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]

撰文丨王聪 编辑丨王多鱼 排版丨水成文 耳聋 是人类最常见的感官障碍，先天性耳聋影响了全世界大约 2600 万人，其中高达 60% 的病例是由基因突变引起。 常染色体隐性遗传耳聋9型 （ DFNB9 ） ，是由 编码 耳畸蛋白 （Otoferlin） 的 OTOF 基因突变导致的感音神经性耳聋，占 遗传性耳聋的 2%-8%。耳畸蛋白能使内耳毛细胞响应声音刺激而释放神经 递质，激活听觉神经元，当其功能受损时，声音信号将无法被传达到大脑，从而导致感音神经性耳聋。 此前， 复旦大学附属眼耳鼻喉科医院 舒易来 教授 、 东南大学 附属中大医院 柴人杰 教授等 已经在低龄儿童患者中证实了 AAV 基因治疗 能够安全且有效地恢 复 DFNB9 患者的听力，相关研究发表于《 柳叶刀 》、 Nature Medicine 、 Advanced Science 等期刊。但还有一些关键科学问题亟待解答—— 大龄 DFNB9 患者是否能够受益于 AAV 基因疗法？是否存在最佳治疗年龄窗口？ 2025 年 7 月 2 日 ， 东南大学 附属中大医院 柴人杰 教授联合加州大学欧文分校 曾凡钢 教授，山东省第二人民医院 （山东省耳鼻喉医 ...

Core Insights - The Beijing Brain Science and Brain-like Research Institute is making significant advancements in brain-machine interface (BMI) technology, which is crucial for improving the quality of life for patients with severe neurological conditions [2][4][6] - The institute has developed the "North Brain No. 1" system, which is the first high-throughput, wireless, fully implanted BMI system with over 100 channels, and it has entered clinical verification stages [2][4] - The research focuses on four main areas: brain cognitive principles, major brain diseases, new technologies in neuroscience, and neural decoding and computation [6][7] Group 1: Brain-Machine Interface Developments - The "North Brain No. 1" system allows for real-time decoding of brain signals, enabling patients to control devices through thought, with a notable accuracy of 63% in decoding common phrases for patients with speech impairments [3][4] - The institute has successfully completed surgeries for five patients with spinal cord injuries and ALS, demonstrating significant improvements in motor functions and the ability to perform tasks previously impossible [3][4] - The upcoming "North Brain No. 2" system aims to implant electrodes deeper into brain tissue for more precise signal capture, enhancing the control capabilities for patients [4] Group 2: Gene Therapy Innovations - The institute is also pioneering gene therapy techniques to treat conditions such as severe epilepsy and vision loss, with a new drug that can be injected directly into the epilepsy focus area to control seizures while preserving normal neural function [7] - A novel light-sensitive protein has been developed for visual restoration in patients with retinal diseases, showing significant improvements in vision for treated patients [6][7] - The institute has established a biotechnology company to facilitate the transition from basic research to clinical applications, aiming to shorten the time for technology commercialization [7]

