我们会介绍关于tBE系统的更多信息和管线进展。

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何春艳

新芽基因

创始人兼首席执行官

演讲主题: DMD Base Editing Drug Development

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姜儒鸿 

ASC Therapeutics

联合创始人

兼首席执行官

演讲主题: Development of AAV-based gene and gene editing therapeutic products for Hemophilia A

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Bose Kalampanayil

纽福斯生物科技

首席技术官 

演讲主题: Neurophth's,“Center of Excellence" in Gene Therapy Development, Manufacturing and Analytical Testing Capabilities with a Diverse Platform, including AAV-based Gene Replacement Therapy Product for Leber's Hereditary Optic Neuropathy

- Global development and manufacturing of Adeno-Associated Virus serotype 2 (AAV2) containing human mitochondrial ND4 gene (NR082) to meet international regulatory requirements

- NR082 has granted orphan drug designation (ODD) from China NMPA, EMA and US FDA. In addition, the NR082 program has completed Phase 1/2 and Phase 3 clinical trials in China and initiated Phase 1/2 trial in USA

- Chemistry Manufacturing and Controls (CMC) for gene therapies is one of the biggest hurdles for achieving global regulatory approval. Here we present the risks, challenges and lessons learned for Scale Up of Plasmid and AAV manufacturing processes

- We highlight many components of QbD, which are used to assess the process and minimize risks, from quality target product profile (QTPP) to Process Validation and PPQ Batches, as preparation for late-state development and BLA-enabling activities

- Discuss Neurophth’s, “Center of Excellence” in Gene Therapy Development, Manufacturing and Analytical Testing Capabilities with the state-of-the-art facility, equipped to handle the complexity of a diverse gene therapy pipelines

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Ales Štrancar

BIA Separations

创始人兼首席执行官

演讲主题: 制造更安全的AAV载体 

Manufacturing of safer AAV vectors

- Recent clinical trials revealed the immune response to AAV vectors might be a bottleneck in AAV gene therapy

- One of the key reasons for the immune response is AAV purity. Improved manufacturing, especially purification process, is therefore mandatory for safer gene therapy

- In addition to hc proteins and hc DNA, removal of empty and partial AAV capsids, infectious viruses and endotoxin will be presented in this paper

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张春

吉恒基因创始人

中国科学院苏州

生物医学工程技术

研究所研究员

演讲主题: What is holding back the development of AAV gene therapy?

Abstract: AAV gene therapy has become the hope to cure many human diseases, especially genetic disorders. However, the development of AAV gene therapy is facing many challenges which prevent success of the AAV gene therapy. Before these challenges are solved, very little progress can be made. Some challenges include:

- rAAV vector quality

- rAAV vector gene expression efficiency

- rAAV vector integration issues

- rAAV packaging for large genes

- rAAV vector for gene editing

Suzhou genehealth biotechnology company focuses on research to address these challenges. Significant breakthroughs have been made and innovative rAAV technologies have been developed. Some of these challenges have been solved which would greatly facilitate the success of AAV gene therapy.

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张艳君

中吉智药生物技术

研发总监

演讲主题: 慢病毒载体造血干细胞基因治疗

HSPC Gene Therapy with Lentiviral Vector.

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方日国

博雅辑因研发副总裁

干细胞平台

演讲主题待更新

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吕璐璐

首席执行官

合源生物

演讲主题: Innovative R&D and the Commercialization Road of CAR-T Cell Therapy for the Treatment of B-Cell Acute Lymphoblastic Leukemia

- Epidemiology, Treatment, and Great Umet Clinical Needs of B-ALL CAR-T Cell Therapy for the Treatment of B-Cell Acut

Lymphoblastic Leukemia in global scale

- The Innovative R&D Road of Inaticabtagene Autoleucel Injection (CNCT19), a Chinese CAR-T Cell Therapy Product

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姚树元

安诺瓴路

首席执行官

演讲主题: An Industry Perspective on Cell Therapy CMC

演讲要点

- CMC Capacity Building of Cell Therapy

- Product Development of Cell Therapy

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王永增

合源生物

首席技术官

演讲主题: Challenges and Approaches of Comparability Study in Autologous CAR-T Cell Therapy

