Key Takeaways Moderna gained full FDA approval for Spikevax in high-risk children aged six months to 11 years. The vaccine was previously available for this group only under Emergency Use Authorization. Updated Spikevax will be available for eligible patients in the 2025-2026 U.S. vaccination season.Moderna (MRNA) announced that the FDA has approved its supplemental biologics license application seeking approval for COVID-19 vaccine, Spikevax, for use in children aged six months to 11 years, who are at in ...

Group 1: Moderna's Vaccine Approval - The U.S. FDA has approved Moderna's supplemental Biologics License Application for Spikevax, allowing its COVID-19 vaccine for children aged six months to 11 years at increased risk for COVID-19 disease [1] - Moderna's CEO emphasized the importance of vaccination for protecting children against severe disease and hospitalization, expressing appreciation for the FDA's review and approval [2] - The updated Spikevax vaccine is expected to be available for eligible populations in the U.S. for the 2025-2026 respiratory virus season [2] Group 2: Legal Challenges and Patent Issues - Pfizer and BioNTech have requested a reversal of a court ruling that found their COVID-19 vaccine infringed on a Moderna patent, while a previous ruling declared one of Moderna's patents invalid [3] - The companies argue that Moderna's mRNA technology is a continuation of earlier research, questioning the validity of the patent [4] - Moderna maintains that the court's decision to uphold the patent is correct and should remain [4] Group 3: FDA's Vaccine Approval Decisions - The FDA's top vaccine official overruled agency scientists to limit the approval of two COVID-19 vaccines, despite recommendations for broader use [6] - The official restricted the vaccines to higher-risk individuals, citing declining hospitalization and death rates from COVID-19 as a factor in the decision [7] - Following the news, Moderna's stock price increased by 3.60% to $33.97 [7]

Core Insights - The emerging autologous CAR-T therapy significantly reduces costs compared to traditional CAR-T therapies, which can exceed hundreds of thousands of yuan, but it faces high technical barriers [1][4]. Industry Developments - AbbVie announced a $2.1 billion acquisition of Capstan Therapeutics to expand its product line in autoimmune disease therapies, marking the highest transaction record in the cell therapy sector for 2025 [1]. - CloudTop New Horizon (01952.HK) reported advancements in its autologous CAR-T projects, which utilize a proprietary targeted LNP (tLNP) system for treating tumors and autoimmune diseases, achieving effective tumor cell clearance in humanized mouse models and validating T cell transfection in non-human primate models [1]. Advantages of Autologous CAR-T Therapy - Compared to traditional CAR-T therapies, autologous CAR-T therapy offers multiple advantages, including large-scale production capabilities, no need for lymphodepleting chemotherapy prior to treatment, predictable pharmacokinetics/pharmacodynamics, adjustable dosing, and better overall quality control [4]. - The mRNA technology in autologous CAR-T therapy allows for a shift from "molecular screening" to "information design," enabling the body to produce its own drugs, thus marking a new era in biopharmaceuticals [4]. Cost Reduction Potential - The core advantage of in vivo CAR-T cell manufacturing lies in significantly simplifying the process and reducing costs, potentially lowering the cost of a single treatment by an order of magnitude compared to currently commercialized CAR-T therapies [4]. Challenges in Autologous CAR-T Therapy - The main challenge for autologous CAR-T therapy is the precise, efficient, and safe delivery of CAR-T constructs to target T cells [5]. - Two primary technical routes have emerged: lentiviral vectors and RNA delivery. Lentiviral vectors can achieve permanent CAR expression but may lack targeting precision, while RNA delivery offers higher safety but only transient CAR protein synthesis [5]. - The high technical barriers in autologous CAR-T technology limit participation to a few companies, requiring proprietary cationic lipids and stringent delivery technology to target spleen and peripheral T cells while avoiding safety risks associated with liver expression [5].

Core Viewpoint - Moderna has experienced a significant increase in stock value, gaining over 12% recently, driven by positive developments in its vaccine research [1]. Group 1: Vaccine Development - Moderna published results from a late-stage study of its seasonal flu vaccine, mRNA-1010, which demonstrated a stronger immune response compared to standard flu vaccines [2]. - The company plans to resubmit its application for a combination COVID-19/flu vaccine, following the withdrawal of its previous application in May [4]. - CEO Stéphane Bancel highlighted the trial results as a significant milestone in reducing influenza burden in older adults [5]. Group 2: Market Sentiment - Despite the positive news regarding the flu vaccine, there is skepticism about whether this development alone will make Moderna's stock a compelling buy [5]. - The company has a robust pipeline of development programs, which could enhance the attractiveness of its shares if any show promise [6].