Core Viewpoint - The 42nd National Pharmaceutical Industry Information Annual Conference highlighted the innovative achievements and industrial ecosystem in Changping District, focusing on areas such as innovative drugs, synthetic biology, and medical devices [1] Group 1: Industry Development - Changping District is home to the Zhongguancun Life Science Park, covering approximately 7.2 square kilometers, which hosts over 800 innovative pharmaceutical companies and high-level research institutions [1] - The district's pharmaceutical health enterprises achieved revenues of 33.79 billion yuan from January to April, representing a year-on-year growth of 15.1%, and a total output value of 17.07 billion yuan from January to May, with an 8.8% increase [7] Group 2: Precision Medicine - The Life Valley International Precision Medicine Industrial Park, a key project in the Zhongguancun Life Science Park, focuses on precision diagnosis, targeted therapy, and immunotherapy, aiming to enhance the integration of research, industry, and education [2] - The park covers a total construction area of 240,000 square meters and aims to create an ecosystem that supports the entire process from research incubation to product acceleration and mass production [2] Group 3: Research and Innovation - The Beijing Brain Science and Brain-like Research Institute has established 11 international first-class technology platforms and published 362 papers in top journals, showcasing its strong research capabilities [3] - The institute's "North Brain No. 1" intelligent brain-machine system is the first semi-invasive system with over 100 channels, currently undergoing clinical trials for various neurological disorders [3] Group 4: Company Highlights - Innovent Biologics has developed significant drugs such as Orelabrutinib, the first BTK inhibitor approved for marginal zone lymphoma in China, and has a rich pipeline including a new generation TRK inhibitor [4] - The Beijing Darts International Innovation Center has supported over 60 biopharmaceutical projects, raising over 3 billion yuan in financing and obtaining more than 300 intellectual property rights [6] Group 5: Ecosystem and Collaboration - The Changping District is fostering a comprehensive ecosystem for advanced therapy medicinal products (ATMPs) with strong support from leading CDMO companies like Yiming Biotechnology, which has established multiple production bases and achieved significant milestones in IND applications [6] - The district aims to enhance international compliance levels for local pharmaceutical companies and contribute to the global competitiveness of China's biopharmaceutical industry [6]

编译丨王聪 编辑丨王多鱼 排版丨水成文 饶毅 教授是中国科学和教育领域的杰出改革者。1980 年代，饶毅在美国开启职业生涯，2007 年，他辞去美国西北大学终身教职，全职回国，出任北京大学生命 科学学院院长，他引入了多项举措，使中国生命科学研究重焕生机，其中包括采用终身教职制度和同行评审来评估学者的学术成就。如今， 饶毅 卸任了首都医学 大学校长职务，在北京大学负责一个前沿脑科学研究实验室，并担任其他领导职务。 饶毅 一向以直言不讳著称。2008 年，他放弃美国护照，以抗议前总统乔治·W·布什在 9·11 事件后推行的政策。他还在新冠疫情期间批评了美国的政策，并强 烈反对实验室泄漏论。此外，他还是中国人才引进计划的积极倡导者。 2025 年 6 月 17 日，国际顶尖学术期刊 Nature 发布了一篇对 饶毅 教授的专访 ， Nature 向饶毅询问了他如何看待中国在生命科学领域的作用，以及中国怎样 才能成为生物科技超级大国。 饶毅 教授 中国如何能成为生物科技超级大国？ Nature ：根据《自然》指数，中国在生命科学领域的实力不如在物理科学领域。这是为什么呢？ 只要中国没有类似于美国 NIH 这样的机构， ...

Core Viewpoint - The article discusses the promising clinical data from Klotho Neurosciences (KLTO) regarding the secreted Klotho protein (s-KL), which has shown potential in extending lifespan and combating age-related diseases [2][10]. Group 1: Clinical Findings - KLTO announced that the delivery of s-KL via AAV9 vector effectively increased serum s-KL levels in mice, leading to a 20% increase in lifespan [2][7]. - The expression of the Klotho gene can reduce age-related degeneration across multiple organs, promoting healthy aging [2][10]. Group 2: Market Impact - Following the positive clinical data, KLTO's stock price surged by 787.8% in the U.S. market, with trading volume exceeding 1 billion shares [3]. - The company has established a strong intellectual property position, securing global exclusive licenses for s-KL from the University of Barcelona and ICREA, along with patents in the U.S., Europe, and China [10]. Group 3: Product Pipeline - KLTO's product pipeline includes KLTO-202 for ALS and MS (expected IND submission in Q4 2025), KLTO-101 for Alzheimer's, Parkinson's, and Huntington's diseases (preclinical), and KLTO-301 for atherosclerosis and kidney diseases (preclinical) [11]. - The focus on neurodegenerative diseases represents a significant market opportunity with unmet needs, and the approach targets the fundamental processes of aging rather than just symptoms [11].