Abstract: Autologous CAR-T cell therapy is featured with personal, living drug in vitro and in vivo, sterile, and complex characteristics, nevertheless, as drug, changes in materials, process, equipment, method, and sometimes manufacturing site are unavoidable along product development, it is challenging to assess the risks of such changes, even more so to rationalize comparability of pre- and post-change. This presentation focuses on explore the approaches of comparability study in autologous CAR-T cell therapy. The key points include:

- Types of changes

- Risk assessment of changes

- Quality attribute study

- Analytical comparability study

- Non-clinical study

- Clinical bridging

- Regulatory consultation

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张红兵

优瑞科

研发副总裁

演讲主题: Overcoming the Challenge of T-cell Immunotherapy in Solid Tumors

Abstract: CAR T-cell therapy has been highly successful in treating hematological malignancies, and as a result, there is growing interest in testing this technology for solid tumors. However, the biology of solid tumors is more complex than that of hematological malignancies. There are two major obstacles in T cell immunotherapy for solid tumors: difficulty of engineered T cell-infiltration into solid tumors, and availability of ideal target antigens. At Eureka Therapeutics, we have developed proprietary technologies to address these challenges. Our ARTEMIS technology demonstrated enhanced T-cell infiltration into solid tumors, and our T Cell Receptor-mimic antibody (TCRm) platform broadens the range of cancer specific antigens that can be targeted, including those that are normally expressed intracellularly. We will present an update on our case studies using these technologies to treat liver cancer (HCC)

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赵阳兵

优替济生

创始人兼首席科学官

演讲主题: Challenges and Solutions for CAR-T Treatment of Solid Tumors

Abstract: Despite impressive clinical efficacy of T cells engineered to express chimeric antigen receptors (CAR) for some hematological cancers, the current applications of CAR T cell therapy, especially for treating solid tumors, are limited by some major challenges, such as the lack of safe cancer specific targets, highly heterogeneities of the tumors and the tumor microenvironment (TME).  To make breakthrough in treating solid tumors, strategies to solve all these challenges are required. We have developed a (CAR or TCR) T cell engineering strategy by incorporating a LACO-Stim molecule for the aim of enhancing engineered T cells’ abilities to counteract with TEM and, at the same time, orchestrating both innate and adaptive immunities against tumors of the patients. This synergistic combination effective CART therapy with tumor vaccine has been proved in pre-clinical syngeneic mouse tumor models and early clinical trials.

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周鹏辉

泛恩生物

创始人兼首席技术官 

演讲主题: Challenges and Opportunities in T Cell Therapy for Solid Tumors

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李懿

瑅安生物

创始人兼首席科学官

演讲主题: The Application of Human Professional Antigen Receptors for the Development of Cell Therapy

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何霆

艺妙神州

创始人兼首席执行官

演讲主题: Phase I Study of a BCMA-Directed CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma Manufactured with the InstanCART platform

Abstract: Although Chimeric antigen receptor (CAR)-T cell therapy has achieved effective efficacy in patients with multiple myeloma (MM), the lack of durable response and high cost are still main directions of improvement of CAR-T cell therapy. The traditional process of autologous CAR-T cells needs to be expanded and cultured in vitro for a long time (9-14 days). Here, we developed an efficient BCMA-targeting CAR-T cell production process called InstanCART platform, which can be produced within 3 days. We evaluated the clinical safety and efficacy of the InstanCART cells in the treatment of relapsed/refractory MM (RRMM) in a Phase I clinical trials. Initial data from this Phase I study demonstrate that low doses of BCMA CAR-T cells manufactured by InstanCART production processes have encouraging clinical activity and a manageable safety profile in patients with RRMM. InstanCART cells expand rapidly in vivo, persist at relatively high levels for prolonged periods, and demonstrate better efficacy.

