FUJIFILM Corp.

行业乐观与悲观情绪夹杂，但都要做好准备，迎接战斗 撰文| 石若萧 当地时间9月9日，美国众议院对《生物安全法案》提案进行了投票表决。 结果并不意外，最终法案以306-81的结果，推进至下一个阶段。 对行业，这毫无疑问是一个坏消息，不过从好的方面看，坏消息里也蕴藏着向好的迹象。 首先，比起今年年初，法案带来的压力已经小了不少。今年5月，美国众议院的监督与问责委员会举行的对该法案的听证会上，投票的结果是40-1，赞同比例高达97.6%。不到4个月时间，这一比例已经下降到79.1%。 然后，任何法案都须参众两院投票通过，并由时任美国总统签字，才能成为法律。但近期美国政坛的重心都在大选上，法案想在今年年底前通过，时间非常紧迫。再说美国国会内部依然充斥着不少反对声音，届时情况会如何发展仍未可知。 最后，法案的波及面没有想象中那么大。根据目前版本，如果一项业务没有涉及美国政府的资助，就不会收到法案的限制影响。此外法案还规定，即便业务合同受到了美国政府的资助，也允许被指定的公司能继续能继续履行合同，期限直至2031年年底。这给予了行业一段相当长的缓冲期。 股价也一定程度上反映出了人们乐观悲观夹杂的情绪。今日一早，港股药明生物开盘迅速下跌，跌幅一度达12%，不过随后逐渐收窄。收盘后，药明生物跌3.88%，药明康德跌超10%，药明合联跌9.44%，华大基因跌2.03%，华大智造跌1.50%。整体来看，跌幅有高有低，但仍在能接受的范围内。 该法案落地，未来究竟会导向何种结果？会以何种形式和力度重新塑造产业生态？关于这一点，行业里看法各异，有悲观亦有乐观。但无论如何，有一点不会改变——中国CXO唯有靠自己，做好准备，迎接战斗，才能在行业风暴中站住脚跟，迎来明天。 一．利空已经出清 第二次世界大战期间，得知日本偷袭珍珠港的消息后，邱吉尔反倒睡了一个安稳觉。在他看来，不确定性消除以后，反而能更好地看清事情的走向。 《生物安全法案》在众议院获通过也一样，说明药明系的利空几乎已经出尽。将来，看清事件的本质，做好最坏的打算，保持积极的预期，才能更好地布局未来。 二，情况会越来越坏，做好准备 这是一个很坏的开头，相关各方必须做好接受进一步打击的准备。 美国人一向擅长利用法律，再加上其于全球“长臂管辖”的权力，对竞争对手进行针对性打击，当年以商业贿赂之名“肢解”阿尔斯通这个世界500强企业就是采用的这一流程。 药明系现下面对的挑战，与当年的阿尔斯通很相似。不过不同之处在于，商业贿赂是一项已存在的罪名，需要的只是找到把柄。但对于药明系公司，已有罪名不适用，需要先创造一个新法律才能达到“师出有名”的效果。 从这一角度出发来考量，《生物安全法案》倘若落地，最大的麻烦还不在法案内容本身，而在于让美国掌握了这个“名”之后，药明系和华大系等中国公司或将迎来更猛烈的打击，并且后续如果再扩大打击面，将新的中国公司纳入法案影响范围也很容易。 三．印度或成最大赢家 一些印度CDMO公司近期表示，其在第二季度收到了更多询价，但对新增业务的实际影响究竟几何，可能还需要4~6个月才能显现。 有专家认为，印度目前有100多家CDMO公司，其中的几家头部企业包括Syngene International、Aurigene和Jubilant等，将会成为《生物安全法案》实施后的最大获益者。另据媒体报道，多家印度CDMO都表示将抓住机会，调整战略，以期更好地应对处于增长中的市场。 不过一个疑问是，印度CDMO或许能得到机会，但能否承接得住整个欧美创新药玩家的需求？很大的可能是，印度公司仍需将一部分业务通过与中国企业合作的形式来实现。 毕竟，印度的API出口额约为40亿美元，而中国的API出口额约为400亿美元。两者存在10倍的差距。并且由于医药行业受到监管影响，供应商生态系统无法在一夜之间出现改变，短期内全球的创新药玩家都不可能完全摆脱对中国的依赖。至于美国的真正目的，恐怕也不是将中国完全踢下牌桌，而是建立一个可替代的供应商生态系统，即使成本更高也在所不惜。 四．日韩也要分一杯羹 这几年，韩国CXO不断扩大的产能来“抢位”，以三星生物为例，其这几年产能在不断扩大。第1工厂于2012年完工，2013年开始运营，产能为3万升；此后加码建设2号、3号厂，1、2、3号工厂合计产能36.4万升；2020年，又投入100亿元开工建设年产能为25.6万升的4号工厂；2023年再次宣布将投资 1.9 万亿韩元（约100亿人民币）在韩国建立一家新工厂，也即5号工厂。一旦5号工厂全面建成，三星生物将以逾78万升的总产能保持其全球生物制造产能的领先地位。 随着中美角力加剧，欧美大药厂更是加大了与三星生物的合作。最新消息显示，三星生物已经与全球前20大药企中的16家达成了合作。 除了三星生物外，韩国企业SK Pharmteco和Lotte Biologics也在不断推进扩大产能的动作。韩国之外，日本企业如富士胶片也在推进收购扩产，向欧美进军，业务不断增长。 不可否认，日韩企业的“趁机抢位”策略是有效果的，跨国药企也在考虑分散供应链及自建产能，整个CXO市场的订单加速分流已成定局。面对不友善的国际地缘竞争环境，中国CXO更应该“打造自己的护城河”，积极直面竞争，进一步提升实力，才能赢得话语权。 五．中美双输 中美合作，好处远大于坏处。这一点，正面例子是特斯拉，如果没有上海工厂的放量生产，特斯拉的技术迭代和市场抢占节奏不会这么快，也无法很好地进行技术迭代。 一个反面例子就是苹果。没有可靠、尖端、敏捷的中国产业链作为基础，新设计根本不敢冒险。任何稍微激进的设计都可能因为孱弱的供应链能力要么导致大延期，要么强行赶工期造成巨大的品牌隐患。导致新款iPhone的创新迭代极其缓慢，被安卓阵营赶超的趋势越来越明显。 医药行业也一样，与全球CXO公司相比，中国CXO最为成熟，研发能力、性价比、效率都是全球领先，并且在与客户长期合作中逐渐已经形成了较为深厚的默契，帮助它们解决了初创企业资金匮乏的最核心危机，这也是为何中国CXO可以在世界立足的根本。如果因为政治原因盲目对中国进行打击，长期来看，会对美国的Biotech研发生态造成难以估计的破坏。 六．开放者必胜，封闭者必败 就在法案通过前两天，中国商务部等3部门发布《关于在医疗领域开展扩大开放试点工作的通知》。根据通知，拟允许在北京、天津、上海、南京、苏州、福州、广州、深圳和海南全岛设立外商独资医院。文件以更加开放的姿态，积极推动生物技术的研发与应用，为外商投资企业打开了新的机遇之门。 相比美国的狭隘，中国展现出了更加开放和包容的姿态。这种开放的态度不仅为中国生物技术产业的蓬勃发展注入了新的活力，也为全球生物技术的合作与交流提供了更加广阔的空间和平台。 在全球生物技术领域，合作与交流已经成为不可阻挡的潮流。我们呼吁美国摒弃封闭和排斥的做法，以更加开放和包容的心态积极参与到全球生物技术的合作与交流中来。同时，中国也应继续坚持开放的态度，积极推动生物技术的研发与应用，为全球生物技术的进步与发展贡献更多智慧和力量。 一审| 黄佳 二审| 李芳晨 三审| 李静芝 精彩推荐 CM10 | 集采 | 国谈 | 医保动态 | 药审 | 人才 | 薪资 | 榜单 | CAR-T | PD-1 | mRNA | 单抗 | 商业化 | 国际化 ｜ 猎药人系列专题 | 出海 启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 医药界·E药经理人 | 中国医药手册 创新100强榜单 | 恒瑞 | 中国生物制药 | 百济 | 石药 | 信达 | 君实 | 复宏汉霖 ｜翰森 | 康方生物 | 上海医药 | 和黄医药 | 东阳光药 |  荣昌 | 亚盛医药 | 齐鲁制药 | 康宁杰瑞 | 贝达药业 | 微芯生物 | 复星医药 ｜再鼎医药｜亚虹医药 跨国药企50强榜单 | 辉瑞 | 艾伯维 | 诺华 | 强生 | 罗氏 | BMS | 默克 | 赛诺菲 | AZ | GSK | 武田 | 吉利德科学 | 礼来 | 安进 | 诺和诺德 | 拜耳 | 莫德纳 | BI | 晖致 | 再生元

