【IGC2024 9月6日精彩演讲摘要】 CGT国际监管与技术前瞻&CGT商业化与BD投融资专场
专场K CGT国际监管与技术前瞻&CGT商业化与BD投融资专场
9月6日，IGC 第八届免疫基因及细胞治疗大会将于北京国际会议中心成功拉开帷幕！
作为国内最大规模的CGT活动之一，今年IGC大会特设12+场深度细分论坛，28大精彩专题，集结170+IO/GCT领域科研学者/KOL、企业领路人等专家讲者，两天活动共计吸引3000+人次的专业精英听众出席。
CGT国际监管与技术前瞻专场现场纪要
话题：基于iPS细胞疗法：从实验室到临床iPS cell-based therapies: from bench to bedside
嘉宾：Kenji Osafune MD, PhD, Deputy Director/Professor, Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan
This presentation delves into the advantages of cell therapy and induced pluripotent stem cells (iPSCs). Cell therapy has achieved remarkable results in clinical trials for over 10 diseases, including Parkinson's and COVID-19, and has been approved for the treatment of inherited neurological and endocrine diseases. Our team has made significant research findings in this field and uncovered its mechanism of action. Furthermore, we have developed an innovative differentiation method, successfully inducing iPSCs into kidney, pancreas, and liver lineage cells and organs. These cells hold potential applications in treating diseases such as chronic kidney disease (CKD), type 1 diabetes, and liver cirrhosis. Meanwhile, we have also successfully generated an ADPKD disease model based on iPSCs. The number of people receiving IPS treatment in Japan is continuously growing, indicating a promising future.
（嘉宾未确认）
话题：CAR -T 细胞治疗制造的产业化
Industrialization of CAR-T Cell Therapeutic Manufacturing
嘉宾：Prof. Dr. Dr. Rolf G. Werner, Honorary Senator and Professor Industrial Biotechnology, University Tübingen, Germany
The presentation delved into the recent advancements in the field of cell and gene therapy. The speaker highlighted the continuous development of cell gene research, drug production processes, and industrial manufacturing technologies, which are collectively driving breakthrough progress in this domain. Notably, the application of Mustang technology in lentivirus purification was mentioned, with its efficient and pure virus vector production capabilities garnering attention. Additionally, the leading position of affinity chromatography technology was acknowledged, providing valuable support for optimizing drug production processes. Furthermore, the emergence of the third-generation Claudin 18.2 CAR-T cell therapy indicates that the field is poised to welcome more innovative treatment approaches. These key developments are anticipated to be significant driving forces for the future growth of the cell and gene therapy sector.
话题：新型CAR细胞疗法进展Novel CAR therapy
嘉宾：Xingxing Zang, Louis Goldstein Swan Professor & Director Albert Einstein College of Medicine, New York（online）
This presentation delved into the cutting-edge advancements of novel CAR therapy, particularly the functional discoveries related to the HHLA2 gene within the B7 family. As a pivotal member of the B7 family, the unique characteristics of HHLA2 were thoroughly examined. The presentation unveiled a novel immune regulatory pathway involving HHLA2, alongside IMIGD2 (or TMIGD2) and KIR3D (or KIR3DL3), which has opened up a new perspective for strategizing CAR therapy, hinting at potential optimizations in treatment approaches.
The presentation also highlighted the remarkable performance of CAR therapy in the field of precision medicine. Its high specificity ensures the precision of treatments, while its potent efficacy enhances therapeutic outcomes. Particularly noteworthy is the broad application prospect of CAR therapy in various types of tumor treatments, showcasing its immense potential. By contrasting the traditional B7-1/B7-2/CD28/CTLA-4 immune pathway with the emerging HHLA2/TMIGD2/KIR3DL3 pathway, the presentation emphasized the significance of deepening our understanding of the B7 family and innovating CAR designs to propel continuous advancements in the field of immunotherapy.