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邹勇

驯鹿生物

CMC副总裁

演讲主题 药品全生命周期下的CMC

MAH制度全面实施后，药品上市许可持有人须对药物研发、临床研究、生产销售、不良反应等全生命周期负责，而CMC是药品生命周期里的非常关键的一环。从药品全生命周期的角度如何看待和开展CMC工作，使其能够更好地为药品全生命周期服务，确保安全、有效的药物得以实现。

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张颖

中盛溯源

联合创始人

首席技术官

演讲主题: CMC and Non-clinical Studies of iPSC-derived Cell Drugs

Abstract: Development of multiple iPSC-derived cell drugs is on the fast track around the globe. We at Nuwacell are investing in the utilization of iPSC-derived mesenchymal stromal cells, natural killer cells, dopaminergic neural progenitors, and pancreatic beta cells to treat human diseases. To make these cells on an industrial scale with controllable qualities, we’ve developed unique CMC for each cell product, including the clinical-grade iPSC itself. Thus far, two of our products had received regulatory clearances into Phase I clinical trials in China.

- CMC of clinical-grade iPSC

- CMC and non-clinical studies of iPSC-derived MSC

- CMC and non-clinical studies of iPSC-derived NK cells

- CMC and non-clinical studies of iPSC-derived imDAP

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杨黎明

广东瑞顺生物技术

董事长兼首席科学官

演讲主题: The off-the-shelf Allogeneic DNT Cells in Immunotherapy

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胡适

海军军医大学

基础医学院副教授

演讲主题: Progress of Therapeutic Engineered CAR-T Cell-derived Extracellular Vesicle

Abstract: Engineered T cells have attracted substantial attention in recent years as an emerging therapy for hematological and non-hematological malignancies. Despite the rapid and robust clinical responses, unexpected toxicity, such as cytokine release syndrome, still remains a major concern in this therapy. Moreover, the intrinsic ability of tumors to evade immune responses could lead to treatment failure especially in patients with solid tumors. These obstacles together highlight a need to improve current Engineered T therapy. Exosomes are small extracellular vesicles secreted by almost all cell types and have the capability of trafficking cargos to mediate many physiological，pathophysiological processes. Therefore, researchers have been trying to utilize exosomes as highly effective carriers to deliver various therapeutic agents to target cells. We reported that Engineered immune cells release extracellular vesicles with the stimulation of antigens, mostly in the form of exosomes that carry synthetic receptors on their surface. These exosomes express a high level of cytotoxic molecules and therefore inhibit tumor growth in an antigen-specific manner. Besides, exosomes do not express programmed cell death protein 1 (PD1), and thus could circumvent the immunosuppressive mechanism caused by tumor cells. More importantly, the administration of exosomes exhibited lower risk compared with engineered T therapy in a preclinical in vivo model of cytokine release syndrome. All these advantages of engineered immune cell derived exosomes suggest that they may be promising therapeutic agents against tumors.

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韩研妍

恒瑞源正

首席科学家

演讲主题: An HLA-class II restricted HPV18 E7 specific TCR cloned from a long-term surviving cervical cancer patient induces tumor remission in murine model

Abstract: T cell receptor (TCR)-engineered T cell therapy is a promising approach for the treatment of solid malignancy, with multiple clinical trials reporting impressive responses using affinity-matured TCRs. However, artificially mutated TCRs can increase the risk of off-target toxicities due to unexpected cross-reactions, which limits the overall safety of this approach. To tackle this challenge, we have developed a novel strategy for cloning tumor-specific endogenous therapeutic TCRs from long-term surviving patients who have responded to immunotherapy. Specifically, we have identified a TCR (10F04) that is specific to human papillomavirus type 18 (HPV18) E7 and restricted to human leukocyte antigen (HLA) class II molecular from the peripheral blood of a patient with HPV-positive metastatic cervical cancer who received multiple antigens stimulating cellular therapy (MASCT) and exhibited sustained T-cell immunological responses. Upon transduction into human T cells, the 10F04 TCR demonstrated robust antitumor activity in both in vitro and in vivo models. Notably, the TCR effectively redirected both CD4 and CD8 T cells to specifically recognize tumor cells and induced multiple cytokine secretion along with durable antitumor activity. Importantly, no cross-reactivity was detected. As a result, this TCR is currently being investigated in a clinical trial for treating HPV18-positive cancers. Our approach of cloning tumor-specific and safe endogenous therapeutic TCRs from long-term surviving patients offers a promising strategy for developing safe and effective TCR-engineered T T-cell therapies for solid tumors. These findings provide important insights into the potential of HLA class II-restricted TCR-T therapy as a novel cancer treatment modality.