CAMBRIDGE, MA USA I September 3, 2024 I BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. “We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001,” said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. “We believe that OpCT-001 has potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our Phase 1/2a clinical study.” OpCT-001 is the first investigational iPSC-derived cell therapy to be clinically evaluated for the treatment of primary photoreceptor diseases. Initiation activities to support a Phase 1/2a study are underway. The Phase 1/2a study is designed as a first-in-human study to evaluate the safety and tolerability of subretinal administration of OpCT-001 in people with primary photoreceptor diseases as well as evaluate the effect of OpCT-001 on retinal structure, visual function and functional vision. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the U.S. Primary photoreceptor diseases are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. Primary photoreceptor diseases affect an estimated 110,000¹ people in the U.S. and only limited treatment options exist for this population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated cells in the retina with functional cells. OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of the strategic R&D and clinical manufacturing collaboration between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001 at their cGMP² facility in Madison, Wisconsin. OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. ¹https://www.ncbi.nlm.nih.gov/books/NBK519518/ ; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/ ; https://www.nidcd.nih.gov/health/usher-syndrome#b ²Current good manufacturing practices are regulations enforced by the FDA. About BlueRock Therapeutics LP BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the U.S. FDA (Food and Drug Administration); has completed Phase 1 clinical trial and is progressing to the next stage of clinical development. OpCT-001 has been cleared by the FDA to begin Phase 1 clinical testing. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com . Find more information at https://pharma.bayer.com Follow us on Facebook: http://www.facebook.com/bayer SOURCE: Bluerock Therapeutics

DUBLIN--( BUSINESS WIRE )--The "Induced Pluripotent Stem Cell (iPSC) Industry Report - Market Size, Trends, and Forecasts, 2024" report has been added to ResearchAndMarkets.com's offering. This global strategic report reveals all major market competitors worldwide, including their core technologies, strategic partnerships, and products under development. It covers the current status of iPSC research, biomedical applications, manufacturing technologies, patents, and