话题：iPSC衍生的视网膜治疗iPSC-derived retinal therapy
嘉宾：Xianmin Zeng, Founder & CEO of RxCell Inc, USA; Visiting Professor, National University of Singapore
The presentation zeroed in on the promising future of iPSC-derived cell therapy products in retinal degenerative disorders, notably AMD. With the AMD market estimated at USD 74.7 billion in 2021 and forecasted to expand at a CAGR of 7.9% over the next decade, the discussion highlighted the company's therapy as a groundbreaking innovation over conventional approaches. Its advantages lie in precise targeting and long-term therapeutic outcomes. The company has successfully concluded IND-enabling activities and proof-of-concept studies, while also addressing immune rejection challenges, exploring iPSC genome editing technologies, and studying disease models. The detailed account of their unique production processes and workflows emphasized the positive influence on next-generation treatments, portending a novel breakthrough for cell therapy in this field.（嘉宾未确认）
话题：先进疗法——现行EMA法规要点及未来发展趋势Advanced therapies – current regulatory issues and future developments
嘉宾：Kieran Breen, Vice Chair, Committee for Advanced Therapies of EMA; Head of Research and Innovation, St Andrews Healthcare, United Kingdom（online）
The presentation focused on the EU Regulation for Advanced Therapy Medicinal Products (ATMPs) under (EC) No 1394/2007, introducing the role of CAT in fostering innovation. CAT annually provides scientific advice to over 50 companies, supporting their early-stage development. Companies engage with EMA through multiple programs to optimize product strategies. Additionally, EMA funds a registry-based study on Spinal Muscular Atrophy (SMA), observing post-treatment diagnostic shifts, conducting data statistics and analysis, and advancing disease treatment. The emphasis was on the collaboration between regulation and research to drive innovation and progress in ATMPs.
（嘉宾未确认）
话题：借助监管政策和技术指导原则，为全球基因治疗药物开发降本提速Leveraging regulatory policies and technical guidance principles to reduce costs and accelerate the global development of new gene therapy drugs
嘉宾：Alvin Luk, HuidaGene Therapeutics, Co-founder, CEO, and Head of Medical
The presentation focused on the revolutionary potential of gene therapy and gene editing. It traced the journey from scientific breakthroughs to commercialization over five decades, highlighting challenges and advancements. Current landscape were discussed, featuring gene/cell/RNA-based drugs in development. A notable trend of increasing clinical trials was emphasized, underlining the need for early, frequent, and well-planned consultations with regulatory agencies to facilitate progress. Different format to accelerate drug approval and different guidelines on study designs to accelerate the development were also discussed.
话题：治疗实体瘤的同种异体NK细胞生产的机遇与挑战Opportunities and Challenges in Manufacturing of Allogeneic Natural Killer (NK) Cells for Immunotherapy targeting Solid Tumors
嘉宾：A/Prof Andy Tan, Principal Scientist and Group Leader, Bioprocessing Technology Institute (BTI) , Agency for Science, Technology and Research, Singapore (A*STAR)
The NK cell therapy market is projected to witness substantial growth at a CAGR of 34% from 2023 to 2030, with a projected market size reaching USD 3 billion by 2030. A recent presentation delved into the backdrop of NK cell adoptive immunotherapies, emphasizing their application in ongoing clinical trials, particularly highlighting the advancements in CAR-NK cell therapy research. The discourse also zeroed in on the technical and regulatory hurdles encountered during the manufacturing process of NK therapies, underscoring the importance of overcoming these challenges to propel the advancement of this field. In summary, NK cell therapy presents a promising outlook, yet necessitates the conquest of numerous obstacles to fully realize its immense potential.
话题：兼具降低CRS风险及适应多平台的CD19 CAR-T细胞生产工艺Cross-Platform Adaptability of a synchronized CD19 CAR T-Cell Manufacturing Process with Reduced CRS Risk
嘉宾：M.H. U. Turabe Fazil, Head of Research & Development, Biocell Innovations Pte Ltd
The speech centered on the pivotal challenges in CAR T-cell therapy manufacturing and efficacy enhancement. The team is continuously striving to optimize BiOCELL's CAR T-cell therapy, particularly targeting B-cell malignancies, with CD19 identified as a potent target. Discussions encompassed the in-house development of CAR T lentiviral (LV) vectors for autologous T-cell immunotherapies, highlighting the successful creation of BC-hCD19 CAR LV vectors. The development of a BioCell Process that depletes IL-6 expression in CAR T cells was discussed, with potential adaptability to multiple scaled-up manufacturing platforms. Experimental outcomes demonstrated that BC-hCD19 CAR T-cells exhibit superior potency compared to control CAR T-cells. Additionally, critical hurdles in CAR T-cell therapy and manufacturing were thoroughly debated.