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李华顺

阿思科力

董事长

演讲主题: The Development Progress of CAR-NK Cell Therapy

Abstract: The remarkable success of CD19 CAR-T in the treatment of B lymphocytic leukemia and lymphoma promotes Ascle Therapeutics to explore other cell types to treat solid tumors.  Natural killer cells is a major type of innate cells that bridge innate immunity to acquired immunity to defend broad spectrum of pathogenic microbes and function as central commanding cells to initiate systemic activation of immune system to eliminate transformed or senescent cells.  Ascle team has been using natural killer cell line NK-92 as a platform to tap into its potential as cancer killer carrier and has developed several lines of products.  One of the CAR-NK products has successfully treated late-stage cancer patients with a remarkable 80% disease control rate.  In addition, selected naval cord blook NK cells and mRNA enabling CAR-NK cells have dramatically reduced or eliminated the tumor load in animal model.  Ascle Therapeutics is well on the way to bring the products to the market for cancer patients with innovative technologies.

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孙艳

上海细胞治疗集团

共同创始人

集团首席运营官

上海细胞治疗集团

药物技术有限公司总裁

演讲主题: Practical exploration of nanobody armed CAR-T in breakthrough of solid tumor immunotherapy

Abstract: With the launch of several kinds of chimeric antigen-receptor (CAR) -T cells both domestically and internationally, outstanding values of CAR-T cells have largely verified in treatment of hematological carcinomas. However, treatment with CAR-T cells alone in solid tumor faces great challenges with poor efficacy. Given that lack of tumor specific antigen, high heterogeneity and immunosuppressive microenvironment in solid tumors, we explore a unique nanobody armored CAR-T cell (NAC) which elicits the dual anti-tumor effect of targeting tumor antigen and changing immunosuppressive tumor microenvironment for solid tumor therapy. Autocrine PD1 nanobody-targeting mesothelin NAC has recently been approved by the National Food and Drug Administration for phase I/II clinical trials. Previous IIT studies have shown that the NAC cells achieve a therapeutic breakthrough in the target population with advanced solid tumors. While NAC targets tumors to produce killing effects, PD-1 nanobodies are continuously secreted locally in tumors to relieve immunosuppression. On the one hand, nanobodies protect CAR-T from tumor microenvironment immunosuppression and maintain tumor killing effect, on the other hand, relieve the immunosuppression of tumor-infiltrating T cells (TILs) in vivo, promote TIL to develop anti-tumor immune response extended to different tumor-associated antigens, overcome the obstacles caused by tumor heterogeneity and immunosuppressive microenvironment, and further improve the long-lasting efficacy and safety of CAR-T cells.

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李志远

普米斯生物技术

功能生物学

与细胞治疗总监

演讲主题: Leading the Innovation of Developing Next Generation Cell Therapy Products against Solid Tumor via CAB-T Cell Therapy Platform

Abstract: In the past decade, immunotherapy has achieved unprecedented success in providing the complete remission rate of hematological tumors. Since 2017, 6 CAR-T products targeting CD19 or BCMA have been successfully marketed and approved for the treatment of hematological malignancies. However, unlike tumor infiltrated lymphocytes (TIL) therapy and TCR-T cell therapy, which have been proved for their effectiveness for treating solid tumors, CAR-T therapy has not yet demonstrated significant clinical efficacy, and there is still an unmet need to develop an HLA independent cellular therapeutic regimen for treating solid tumors with good clinical efficacy and manageable clinical side effects. Biotheus’ CAB-T is a novel cell therapy platform combining the advantages of CAR-T and using T cells as an anti-CD3 T cell engager delivery source. Here, we will present a comprehensive overview of our CAB-T programs with proof of safety and efficacy in multiple in vivo efficacy studies and investigator initiated trials (IIT).

