撰文丨王聪 编辑丨王多鱼 排版丨水成文 随着肥胖症、糖尿病及其他代谢综合征的日益普遍， 代谢功能障碍相关脂肪性肝炎 （MASH） 的发病率也在上升。目前，全球约有 1 亿人患有 MASH，预计到 2030 年，这一数字将达到 3.57 亿。 MASH 是肝硬化、肝细胞癌进展的关键因素，也是导致肝移植的主要原因。尽管饮食和运动等生活方式的改变是治疗 MASH 的主要手段，但对大多数患者而言， 通过减重实现逆转颇具挑战性。迄今为止，美国 FDA 仅批准了一款药物 Resmetirom 用于 MASH 的治疗，因此，迫切需要确定新的药物靶点并开发出有效的治 疗手段来应对患者群体庞大且日益增长的 MASH。 2025 年 6 月 30 日，江苏大学 严永敏 团队在 Cell 子刊 Cell Biomaterials 上发表了题为 ： Oral administration of DSPE-PEG-cholic acid-modified ACDVs loaded with siLIMA1 for anti-MASH therapy 的研究论文。 该研究提出了一种大规模获取 人工细胞来源囊泡 （ACDV） 的新策略， ...

Core Viewpoint - Gene therapy is rapidly transforming the landscape of disease treatment, with a focus on integrating artificial intelligence (AI) and biomedicine to enhance accessibility and effectiveness for patients [1] Group 1: Company Background and Development - The company, founded by Zhou Lu, transitioned from business development services to innovative drug development during the COVID-19 pandemic, recognizing the potential in gene therapy [3][5] - The company has successfully secured its first round of investment in 2023, despite entering the market during a capital winter, indicating resilience and strategic timing [6][7] Group 2: Technological Innovations - The company is developing a fifth-generation lentiviral vector platform, which aims to improve the safety and efficacy of gene therapy by enabling in vivo modifications rather than ex vivo [8][10] - The new approach focuses on enhancing the body's natural tumor suppressor genes, rather than merely targeting cancer cells, representing a paradigm shift in cancer treatment [12][20] Group 3: Market Trends and Opportunities - The biopharmaceutical sector has seen significant international transactions, with a total of nearly $50 billion in overseas deals in the first half of the year, surpassing the total sales of electric vehicles [7][25] - The company aims to capitalize on the growing recognition of the biopharmaceutical industry's value, especially as the market rebounds from previous downturns [7][19] Group 4: Regulatory and Ethical Considerations - The company emphasizes the importance of intellectual property (IP) protection and compliance with regulatory standards, which are critical for successful market entry and international collaboration [25][28] - The regulatory landscape is acknowledged as stringent but necessary for ensuring patient safety and drug efficacy, with a commitment to high compliance standards [28][30] Group 5: Talent and Industry Dynamics - The company highlights the need for interdisciplinary talent, combining expertise in medicine, life sciences, and artificial intelligence to drive innovation in drug development [38][39] - The rapid evolution of the biopharmaceutical industry necessitates continuous adaptation of educational programs to align with current technological advancements [39][40]

Core Insights - Gene therapy is rapidly transforming the landscape of disease treatment, with a focus on unlocking its broader, more precise, and accessible potential [1] - The integration of artificial intelligence (AI) with biomedicine is seen as a key variable for the future of gene therapy [1] Company Background - The co-founder of Shentuo Biotechnology, Zhou Lu, transitioned from academia to entrepreneurship, previously establishing a business development service in the UK that assisted over 30 listed companies [3][4] - The COVID-19 pandemic prompted a shift in focus towards innovative drug development, leading to the establishment of Shentuo Biotechnology in 2023 [5][6] Market Dynamics - The capital market showed significant interest in innovative therapies during the early stages of the COVID-19 pandemic, but enthusiasm waned by late 2022, leading to a "capital winter" [6][7] - In the first half of 2023, there was a resurgence in capital investment in biomedicine, with the total overseas transaction amount for biopharmaceuticals reaching nearly $50 billion, surpassing the total sales of electric vehicles [7][8] Technological Innovation - Shentuo Biotechnology is focused on developing a fifth-generation lentiviral vector platform, which aims to enhance safety and efficacy through iterative improvements [8][9] - The new platform allows for in vivo modification of cells, potentially reducing costs and increasing accessibility for patients [10][11] Treatment Paradigm Shift - The company's approach emphasizes enhancing the natural protective capabilities of cells, specifically targeting tumor suppressor genes rather than merely attacking cancer cells [12][13] - This method is likened to "reprogramming" cells, allowing for the potential transformation of cancer cells back into normal cells [14][15]