Pipeline and Clinical Trials - Cartesian Therapeutics is pioneering mRNA cell therapies for autoimmunity, with multiple anticipated near-term catalysts[5] - Phase 3 AURORA study of Descartes-08 for Myasthenia Gravis (MG) is expected to commence in 1H25[8] - An open-label Phase 2 trial of Descartes-08 in Systemic Lupus Erythematosus (SLE) is ongoing, with data readout expected in 2H25[8] - A Phase 2 pediatric basket trial, including juvenile SLE, juvenile MG, and other conditions, is expected to initiate in 2H25[8, 12] - Dosing is underway in a first-in-human Phase 1 dose escalation trial for Descartes-15, a next-generation mRNA CAR-T candidate[8] Descartes-08 Efficacy and Safety - In a Phase 2b trial, participants treated with Descartes-08 maintained deep and durable responses over 12 months[16] - At Month 4, participants in the primary efficacy dataset experienced an average MG-ADL reduction of 5.5 points[23] - 33% of participants achieved minimum symptom expression at Month 6[23] - 80% of participants reaching Month 12 maintained a clinically meaningful response[23] - In participants with no prior exposure to biologics, the average MG-ADL reduction was 6.6 points at Month 4[26] - 57% of participants with no prior exposure to biologics achieved minimum symptom expression at Month 6[26] - 100% of participants with no prior exposure to biologics reaching Month 12 maintained a clinically meaningful response[26] - The safety profile of Descartes-08 supports outpatient administration, with no new types of adverse events reported[16, 28] Financial Position - Cartesian Therapeutics has a strong balance sheet with approximately $220.9 million as of September 30, 2024[9] - This is expected to support planned operations, including completion of the planned Phase 3 trial of Descartes-08 for MG, into mid-2027[9]

新华财经上海7月4日电（谷青竹）早在1961年就被发现的mRNA（信使核糖核酸），疫情期间曾发挥重 要作用。当应急红利已成历史注脚，这项技术如何在更广泛的治疗领域里找到用武之地？ 近期，在港股上市的创新药企云顶新耀举办"mRNA创新技术平台研发日"。透过其展示的AI+mRNA技 术自主平台，以及基于该平台开发的肿瘤及自身免疫疾病领域核心管线的最新进展，公众得以窥见这一 命题的潜在答案。 重估mRNA平台价值 红红火火的mRNA新冠疫苗管线，近年来逐渐走向降温。 以行业巨头为例：2020年疫情期间，因mRNA疫苗具有研发周期短、免疫原性强等优势，英国政府批准 使用辉瑞（Pfizer）和拜恩泰科（BioNTech）合作生产的新冠疫苗，同年底莫德纳（Moderna）旗下的 mRNA新冠疫苗也获得美国FDA的紧急使用授权。到2021年，莫德纳实现营收184.71亿美元、净利润 122.02亿美元，拜恩泰科实现营收189.8亿欧元，双双跻身全球药企TOP50行列。 随着疫情结束，2023年莫德纳全年营收68亿美元，到2024年更是腰斩至32亿美元。拜恩泰科的情况同样 不乐观，在2024年出现了约7亿欧元的净亏损。 不过 ...

Core Viewpoint - Ningbo Meihua Pharmaceutical Co., Ltd. (Meihua) expects significant profit growth for the first half of 2025, driven by increased revenue and improved gross margins [1][2]. Financial Performance - The projected net profit attributable to shareholders is between 46 million to 52 million yuan, representing a year-on-year increase of 142.84% to 174.52% [1]. - The net profit after deducting non-recurring gains and losses is expected to be between 30.6252 million to 36.6252 million yuan, reflecting a year-on-year increase of 55.80% to 86.33% [1]. Business Growth Drivers - The growth in Meihua's performance is attributed to rising pharmaceutical demand, optimized cost control, and increased R&D investment [2]. - The company’s product offerings align with market demands driven by an aging population, enhancing profitability and core competitiveness [2]. International Expansion - Meihua's overseas business has been growing rapidly, with foreign revenue accounting for 69.77% of total revenue in 2024, predominantly from the European market [2]. - The company has established long-term partnerships with renowned pharmaceutical firms such as KRKA, MSD, and SERVIER, showcasing a competitive advantage through an integrated model of formulations and active pharmaceutical ingredients [2]. Innovation and R&D - Meihua is advancing its internationalization efforts while increasing its focus on innovative R&D, including a strategic collaboration with the University of Michigan for the development of the GLP-1 delivery system product "JH389" [3]. - Positive data from animal models have been reported, with plans for commercialization in Europe expected to commence after regulatory approval in 2026 [3]. Diverse Growth Strategies - The company is pursuing multiple business lines, including formulations, CDMO, and innovative projects, which collectively support diversified growth [4]. - Meihua's dual strategy of leveraging both international and domestic markets positions it well to capitalize on global opportunities while ensuring steady development through local resources [4].