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张同存

波睿达

董事长兼首席执行官

演讲主题: CD30 CAR-T cell therapy for relapsed/refractory CD30+ lymphoma patients

Abstract: CD30 is a membrane protein that is constitutively overexpressed on all stages of cells in classical Hodgkin lymphoma (HL) and anaplastic large-cell lymphoma (ALCL) with minimal/negligible expression on normal cells, rendering its an ideal target for CAR-T cell therapy to treat relapsed/refractory HL/ALCL patients. Thus, we designed a new third-generation anti-CD30 CAR, and conducted a pilot study of 15 r/r HL/ACLC patients to test the efficacy and safety of CD30 CAR-T cell therapy, resulting in 93.3% ORR, 86.6% CR, and none CRS/ICNAS/CRES over grade 3. Thereby, a Phase I registered clinical trial was carried out in a classic 3 + 3 dose escalation manner to further validate the safety and efficacy of CD30 CAR-T cell therapy. Expectedly, 100% ORR and 83.3% CR were observed in median/high doses, while none Grade ≥ 3 CRS was found in all 9 patients. Taken together, these clinical studies demonstrated CD30 CAR-T cell therapy as a safe and effective treatment for relapsed/refractory CD30+ lymphoma patients.

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张宇

中源药业

首席执行官

演讲主题: Key Considerations during Cell Therapy Product Development

- Advances of cell therapy products worldwide

- Cell therapy products: a pearl on the crown in pharmaceuticals industry

- Key considerations in CTP development

- Cell resource selection

- CMC in IND vs in BLA/NDA

- IIT study vs IND trial

- License-out vs in-house development

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张长风

上药生物

注册总监

演讲主题: Regulatory Trends in Investigator-Initiated Trials (IITs) of Cell and Gene Therapies

Abstract: Investigator-initiated trials (IITs) are of importance in cell and gene therapies.  However, few regulatory guidelines are available compared with those for IND trials. Recently, a draft guidance document applying to the IIT trials of somatic cell products was issued by the National Health Commission.  Here the speaker will review the newly issued draft guidance and related active or draft guidance documents dated 2019 and 2015 for cell products, and illustrate the emerging regulatory trends in IIT trials. Finally, recommendations for potential IIT sponsors will be discussed.

- Key points in the newly issued draft guidance document

- The emerging regulatory trends in IIT trials of cell products

- The relationship between IIT and IND trials from a regulatory perspective

- The dos and don’ts for potential IIT sponsors

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陆佩华

陆道培医院医疗

执行院长

北京陆道培血液病

研究院院长

演讲主题: The advances and challenges of CD7 CAR-T Therapy in treating T-cell malignancies

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杨林

博生吉创始人

董事长兼CEO 

演讲主题: PersonGen Potential First-in-class CAR-T for Unmet Clinical Needs

A number of CAR-T cell drugs have been approved for marketing around the world, and most of them have achieved beautiful commercial sales data. But these marketed CAR-T products focus on B-cell-derived malignancies. The research and development of CAR-T cell drugs for T-cell-derived malignant tumors faces many technical and clinical obstacles, and its development is relatively lagging behind. Through innovative research and development, PersonGen successfully developed the world's first CAR-T product for malignant T cell tumors, and achieved excellent performance in clinical trials.

- Innovative CAR-T target and indications

- T cell derived malignancies

- Fully automated manufacturing process

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孙敏敏

易慕峰创始人

首席执行官

演讲主题: Empower CAR-T Cells to Treat Solid Tumors

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”.

- Pipeline and preliminary clinical progress

- Innovative CAR-T technology platforms

- The prospect of CAR-T industry

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黄可

济因生物

首席执行官

演讲主题:  In vivo CAR-T - The Next Generation of Universal Cell Therapy

Personalized CAR-T cell therapies have provided great promise in treating leukemia and even some types of solid tumor, while the high costs of traditional CAR-T manufacture limited their clinical applications and commercialization. So far, novel strategies have emerged in the generating off-the-shelf/universal cell therapies, including UCAR-T, PB/UCB CAR-NK, iPS CAR-NK and in vivo CAR-T. Compared to the UCAR-T and CAR-NKs, the in vivo CAR-T simplified the immune cell manufacture by directly generating CAR-T in vivo, and meanwhile harnessing the persistence of autologous T cells in patients. We have developed a VivoExpress system to precisely and efficiently deliver the CAR payload into the non-divided T cells to generate the in vivo CAR-T. These cells could efficiently eliminate the target tumor cells in vitro and in vivo. While the costs of in vivo CAR-T could be less than 1/10 of the traditional CAR-T. In the presentation, we will give an overview on the current progresses, challenges and potential benefits of in vivo CAR-T.

