数据来源：沙利文 小核酸药物，即寡核苷酸药物，是由十几个到几十个核苷酸串联组成的短链核酸，作用于 premRNA或mRNA，通过干预靶标基因表达实现疾病治疗目的。 进入高速爆发期的小核酸药物，正逐渐成为市场关注的焦点。 01 什么是小核酸药物？ 核酸药物可主要分为小核酸药物和mRNA两大类。小核酸药物主要包括反义核酸（ASO）、小干扰 核酸（siRNA）、微小RNA（miRNA）、核酸适配体（Aptamer）和转运RNA（tRNA）碎片。mRNA产 品可分为mRNA疫苗和mRNA药物。 自二十世纪70年代以来，随着科学界对于机制的不断发现，小核酸药物开启早期的研发探索。1978 年哈佛大学科学家首次提出反义核酸的概念，1998年Andrew Fire和Craig Mello在线虫中首次揭示了 RNA干扰的作用机理（RNAinterference，RNAi），两人因此于2006年获得诺贝尔奖。 但在2010年左右，由于RNAi的不稳定性、潜在的免疫原性及递送系统的缺乏，研发迟迟未有突 破，导致大型制药企业纷纷撤资，行业发展遭遇瓶颈。2014年后，随着小核酸偶联递送系统Gal Nac的 发展，极大地促进和推动了小 ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 诺贝尔奖 于 10 月 6 日陆续揭晓，率先揭晓的将是 诺贝尔生理学或医学奖 ，该奖项是 旨在表彰在生理学或医学领域作出最重要发现的人。自 1901 年 首次颁发以来，该奖项共颁发 115 次，共有 229 人获奖。其中， 屠呦呦 获得 2015 年度诺贝尔生理学或医学奖，是首位获得诺贝尔科学奖项的中国本土科学 家，也是迄今为止唯一一位获得生理学或医学奖的华人科学家。 2025 年 诺贝尔生理学或医学奖 授予 Mary Brunkow 、 Fred Ramsdell 、 Shimon Sakaguchi （ 坂口志文 ） 三人。 获奖理由 ：for their discoveries concerning peripheral immune tolerance（ 因他们在外周免疫耐受方面的发现而获奖 ）。他们发现了免疫系统的"保安"—— 调节性T细胞（Treg），为新研究领域奠定基础。这一发现促成了潜在医疗手段的发展，目前已进展到临床试验中。人们希望借此能够治疗或治愈自身免疫疾病， 提供更有效的癌症治疗方法，并预防干细胞移植后的严重并发症。 诺贝尔奖官网网 ...

Group 1 - The company Xinnoway (300765.SZ) signed a share transfer agreement with Enbipu Pharmaceutical to acquire a 29% stake in Jushi Biotech for a cash consideration of 1.1 billion yuan, increasing its ownership from 51% to 80% [1] - The transaction is based on an asset evaluation report from a qualified institution in accordance with the Securities Law [1] Group 2 - Jushi Biotech is an innovative biopharmaceutical company focused on antibody drugs, antibody-drug conjugates (ADC), and mRNA vaccines, with a strong emphasis on independent research and development [2] - The company has established a competitive R&D system and a promising product pipeline, covering three major therapeutic areas: oncology, autoimmune diseases, and major infectious diseases [2] - Jushi Biotech has numerous projects in development, with key products like Enlansumab and Omabuzumab set to launch in 2024, and several others in critical clinical trial phases [2] - The company has become a benchmark in the domestic monoclonal antibody and ADC innovation drug industry, providing safe, effective, and accessible biotherapy solutions globally [2] - Jushi Biotech is one of the first domestic companies to achieve mRNA vaccine industrialization and has a comprehensive layout in ADC and monoclonal antibody pipelines [2]

科技是同疾病较量最有力武器 转自：新华财经 又是一年丹桂香。2025首都国际医学大会如约而至。国内外医药卫生及相关领域政、产、学、研、商多 方力量日前汇聚北京首钢园，与会嘉宾围绕全球卫生健康治理、科技创新与前沿突破、国际医改经验与 中国实践等重要议题展开深度交流，为促进国际医学发展和全球卫生治理积极建言献策。 "人民健康是社会文明进步的基础。北京作为首都，坚决贯彻落实健康中国战略，全力实施健康优先发 展战略，将健康融入所有政策，以三医联动为抓手，全方位、全周期保障人民健康。"北京市政府副秘 书长靳伟说。 医疗服务能力持续提升，全市1.2万家医疗卫生机构面向全国提供疑难重症诊疗服务；医学创新资源不 断丰富，拥有12类13家国家医学中心，23家国家临床医学研究中心以及北生所、北脑所等4个新型研发 机构；国际创新医药公园吸引辉瑞、默沙东、礼来等8家知名药企在京新设创新主体；医药健康产业发 展日益活跃，成为全国首个医药健康产业破万亿元城市…… 亮眼成绩单的背后，惠及人民健康的医学科技成果也积极落地。 "北京大学以AI赋能临床研究和整体诊疗平台。以肾病防控和精准诊疗领域为例，经过长期积累和实 践，我们开创了首个肾病领域 ...