Core Insights - The article highlights differentiated growth in specific segments, with semiconductor materials growing at 50%, new energy materials at 52%, and biomedical materials at 87%, while traditional structural materials maintain a stable growth rate of 8-10% [2][10]. - Emerging fields are rapidly rising, such as AI servers with high-frequency materials growing at 60%, new energy vehicles with MLCC demand increasing by 100%, and hydrogen energy with a 60% localization rate for proton exchange membranes [2][10]. - The industry chain is evolving, with semiconductor materials seeing a "wafer factory + material factory" bundling development model, and new energy materials adopting a three-in-one model involving automakers, battery manufacturers, and material suppliers [2][12]. Market Dynamics - Channel transformation is evident, with traditional distribution dropping to 40%, while customized services account for 35%, technology licensing for 15%, and joint research for 10% [3][13]. - Reverse innovation is on the rise, with downstream applications leading material customization, breaking the traditional linear research-production-sales model, and it is expected that by 2030, 30% of new material innovations will be driven by application scenarios [3][20]. - Companies are making strategic choices, with leading firms focusing on "materials + equipment + algorithms" full-stack capabilities, SMEs concentrating on niche technologies, and startups exploring disruptive innovations [3][23]. Technological Advancements - Material genome engineering is revolutionizing the R&D model, while breakthroughs in production processes are reshaping cost curves [4][16]. - Future technological directions include extreme performance breakthroughs, intelligent upgrades, green manufacturing, and cross-industry integration [4][20]. Market Outlook - The market is projected to reach 1 trillion yuan by 2025 and exceed 3 trillion yuan by 2030, maintaining a CAGR of 18%, driven by domestic substitution, technological iteration dividends, and the expansion of emerging applications [4][19]. - Key materials to watch include high-end photoresists, aerospace engine materials, solid-state batteries, high-temperature superconductors, perovskite photovoltaic materials, high-frequency materials, MLCCs, UTG glass, and biodegradable materials [4][10]. Industry Background - The innovative materials sector is a cornerstone for China's manufacturing transformation, with the industry size surpassing 6 trillion yuan in 2024, maintaining a 20% annual growth rate [7][8]. - The industry is characterized by intensive policy support, accelerated technological breakthroughs, and expanded application scenarios, particularly in fields like solid-state battery materials and high-temperature superconductors [8][10]. Competitive Landscape - The industry is witnessing an increase in concentration, characterized by a dual-track model of "national teams leading + specialized private firms" [12]. - The collaborative model in the supply chain is innovating significantly, with semiconductor materials adopting a bundling development model and new energy materials forming a three-in-one R&D approach [12][13]. Policy and Institutional Innovation - National strategic layouts provide strong support, with the Ministry of Industry and Information Technology outlining key development directions for advanced materials [15]. - The establishment of a standard system that aligns with international standards is accelerating, although challenges remain due to new EU regulations [15][16]. Investment Strategy Recommendations - Focus on three major tracks: high certainty in domestic substitution (semiconductor precursors, medical-grade polylactic acid), beneficiaries of technological iteration (solid-state electrolytes, superconducting materials), and platform technology companies (materials AI design software) [24]. - Companies should build long-term agreements for certification and procurement, while material firms need to integrate into automotive battery technology roadmaps [23][24].