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周国庆

荣瑞医药

创始人

演讲主题: Oncolytic Virus Vaccine Drives Combination Immunotherapy

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陈锐

厚无生物

研发副总裁

演讲主题: Strategies for Developing Next Generation TIL Therapy

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王文博

立凌生物

创始人兼首席执行官

演讲主题: From Antibodies to T Cell Receptors: the Next Wave of Innovative Biologics

Abstract: Antibodies as the core component of humoral immunity have been employed to develop novel biologics including cellular immunotherapy, mAbs and its derivatives for almost 50 years. As the counterpart of antibodies, TCRs as the most important functional molecules of cellullar immunity are also developed for novel therapeutic modalities including TCR-T cell therapy, TCR bispecifics, etc.. Because of unique features of TCRs, challenges and opportunities both exist in the way for successfully developing commercialized TCR based therapies. Nevertheless, what can be foreseen is that successful stories and failure examples will be recorded and people who are leading this area will be remembered in the history of drug development.

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高晓飞

西湖生物

创始人兼总裁 

西湖大学

生命科学学院PI

演讲主题: Overcoming Immunotherapy Resistance by Therapeutic Red Blood Cells

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高基民

温州医科大学教授

演讲主题: Pilot Trials of Armored Nectin4/NKG2DL/FAP-targeted Hi-TCR-T in the Treatment of Solid Tumors

Abstract: Our armored HLA independent (Hi)-TCR-T integrates the features of TCR-T and the fourth generation CAR-T such as multi-targeting and immune regulatory factors-secreting. The Hi-TCR-T can effectively reprogram an intact TCR complex to recognize tumor surface antigens and thus induce more efficient anti-tumor responses and cause little cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) compared with classic CAR-T. The armored Hi-TCR-T cells recognize highly expressed surface antigens on tumor cells, activating all of the subunits of the complete TCR machinery to generate a broad and controlled response allowing for more potent tumor cell killing, faster migration to the tumor, and longer persistence so as to have a superior therapeutic index relative to CAR-T cells. Currently, our clinical trials are being carried out to treat hematological and solid malignancies with preliminary studies showing that the armored Hi-TCR-T outperformed CAR-T with increased anti-tumor efficacy and reduced toxicity. For example, We have been carrying out the first-in-class clinical trial of Nectin4/NKG2DL/FAP-targeted Hi-TCR-T cells secreting IL7 and CCL21 for therapy of relapsed/refractory advanced solid tumors such as non-small-cell lung cancer and liver cancer with impressive efficacy with only fever in absence of CRS and ICANS.

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王汉明

武汉滨会生物科技

副总裁

演讲主题待更新

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张骥

杭州云心质力

COO, 联合创始人

演讲主题待更新

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蒋云

清华工研院

细胞与基因治疗

创新中心负责人

演讲主题: Analysis and Research Examples of the Development of Nucleic Acid Drug Industry

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赵春林

安龙生物创始人

演讲主题: 安龙生物在核酸药物开发的探索

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张鸿声

雅科生物董事长

兼首席执行官 

演讲主题: Developing Cancer Treatment with CAR-T Cell Therapies: A Biotechnology Research Company’s Perspective

Abstract: CAR-T cell therapy is a revolutionary new pillar in cancer treatment. Although treatment with CAR-T cells has produced remarkable clinical responses with certain subsets of B cell leukemia or lymphoma, many challenges limit the therapeutic efficacy of CAR-T cells in solid tumors and hematological malignancies. Barriers to effective CAR-T cell therapy include severe life-threatening toxicities, modest anti-tumor activity, antigen escape, restricted trafficking, and limited tumor infiltration. In addition, the host and tumor microenvironment interactions with CAR-T cells critically alter CAR-T cell function. Furthermore, a complex workforce is required to develop and implement these treatments. In order to overcome these significant challenges, innovative strategies and approaches to engineer more powerful CAR-T cells with improved anti-tumor activity and decreased toxicity are necessary. In this presentation, we will discuss our company’s strategy for developing CAR-T cell therapies and present the results from clinical trials in the treatment of hematological malignancies, such as leukemia, lymphoma, and multiple myeloma. We will also present our recent endeavor in developing advanced CD7 CAR-T technology that overcomes fratricide for the treatment of T-cell leukemia and lymphoma. This technology was recognized as one of the ten greatest advances in Chinese Hematology in 2021.