funding events, as well as all known trials for the development of iPSC-derived cell therapeutics worldwide. Importantly, it profiles leading market competitors worldwide and presents a comprehensive market size breakdown for iPSCs by Application, Technology, Cell Type, and Geography (North America, Europe, Asia/Pacific, and Rest of World). It also presents total market size figures with projected growth rates through 2030. Since the discovery of induced pluripotent stem cell (iPSC) technology in 2006, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated. iPSCs can be used to explore the causes of disease onset and progression, create and test new drugs and therapies, and treat previously incurable diseases. Today, methods of commercializing induced pluripotent stem cells (iPSCs) include: Cellular Therapy: iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease. Disease Modelling: By generating iPSCs from patients with disorders of interest and differentiating them into disease-specific cells, iPSCs can effectively create disease models "in a dish". Drug Development and Discovery: iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery. Personalized Medicine: The use of techniques such as CRISPR enable precise, directed creation of knock-outs and knock-ins (including single base changes) in many cell types. Pairing iPSCs with genome editing technologies is adding a new dimension to personalized medicine. Toxicology Testing: iPSCs can be used for toxicology screening, which is the use of iPSCs or their derivatives (tissue-specific cells) to assess the safety of compounds or drugs within living cells. Tissue Engineering: iPSCs can be seeded onto scaffolds made from biocompatible materials. These scaffolds mimic the structure and properties of the target tissue and can provide a supportive environment for cell growth and differentiation. Organoid Production: iPS cells can be coaxed to self-organize into 3D structures called organoids, which mimic the structure and function of organs. Organoids can be used for studying organ development, modeling diseases, and testing drugs. Gene Editing: iPS cells can be genetically modified using techniques like CRISPR-Cas9 to correct disease-causing mutations or introduce specific genetic changes. These edited iPS cells can then be differentiated into the desired cell type for transplantation or disease modeling. Research Tools: iPSCs and iPSC-derived cell types are being widely used within a diverse range of basic and applied research applications. Stem Cell Banking: iPSC repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC-derived cell types produced from both healthy and diseased donors. Cultured Meat Production: iPSCs are being utilized in clean meat production by serving as the cellular foundation for the creation of lab-grown meat. 3D Bioprinting: iPSCs can be directed to differentiate into cell types of interest, such as skin, heart, or liver cells, which are then incorporated into bioinks. Wildlife Conservation and De-extinction Projects: iPSCs