CGT商业化与BD投融资专场现场纪要
圆桌讨论：CGT商业化与商业保险支付现状与趋势
主持人：刘博锋，陆道培医院国际中心，主任
谢祖全，信念医药，商务管理副总裁
蒋璐，暖哇科技，带病体保险部总经理
张卓敏，北京圆心惠保科技有限公司，医药创新总监
刘博锋：对于整个CGT行业定价很高，怎么考虑对于患者而言的接受度？
谢祖全：血友病基因治疗产品在美国350万美金左右，最长疗效有9年的数据，如果合计每年凝血因子的费用支出，基因治疗产品大大节约了患者的总支出并展现了巨大的价值利益。对于基因治疗产品除了疗效和价值之外，企业定价也会考虑患者数量，企业的研发和生产成本，由于综合成本比较高，未来需要有商业保险、国家保险多方参与进来。
刘博锋：未来会考虑增加医保吗？
谢祖全：在积极和国家部门沟通，如果30万的水平估计没有哪个企业的成本可以覆盖到。基因治疗的疗效是很多年的，不能用一年来衡量。
刘博锋：怎么平衡医院的诉求？怎么看待市场定价？
张卓敏：关于定价的问题可以从两方面来提供一些想法，第一、如果通过DTP药房来销售，药企与药房直接签订供销合同，去除了很多不必要的中间环节，且药企相对占据主导地位，可以降低铺货成本，降低部分定价压力。但另一方面，由于本身工艺制备、个性化定制等原因，此类产品的成本无法降到类似其他常规药品的水平，所以价格不会太低，同时也需要综合考虑后续上市后营销策略和同类竞品的定价来综合调整。
刘博锋：定价不符合大部分患者的支付能力，有哪些新的模式可以帮患者提高必需药品的支付比例？
蒋璐：业务针对罹患疾病的人群，发现过往更多是围绕健康体做服务，而患病体市场空白，商保是很好的手段。一是传承，“百万医疗”当年的效果值得效仿，二挑战，商保更多解决健康状态下的，保险公司后续会遇到很多挑战，过往经营在健康人群发病基础上，而对带病人群是空缺的。目前在做的带病保险，包含特药，未来可能会是新的思路。“质慧保”，质子疗法。新的形势逐段涌现。目前拓展纯商保形式就医，但是初期阶段，也一直在不断研究新的方法，争取新的突破。
刘博锋：如何对待解决患者的高期待值问题？
谢祖全：基因治疗国外目前有新的支付方式——按疗效付费。目前有好几个基因治疗的产品采取这种方案支付。
张卓敏：可以针对部分疾病，可以联合多家药企与保险公司共同研发一款针对疗效和病程进展的保险产品来平衡患者对药品疗效的担忧，另一方面也可以直接和保险公司合作共同开发疗效保险，去分担患者的支出，当然无论哪种方式，为了做出更好的产品相应的数据共享必不可少。当然圆心由于有自营的药房，可以为药企的患者提供全方位患者管理服务，特别针对细胞或基因疗法这类需要特殊流程的治疗手段，患者的全病程陪护和管理服务将会极大提高患者的满意度，也提高患者获益。
蒋璐：一种是疗效险，三方参与，药企、保险公司共同对标，可以提高患者的用药信心，也能帮助药企更好的推广产品。另外一种是意外险，准确来说是手术意外险。联合保险公司针对手术过程中的意外进行保障，通过这种保险帮助患者兜底。
刘博锋：面对新药层出不穷的趋势，以及医保控费，商保压力更大，在这种情况下，怎么看待未来的结合？
谢祖全：基因治疗产品目前上市不多，刚上市靠商保更多，但未来最终还是希望国家医保一起来承担，从长远来看，希望一起努力能够有机会按疗效付费在医保端执行。另外现有普遍较贵基因治疗产品较贵的原因是因为都针对罕见病，未来常见病的基因治疗产品，成本不断下降，相信是能走到医保范围的。