上市公司如何平衡好经营的可持续性与未来性？ 日前，在云顶新耀医药科技有限公司(以下简称"云顶新耀")的mRNA(信使核糖核酸)创新技术平台研发 日上，云顶新耀首席执行官罗永庆对《证券日报》记者表示，通过"授权引进+自主研发"的双轮驱动战 略，云顶新耀逐渐实现了这一平衡。 具体来看，在经营层面，云顶新耀以引进的大单品实现了较为稳定的现金流；在布局创新上，公司通过 聚焦mRNA疗法等前沿技术，陆续产出了一些成果。罗永庆表示，基于mRNA创新技术平台，云顶新耀 已开发了通用型现货肿瘤治疗性疫苗EVM14、个性化肿瘤治疗性疫苗EVM16、自体生成CAR-T(嵌合抗 原受体T细胞免疫疗法)三款核心产品管线。"公司已与全球排名前20的药企广泛接洽，未来将通过与国 际伙伴合作，推动这些创新成果走向全球市场。" 夯实竞争优势 彻底治愈肿瘤是许多生命科学家的梦想。然而，现有的治疗技术难以在人体内建立起持久的免疫记忆， 因此对降低肿瘤复发率的作用有限。通过改造mRNA，却有可能取得突破。 "mRNA疗法借由基因编辑技术，改造细胞内负责传递遗传指令的mRNA，进而让细胞生产出特定的蛋 白质，可用于治疗肿瘤、传染病、自身免疫性疾病等 ...

Oncology Pipeline & Strategy - BioNTech has a multiplatform oncology portfolio with over 20 ongoing Phase 2 or 3 trials[7] - BNT327, a PD-L1/VEGF-A antibody, is a priority program with potential as a next-generation IO-backbone, with clinical activity observed across multiple indications in over 750 patients enrolled in 20+ clinical trials[7, 19, 20] - BNT327 combined with chemo in 1L TNBC showed an objective response rate (ORR) of 769% in PD-L1 negative, 563% in PD-L1 low, and 100% in PD-L1 high patients[25] - FixVac and iNeST are novel mRNA cancer immunotherapies targeting tumor-associated antigens and cancer mutations[7, 37] COVID-19 Vaccine & Financials - BioNTech anticipates maintaining a high market share in the U S, EU, and Japan for its COVID-19 vaccine[49] - The company expects total revenues between €1700 million and €2200 million for the financial year 2025[51] - R&D expenses for FY 2025 are projected to be between €2600 million and €2800 million[51] - SG&A expenses are estimated to be between €650 million and €750 million, with capital expenditure for operating activities between €250 million and €350 million[51] Financial Position - As of December 31, 2024, BioNTech's total cash plus security investments amounted to €174 billion, including €97619 million in cash and cash equivalents, €65362 million in current security investments, and €10611 million in non-current security investments[7, 8]

mRNA技术的突出优势在于将药物开发从"分子筛选"转变为"信息设计"，将人体自身变成了一个生产蛋 白药物的智能工厂。此外，mRNA技术集合了软件式平台、通用型生产平台的优势，具备覆盖从肿瘤到 自免、代谢性疾病、传染病、罕见病等多种难治性疾病的药物开发能力。 来源：环球网 【环球网报道 记者 姚倩】BioNTech今年年初在《Nature》发表了题为：RNA neoantigen vaccines prime long-lived CD8+T cells in pancreatic cancer的文章，引发了学术界和制药界的关注，该研究展示了mRNA 新抗原疫苗在胰腺癌治疗中的潜力，通过诱导长效、功能性的T细胞反应，有望为癌症免疫治疗带来新 希望。 肿瘤的复发和转移仍是临床治疗非常大的难题。在肿瘤免疫治疗中，mRNA技术如何发挥作用，国内生 物医药企业的自研mRNA平台又取得了哪些突破性的进展？近日，云顶新耀首席执行官罗永庆接受了环 球网记者的采访。 mRNA肿瘤疫苗预防肿瘤复发后转移的原理是什么？据罗永庆介绍，mRNA技术通过特定编码的mRNA 序列，进入到细胞后，会通过抗原递送细胞捕获、刺激T细胞和B细胞， ...