are being used in wildlife conservation and de-extinction projects. For example, Colossal Biosciences is using iPSC technology in an effort to achieve woolly mammoth de-extinction. Key Topics Covered: 1. REPORT OVERVIEW 2. INTRODUCTION 3. CURRENT STATUS OF IPSC INDUSTRY 3.1 Progress made in Autologous Cell Therapy using iPSCs 3.2 Allogeneic iPSC-based Cell Therapies 3.3 Share of iPSC-based Research within the Overall Stem Cell Industry 3.4 Major Focus Areas of iPSC Companies 3.5 Commercially Available iPSC-derived Cell Types 3.6 Relative use of iPSC-derived Cell Types in Toxicology Testing Assays 3.7 iPSC-derived Cell Types used in Clinical Trials 3.8 Currently Available iPSC Technologies Nucleofector Technology Opti-ox Technology MOGRIFY Technology Transcription Factor-based iPSC Differentiation Technology Flowfect Technology Technology for Mass Production of Platelets SynFire Technology 4. HISTORY OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 5. RESEARCH PUBLICATIONS ON IPSCS 6. IPSC: PATENT LANDSCAPE ANALYSIS 6.1 iPSC Patent Applications by Jurisdiction 6.2 iPSC Patent Applicants 6.3 Inventors of iPSC Patents 6.4 iPSC Patent Owners 6.5 Legal Status of iPSC Patents 7. IPSC: CLINICAL TRIAL LANDSCAPE 7.1 Number of iPSC Clinical Trials 7.2 Recruitment Status of iPSC Clinical Trials 7.3 iPSC Clinical Trials Study Designs 7.4 Therapeutic & Non-Therapeutic iPSC Clinical Trials 7.5 iPSC-based Trials by Phase of Study 7.6 iPSC Clinical Trials by Funder Type 7.7 Geographic Distribution of iPSC-based Clinical Trials 7.8 Promising iPSC Product Candidates CYP-001, CYP-004 & CYP-006 from Cynata Therapeutics BioVAT-HF from Repairon GmbH HS-001 from Heartseed CNTY-101 from Century Therapeutics FT-576 & FT-819 from Fate Therapeutics RPE from National Eye Institute QN-019a from Qihan Biotech iPSC-CL from Heartworks, Inc. 7.9 Companies having Preclinical iPSC Assets Aspen Neuroscience Ryne Biotech Bluerock Therapeutics Vita Therapeutics Hopstem Biotechnology Res Nova Bio, Inc. Cytovia Therapeutics Hebecell Corporation Sana Biotechnology SCG Cell Therapy Pte Cytomed Shoreline Biosciences Neukio Biotherapeutics Exacis Biotherapeutics CellOrigin Biotech 8. M&A, COLLABORATIONS & FUNDING ACTIVITIES IN IPSC SECTOR 8.1 Mergers and Acquisitions (M&A) Sector Century Therapeutics & Clade Therapeutics Evotech & Rigenerand Fujifilm Corporation & Atara Biotherapeutics Catalent & RheinCell Therapeutics Axol Biosciences & Censo Biotechnologies Bayer AG & Bluerock Therapeutics Pluriomix & Axiogenesis 8.2 Partnership/Collaboration & Licensing Deals in iPSC Sector Shinobi Therapeutics & Panasonic SCG Cell Therapy and A*STAR Charles River Laboratories & Pluristyx, Inc. Pluristyx, Inc. & National Resilience, Inc. University of Texas & GeneCure Heartseed, Inc. & Undisclosed Biotech Bluerock Therapeutics & Bit.bio Applied Stem Cell, Inc. & CIRM Resolution Therapeutics & OmniaBio, Inc. REPROCELL, Inc. & CIRM REPROCELL, Inc. & BioBridge Global Elevate Bio & CIRM Evotec & Sernova Evotec & Almiral Quell Therapeutics & Cellistic