Core Viewpoint - The article highlights the advancements in citrus breeding technology that have led to the development of more flavorful and nutritious citrus varieties, transforming the market availability of fresh citrus fruits throughout the year [2][3]. Group 1: Citrus Breeding Technology - Traditional breeding methods involve selecting superior plants from various regions, followed by cross-pollination and extensive evaluation of hybrid offspring to identify desirable traits [2]. - Advanced techniques such as embryo grafting have significantly reduced the time required for hybrid varieties to flower and bear fruit, shortening the breeding cycle to just three to five years [2]. - The development of genomic and phenomic technologies has led to the creation of a citrus "chip," which allows for the identification of over 40,000 core genetic markers from more than 300 representative citrus germplasm resources [2][3]. Group 2: Genetic Editing and Future Prospects - The citrus breeding process has been enhanced by gene editing technologies, specifically the CRISPR-Cas9 system, which allows for precise modifications of genetic material to improve traits such as disease resistance and fruit quality [3][4]. - Theoretically, it is possible to concentrate desirable traits such as high sweetness, juiciness, vitamin content, and high yield into a single variety, although these new varieties are currently limited to laboratory research and not yet available in the market [4]. - With the support of new technologies, the industry anticipates the continuous introduction of tastier and healthier citrus varieties, enhancing consumer experience [5].

Core Viewpoint - The recent successful application of personalized gene editing therapy on a 6-month-old infant marks a significant milestone in the treatment of genetic diseases, opening new avenues for patients lacking effective treatment options [1] Group 1: Gene Editing Technology Overview - Gene editing technology allows for precise deletion, insertion, or replacement of specific genes, akin to a "molecular scissors" that can correct and modify defective genes [2][4] - Unlike transgenic technology, which randomly integrates foreign genes into an organism's genome, gene editing modifies the organism's own genes without disrupting the overall structure [2][4] - The evolution of gene editing technology has progressed rapidly, particularly since the advent of CRISPR technology in 2012, which has simplified the process and significantly reduced costs [5][6] Group 2: Applications in Medicine - Gene editing technology is being applied in the treatment of genetic diseases, such as thalassemia, where CRISPR can edit a patient's hematopoietic stem cells to restore normal gene expression [7] - In cancer treatment, CAR-T therapy utilizes gene editing to enhance the immune cells' ability to combat cancer cells, demonstrating the technology's potential in oncology [7] - The technology also aids in modeling complex diseases in research, accelerating drug development by allowing scientists to observe disease progression in genetically edited organisms [7] Group 3: Applications in Agriculture and Bio-manufacturing - In agriculture, gene editing has led to the development of new rice varieties that are resistant to diseases and environmental stress, contributing to global food security [8] - In bio-manufacturing, gene editing enhances production efficiency and reduces costs, such as in the production of biofuels and scarce pharmaceuticals [8] Group 4: Ethical Considerations - The advancement of gene editing technology raises ethical concerns, particularly regarding the editing of human germline cells, which could permanently alter the human gene pool [10] - Ethical guidelines emphasize the importance of prioritizing non-heritable somatic cell editing for therapeutic purposes and prohibiting germline editing in clinical applications [10][11] - The establishment of strict technical boundaries and international regulatory frameworks is essential to prevent ethical violations and ensure that gene editing serves societal welfare [10][11]

Core Viewpoint - The company received an inquiry letter regarding its 2024 annual report from the Shanghai Stock Exchange, which it took seriously and responded to with detailed analyses and clarifications on various financial aspects [1]. Revenue Analysis - In 2024, the company achieved a total revenue of 559 million yuan, with biological culture revenue at 172 million yuan (up 9.08%) and liquid handling revenue at 337 million yuan (up 22.64%) [2][6]. - Overseas revenue reached 351 million yuan, accounting for 63% of total revenue, with a year-on-year increase of 23.50% [2][6]. - The gross profit margin for overseas sales was 43.31%, higher than the domestic margin of 37.54% [2]. Product Sales Breakdown - The sales revenue for liquid handling products significantly contributed to the overall revenue growth, with a notable increase from pipette products, which alone accounted for 30.64% of the total revenue increase [6][11]. - The average selling price of major products increased in 2024 due to price adjustments and a higher proportion of high-value products sold [10][11]. Customer Sales Information - The top five customers for both domestic and overseas sales remained stable, with no significant changes in relationships or sales dynamics [14][23]. - The sales collection rate for domestic customers reached 72.58% by the end of 2024, improving to 100% by May 31, 2025 [14][23]. Third-Party Data Verification - The company matched its export tax refund declarations with actual overseas revenue, showing a minimal discrepancy of -0.67% [23][24]. - The foreign exchange administration's collection data also showed a small difference of 1.84% compared to actual overseas revenue, indicating reliable revenue reporting [23][24].