相比之下，共价闭合的 环状 RNA （circRNA） 在稳定性、免疫原性以及安全性方面均优于经过化学修饰的 mRNA 和基于病毒载体的疗法。此外，circRNA 通过 使用内部核糖体进入位点 （IRES） 序列，无需昂贵的 5' 端加帽处理，且无需化学修饰即可获得低免疫原性。这些优势使 circRNA 成为下一代 RNA 疗法平台的 有力候选者。 2025 年 9 月 26 日，上海交通大学医学院/海南医科大学 邹卫国 、 Suo Jinlong 、中国科学院分子细胞科学卓越创新中心 黄友葵 、中南大学湘雅医院 雷光华 、 在 Nature 子刊 Nature Communications 上发表了题为： Circular RNA-based protein replacement therapy mitigates osteoarthritis in male mice 的研究 论文。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 mRNA 疫苗 在 COVID-19 期间大获成功 ，这重新激发了人们对 RNA 疗法在临床应用方面的兴趣。然而，mRNA 疗法在稳定性、瞬时表达、免疫原性以及使用 化学修饰核苷导致 ...

Group 1 - The core viewpoint of the news is the collaboration between Baiaosaitu and Merck to evaluate antibody-conjugated lipid nanoparticles (Ab-LNP) for new applications in nucleic acid drug delivery, highlighting the strong interest in "precise delivery" in the biopharmaceutical sector [1][5] - The nucleic acid drug market is rapidly growing, with mRNA therapies gaining mainstream acceptance, but the delivery of nucleic acids remains a significant bottleneck [2][5] - The global lipid nanoparticle (LNP) market is projected to grow from $1.1 billion in 2025 to $3.5 billion by 2034, with a compound annual growth rate (CAGR) of 13.3% [2] Group 2 - Antibody-modified LNPs have shown promising results in animal models, significantly improving delivery efficiency to non-liver tissues, which is crucial for treating various diseases [2][3] - The partnership between Baiaosaitu and Merck marks the third deepening of their collaboration, indicating a new phase in the "antibody × delivery" model [4] - The trend towards platform and modular design in LNP technology is emerging, with new molecular forms like antibodies and bispecific antibodies expanding delivery capabilities [4] Group 3 - The investment landscape is shifting towards antibody-conjugated LNPs as a potential solution to the delivery bottleneck in nucleic acid drugs, with significant capital being directed into this area [3][5] - The collaboration between Baiaosaitu and Merck reflects the increasing involvement of Chinese biotech companies in the global restructuring of drug delivery technologies [5]

Core Insights - BioNTech has evolved from being a leading mRNA vaccine company to a diversified player in the biopharmaceutical industry, engaging in multiple therapeutic areas including dual antibodies (ADC) and mRNA vaccines [1][14] - The company has made significant strategic moves, including a $12.5 billion acquisition of CureVac and a $111 billion licensing deal with BMS for the PD-L1/VEGF dual antibody BNT327, which has reshaped the competitive landscape of the mRNA market [2][5] Group 1: Strategic Developments - BioNTech's acquisition of CureVac has strengthened its position in the mRNA sector, allowing for enhanced capabilities in cancer immunotherapy and a more robust pipeline [10][11] - The company has established a diverse pipeline in ADCs, with significant investments in multiple ADC candidates, including DB-1303/BNT323 and DB-1311/BNT324, totaling over $16 billion [4][12] - BioNTech's collaboration with Chinese biotech firms has expanded its ADC and dual antibody pipeline, enhancing its future growth prospects [4][10] Group 2: Financial Performance - BioNTech's market capitalization reached $27.04 billion, significantly higher than Moderna's $9.78 billion, reflecting investor confidence in its diversified strategy [2] - The company generated substantial revenue from its COVID-19 vaccine, with sales reaching $15 billion in 2021 and $17.3 billion in 2022, which has been reinvested into new therapeutic areas [2][14] Group 3: Clinical Advancements - BioNTech's PM8002/BNT327 is currently in advanced clinical trials, showing promising results in treating extensive-stage small cell lung cancer (ES-SCLC) with a confirmed overall response rate (cORR) of 76.3% [6][7] - The company is actively conducting multiple clinical trials combining PM8002/BNT327 with various ADCs, indicating a strong commitment to innovative treatment strategies [8][9] Group 4: Future Outlook - BioNTech aims to leverage its extensive pipeline and strategic partnerships to maintain its leadership in mRNA technology and expand its presence in oncology and infectious diseases [14] - The company is focusing on the commercialization of its diverse product offerings, particularly in the oncology space, where it has established a strong foothold [10][14]