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李斌

余㵑学者

上海交大特聘教授 

上海市免疫学研究所

科研副所长

生命科学学院PI

演讲主题: Functional Stability of FOXP3+ Treg subsets and their clinical applications

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方宏亮

晨泰医药

首席开发官

演讲主题: NK Cell Therapy against Solid Tumor: Memory NK Cells against Cancer

Abstract: Autologous CAR-Ts are great, But many challenges remain. Natural Killer (NK) cells are the most common type of naturally allogeneic immune cells, and more recently, use of NK cells that naturally exclude a typical TCR and can be used off-the-shelf. NK Cells achieved Similar Initial Efficacy in BCL to that Observed with Autologous CAR-Ts and appeared to have Improved Safety. Achieving activity in solid tumors would be transformational for NK Cells and the cellular therapeutic space more broadly. Memory-Like Nk cells was identified in 2009, which showed long-lived, super-charged NK cells with enhanced ability to kill cancer cells. Cellular therapeutics have yet to reach their full potential in solid tumors. However, Wu-NK cells have several compelling features: (1) Increases cytotoxicity capacity by engaging ADCC; (2) Increased trafficking, penetration and persistence in TME through sequential dosing-coating of tumor with Ab prior to NK cell administration; (3) Potential decrease T cell rejection due to impaired T cell function in TME; (4) May salvage CPI failure as well as synergize with CPI. We view signals of activity in solid tumors as potentially transformative for the space.

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胡璧梁

楚天思为康 

首席科学家

演讲主题: Automatic equipment in transforming the process of CART production

Abstract: Chimeric antigen receptor T cells (CART) have been proved a powerful weapon to treat cancers. Extensive efforts are focused on improving the efficacy of CART in solid tumors. Phase I trial of IL18 secreting CART targeting CD19 showed remarkable clinical effects in patients with relapse from previous CART therapy, supporting the notion that IL18 may serve as an important tool to enhance CART potency in solid tumor. Meanwhile, the complicated process of autologous CART production is labor intensive and costly. Various approaches are being explored to optimize the clinical translation of universal CAR armored immune cell therapies, such as universal CART and iPS derived CAR-NK.  Alternatively, the process of CART production can be significantly accelerated by utilizing automatic equipment. The presentation will discuss how Truking Gene CART series achieve a streamline process of CART production:

- Automatic processing of patient blood samples to obtain PBMC

- Automatic isolation of T cells by magnetic beads simultaneously activating purified T cells

- One step continuous expansion of CART cells

- Automatic formulation of CART final product

The Truking gene CART series operate with sterile, closed and single use consumables to avoid microbe and cross contamination. The automated streamline process not only minimizes the risk of human errors during production, but also helps pharmaceutical companies to expand the manufacturing capacity for treating more patients.  

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崔昕晖

索尼（中国）生命科学

高级应用专家

演讲主题: A Full Closed And GMP-Level Cell Isolation System to Empower Next Generation Cell & Gene Therapy - Sony CGX10 Cell Isolation System

Abstact: The Sony CGX10 cell isolation system is innovative flow cytometry sorting platform specifically designed for cell and gene therapy manufacturing processes. The patented hydrodynamic design realizes gentle processing of cells in microfluidics chip, with five following advantages: "Closed system, GMP-ready, simple operation, high cell viability, and reliable data". Sony CGX10 can isolate target cells with multi-parameter labelling, regulate the composition of subsets in cell therapy products, and shorten manufacturing time to obtain various specific CAR-X products for patients, helping biomedical companies empower the next generation of CGT therapies development and help break through existing bottlenecks to achieve the best results.

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陈丽娟

跃赛生物

首席运营官

演讲主题: Clinical Considerations for Stem Cell therapy in CNS indications
- Commercial prospects for stem cell therapy in CNS indication

- Challenges in clinical development of stem cell therapy

- Addressing challenges and acceleration strategies

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丁平

康德赛医疗 

创始人兼首席执行官

演讲主题: mRNA编辑的细胞药物开发