金十数据7月4日讯，从东南大学附属中大医院获悉，该院耳鼻咽喉头颈外科柴人杰教授联合多家单位在 国际顶级医学期刊《自然·医学（Nature Medicine）》发表研究论文，揭示了腺相关病毒基因疗法在常染 色体隐性遗传性耳聋中的作用。该院表示，这一发现具有重要临床指导价值，有望让全球数百万遗传性 耳聋患者获得"听清世界"的希望。 （央视新闻） 我国科研人员在基因治疗耳聋研究方面取得新突破 ...

Core Findings - An international research team published a study in *Nature* indicating that fine particulate matter pollution may increase the risk of cancer gene mutations in non-smokers, leading to a higher likelihood of lung cancer compared to the effects of secondhand smoke [1] Group 1: Research Overview - The study was conducted by researchers from the National Institutes of Health and the University of California, San Diego, analyzing tumor samples from 871 non-smoking lung cancer patients across 28 regions globally [1] - The research found a strong correlation between fine particulate matter pollution, including vehicle emissions and industrial discharges, and the increase in cancer-related gene mutations in non-smokers [1] Group 2: Genetic Implications - The study identified mutations in the TP53 gene and various other mutations previously associated with smoking, which were linked to exposure to air pollution [1] - Air pollution was found to cause premature shortening of telomeres, leading to early cellular aging and potentially accelerating tumor progression [1] Group 3: Comparative Analysis - The findings suggest that air pollution has a higher likelihood of inducing gene mutations in non-smokers compared to secondhand smoke [1]

Core Viewpoint - Rocket Pharmaceuticals, Inc. is facing a class action lawsuit due to allegations of providing misleading information regarding its clinical trials, particularly concerning the safety of its gene therapy product RP-A501 for Danon disease [3][4]. Group 1: Lawsuit Details - The class action lawsuit is titled Ho v. Rocket Pharmaceuticals, Inc. and covers purchasers of Rocket Pharmaceuticals securities from February 27, 2025, to May 26, 2025 [1]. - The lawsuit alleges that Rocket Pharmaceuticals and a top executive violated the Securities Exchange Act of 1934 by concealing material adverse facts about the safety and clinical trial protocol of RP-A501 [3]. - It is claimed that the company failed to disclose serious adverse events, including the death of a participant, which occurred after a protocol amendment that introduced a new immunomodulatory agent [3][4]. Group 2: Impact on Stock - Following the announcement of a clinical hold by the U.S. Food and Drug Administration on May 27, 2025, due to a serious adverse event, Rocket Pharmaceuticals' stock price experienced a decline [4]. Group 3: Legal Process - Investors who purchased Rocket Pharmaceuticals securities during the class period can seek appointment as lead plaintiff in the lawsuit, which allows them to act on behalf of other class members [5]. - The lead plaintiff can choose a law firm to represent the class, and participation as lead plaintiff does not affect the ability to share in any potential recovery [5]. Group 4: About Robbins Geller - Robbins Geller Rudman & Dowd LLP is a leading law firm specializing in securities fraud and shareholder litigation, having secured over $2.5 billion for investors in 2024 alone [6]. - The firm has been ranked 1 in the ISS Securities Class Action Services rankings for four out of the last five years, indicating its strong track record in obtaining monetary relief for investors [6].