MDimmune & YiPSCELL Edigene & Neukio Biotherapeutics Matricelf & Ramot Evotec & Boehringer Ingelheim Pluristyx, Pancella & Implant Therapeutics Century Therapeutics & Bristol Myers Squibb Fujifilm Cellular Dynamics & Pheno Vista Biosciences Metrion Biosciences & Bioqube Ventures Cytovia Therapeutics & Cellectis Exacis Biotherapeutics & CCRM Cynata Therapeutics & Fujifilm Corporation Bone Therapeutics & Implant Therapeutics REPROCELL & TEXCELL Jacobio & Herbecell NeuCyte & KIF1A.ORG Kite & Shoreline Biosciences Neuropath Therapeutics & Hopstem Biotechnology Allele Biotech & Cellatoz Bluerock Therapeutics, Fujifilm Cellular Dynamics & Opsis Therapeutics Newcells & Takeda Biocentriq & Kytopen Fujifilm Cellular Dynamics & Sana Biotechnology Evotec & Medical Center Hamburg-Eppdorf (UKE) NeuCyte & Seaver Autism Center for Research and Treatment Cytovia Therapeutics & National Cancer Institute Mogrify & MRC Laboratory of Molecular Biology 8.3 Venture Capital Funding in iPSC Sector Asgard Therapeutics Kenai Therapeutics Pluristyx Fujifilm Cellular Dynamics Mogrify Ltd. Heartseed, Inc. Elevate Bio Aspen Neurosciences Axol Biosciences Thyas, Co. Ltd Synthego Cellino Biotech, Inc Curi Bio Ncardia Evotec SE bit.bio Clade Therapeutics Shoreline Biosciences Kytopen Cytovia Therapeutics & CytoLynx TreeFrog Therapeutics HebeCell Corporation Neukio Biotherapeutics Stemson Therapeutics Vita Therapeutics Century Therapeutics Heartseed Mogrify Metrion Biosciences Elevate Bio Vita Therapeutics 9. GENERATION OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 9.1 OSKM Cocktail 9.2 Pluripotency-Associated Transcription Factors and their Functions 9.3 Delivery of Reprogramming Factors 9.3.1 Integrating Systems 9.3.2 Non-Integrative Delivery Systems 9.3.3 Comparison of Delivery Methods 9.4 Genome Editing Technologies in iPSC Generation 9.5 Available iPSC Lines and their applications 10. HUMAN IPSC BANKING 10.1 Major Biobanks Storing iPSCs & iPSC Lines 10.1.1 RIKEN 10.1.2 WiCell 10.1.3 Fujifilm Cellular Dynamics, Inc. 10.1.4 Sampled 10.1.5 Coriell Institute for Medical Research 10.1.6 European Bank for Induced Pluripotent Stem Cells (EBiSC) 10.2 Cell Sources for iPSC Banks 10.3 Reprogramming Methods in iPSC Banks 10.4 Ownership and Investments made in iPSC Banks 11. BIOMEDICAL APPLICATIONS OF IPSCs 11.1 iPSCs in Basic Research 11.2 Applications of iPSCs in Drug Discovery 11.3 Applications of iPSCs in Toxicology Studies 11.4 Applications of iPSCs in Disease Modeling 11.5 Applications of iPSCs in Cell-Based Therapies 11.6 Other Novel Applications of iPSCs 12. MARKET ANALYSIS 12.1 Global Market for iPSCs by Geography 12.2 Global Market for iPSCs by Technology 12.3 Global Market for iPSCs by Biomedical Application 12.4 Global Market for iPSCs by Derived Cell Type 12.5 Market Drivers 12.5.1 Current Drivers Impacting the iPSC Market Place 12.6 Market Restraints 13. COMPANY PROFILES For more information about this report visit https://www.researchandmarkets.com/r/pf57qx About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.