张卓敏：药企的药物研发之初就要考虑后商业化后的市场和营销策略，包括是否直接冲击医保还是通过商保的方式来先行，这些都是需要综合考虑的。另一方面对于IGC的产品，个人认为短期内利用商保来提供更好的支付支持，是更现实且有效的手段。通过参与不同商业保险来获得叠加赔付，再加上药企一定程度让利活动，是可以让患者能够销售先进疗法的获益，分散患者的支付压力，让高价值药品快速在市场上落地并抢占市场。
蒋璐：阶段形式变迁，过往的形势在现阶段不能高效，认为未来能够有新的阶段形式融合，新的发展形势是必然的。医疗改革压力之下，药企医院保险的融合是趋势，精准模型定价，性价比更高。
圆桌讨论：CGT投融资与BD合作环境与趋势
主持人：余永平，鋆昊资本医疗基金合伙人，清华校友总会生命科学与医疗健康专委会秘书长
邓灵泉，幂方健康基金合伙人
薛园，北京生命园创投基金合伙人
孟凡勇，富汇创投，执行董事
余永平：在投资细胞领域不确定性情况怎么看？
邓灵泉：现在投融资市场很冷，大家投资创业都需要非常谨慎，对自己负责。没做好充足的准备、未达到一定水平不要轻易创业。如果评估觉得已达到，则要花心思找好的合作伙伴及投资机构，把大方向整理清楚。投资机构也是一样要谨慎，得熟悉公司行业。破局办法是追求国际竞争力，与海外同类型公司直面竞争时也能脱颖而出。如果具备上面说的这些条件，任何时候都是可以做事情的。
薛园：追求国际化创新，从投资角度讲，尽量寻找一些价值和确定性。我们基金长期认可CGT的价值，也是很有前景。企业根据自身的情况去做选择，追求自己真正想做的事情，建立差异化优势，不仅在国内市场站稳脚跟，还要能在海外同类公司中脱颖而出，实现全球化发展。
孟凡勇：目前资本市场行情确实不好，但是我们基金仍然坚持在在看、投一些有差异化特点的项目。我们在搜索项目中确实发现，教授创业的项目技术确实很新，但是对于研发规划、产业化和市场的理解远远达不到工业界的标准，我们对于这类项目会劝退。对于部分罕见病基因治疗项目，我们也相对谨慎，整体认为国内罕见病发病区域还是以经济欠发达区域人群居多，这类人群对于治疗付费能力上会更弱，在国内线性医疗支付体系的情况下，我们比较担心商业化，担心基金的投资回报问题。
余永平：你的能力在哪里，你的资源在哪里，就选择什么方向，根据自己的研发能力、商业化能力找到适合自己的方式。创业者和创业投资者是共同的合伙人。
余永平：BD有方法，临床有方法，大胆预测一下我们未来怎么发展？
邓灵泉：最近在细胞和基因治疗领域我新投资创立了两家公司，一个是基因编辑领域的项目，一个细胞治疗领域的项目。为什么要投呢？因为这两家公司要做的事情和产品十分清晰的被临床和市场所需要，另外你得能找到做那个事情全市场最优秀的那几个人合作，能做到世界前三的人。这样再去做事成功率就高。
薛园：希望从客观本质出发，能满足真实的未满足临床需求，有一个好的验证呈现，看好干细胞与再生医学，通用型疗法未来的发展潜力很大。
孟凡勇：我们会跟我们已投的项目团队去提醒，要绷紧BD这根弦。在对外BD过程中，要说清楚产品的差异化特点，是否和潜在的买家/合作方匹配。我们希望在短暂的时间内，资本市场行情能够转暖，国内的医疗的支付体系有改革，创业团队、投资方、患者各方都能够收益。
观众提问：对于创业者的一些期待和要求？
薛园：我们就是一个长期耐心资本，我们团队一直跟着科学家和企业家们一起成长，我们基金有个slogan：发现改变世界的科学家和企业家，共同创造真实社会价值。所以资本的耐心呢，是跟着这个“真实社会价值“走的。希望对需要有耐心的事有耐心，并且团队需要有高度的执行力。
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