Core Viewpoint - The recent events indicate that the CDC is facing unprecedented challenges in its internal operations and external trust, particularly following the dismissal of its director and subsequent resignations of senior officials [1][6]. Group 1: Leadership Changes - CDC Director Susan Monarez was dismissed less than a month after her appointment due to conflicts with HHS Secretary Robert F. Kennedy, Jr. regarding vaccine policies [2][3]. - Four senior officials from the CDC resigned in protest, highlighting the detrimental impact of political interference on scientific decision-making [6][8]. - Kennedy's leadership has led to significant changes in the CDC, including the replacement of the entire Immunization Practices Advisory Committee and the cessation of mRNA vaccine research funding [4][5]. Group 2: Internal Morale and Trust Issues - The morale within the CDC is reported to be low, with employees feeling anxious and uncertain about their roles and the agency's direction [7][9]. - The agency has faced budget cuts and staff reductions, which have further eroded trust and operational efficiency [7][8]. - Recent violent incidents, including a shooting at the CDC headquarters, have exacerbated the atmosphere of fear and insecurity among staff [7]. Group 3: Public Health Implications - The ongoing turmoil within the CDC comes at a critical time as the U.S. enters a season of increased respiratory illnesses, raising concerns about the effectiveness of public health responses [8][9]. - The politicization of public health issues under the current administration may lead to decreased vaccination rates and undermine the effectiveness of public health measures [8][9]. - The CDC's ability to respond to public health emergencies is at risk due to leadership instability and potential talent loss, which could weaken the overall public health infrastructure in the U.S. [9].

Core Insights - Yale University researchers have developed a novel mRNA vaccine platform aimed at significantly enhancing immune responses and improving the efficacy of mRNA vaccines, expanding their potential applications in various disease prevention and treatment [1][2] Group 1: Vaccine Technology - The new platform, named "Molecular Vaccine Platform" (MVP), addresses the limitations of current mRNA vaccines by ensuring that antigens are effectively recognized by the immune system [1] - The MVP technology incorporates a "cellular GPS" module that directs mRNA-encoded proteins to the cell surface, enhancing antigen exposure and improving immune system recognition and response [1][2] Group 2: Experimental Results - Laboratory tests on various pathogens, including monkeypox, human papillomavirus (HPV), and varicella-zoster virus (which causes shingles), demonstrated stronger antigen expression, higher antibody levels, and more active T-cell responses [2] - Researchers are working to extend this technology to a broader range of diseases, including cancer, HIV, and autoimmune diseases, aiming to transition mRNA technology from infectious disease prevention to comprehensive medical applications [2]

Core Insights - The company reported a revenue of 1.17 billion yuan for the first half of 2025, with R&D investment amounting to 209 million yuan, representing 17.91% of revenue, an increase of 7.54 percentage points year-on-year [1] - The company has established eleven core technology platforms focusing on various drug types, including nucleic acid drugs and high-end traditional Chinese medicine, and has a pipeline of over twenty key projects [1] - As of June 30, 2025, the company has 42 R&D projects, including 21 innovative drugs and 21 generic drugs, and has accumulated 349 patents, with 21 new applications and 32 new patents granted in the first half of 2025 [1] R&D Progress - The company made significant advancements in key innovative drugs, with several products in the NDA review stage, including traditional Chinese medicine injections and other innovative formulations [2] - The company has successfully launched its small nucleic acid drug and mRNA vaccine R&D and pilot production platforms, which are now operating efficiently [2] - The company aims to focus on three major therapeutic areas: cardiovascular diseases, tumors, and infectious diseases, while continuing to develop a diverse pipeline through various methods such as independent R&D and collaborations [2]