Core Viewpoint - The research conducted by Washington University and Altius Biomedical Science Institute successfully designed synthetic enhancers that are more efficient and simpler than natural enhancers, achieving unprecedented cell-type specificity in human cells through iterative deep learning technology [2][6]. Group 1: Research Challenges - Traditional enhancer discovery faces three major challenges: the vast number of candidate enhancers in the human genome, the lack of precision in existing enhancers that often activate multiple cell types, and the complexity of regulatory rules involving various transcription factor combinations and spatial arrangements [6]. Group 2: Research Methodology - The research team developed an iterative deep learning design system, which underwent two cycles of "design-experiment-optimize," starting from 29,891 natural enhancer MPRA activity data to train the model, resulting in the design of 1,037 synthetic enhancers [6]. - The model was refined using real measurement data of synthetic enhancers, reducing the training data volume by 30 times compared to previous generations, and introducing L2 regularization to prevent over-reliance on a single transcription factor [6]. - The second generation achieved a breakthrough with the design of 688 new enhancers, significantly increasing median expression levels in specific cell types, such as a 46.2-fold increase in HepG2 cells and a 6.7-fold increase in K562 cells [6][7]. Group 3: Research Highlights - The specificity of the deep learning-designed enhancers surpassed that of natural controls, and the sequence grammar used for synthetic enhancers was more compact than that of natural enhancers [8]. - Iterative retraining of synthetic enhancers led to designs with superior specificity, and the activity of synthetic enhancers was correlated with single-cell transcription factor expression [8]. Group 4: Applications - The research opens three major application directions: targeted gene therapy for liver cancer, customized tissue-specific enhancers for rare genetic diseases, and the construction of cell-type-specific biosensors in synthetic biology [10]. - This study marks a fundamental shift in the design paradigm of gene regulatory elements, moving from traditional methods to an AI-driven approach that significantly increases success rates [10].

Group 1 - The article highlights the global debut of the AI catheter shaping robot at the OCIN2025 conference, showcasing advancements in intelligent diagnosis and treatment for cerebrovascular diseases [3][6] - The AI catheter shaping robot, developed in collaboration with leading medical teams, has received China's first "AI + treatment" Class III medical device registration certificate [5] - The robot's live surgery demonstration allowed experts to observe its ability to automatically shape catheters and navigate complex vascular paths, emphasizing the innovation in cerebrovascular intervention surgery [6] Group 2 - Lingyi Biotech presented significant clinical progress on gene therapy drugs for Wilson's disease and Gaucher disease type I at the ASGCT2025 conference, indicating important scientific and clinical implications [8] - Huayi Lekan introduced a novel gene therapy candidate for phenylketonuria (PKU) at ASGCT2025, which utilizes an innovative mechanism to silence mutated PAH genes and restore normal function [11][12] - New gene editing therapy GEN6050X for Duchenne muscular dystrophy (DMD) showed promising biomarker data, suggesting its potential to enhance cardiac function and overall patient outcomes [14] Group 3 - Xibiman Biotech announced early clinical results for C-CAR168, a novel CAR-T therapy targeting refractory lupus nephritis, indicating significant progress in treating autoimmune diseases [15] - The NyokAssist® artificial heart device demonstrated encouraging preliminary results at the ISHLT2025 conference, showcasing its design and low complication rates [18] - Shizai Biotech received approval for clinical trials of a new drug for amyotrophic lateral sclerosis (ALS), marking a significant step in addressing one of the "five major incurable diseases" [20] Group 4 - The establishment of a key laboratory for recombinant protein synthesis in Beijing, co-founded by Yuanwei Technology and Tsinghua University, aims to tackle challenges in the industrial application of synthetic biology [22][24] - Haowei Medical completed the first patient enrollment for a clinical trial of absorbable embolization microspheres for symptomatic uterine fibroids, marking a milestone in interventional treatment in China [26] - Sequoia China has invested in over 200 high-growth medical health companies, with more than 45 successfully completing IPOs across various stock markets [27][28]

Core Viewpoint - The EASY system, developed by Westlake University and the Westlake Coacervate Research Team, is a novel non-viral, non-liposomal nucleic acid delivery platform that has been recognized at the American Society of Gene & Cell Therapy (ASGCT) annual meeting, highlighting its potential in gene delivery applications [1][3][19]. Group 1: Introduction to EASY System - The EASY system is the first mammalian coacervate-based delivery system, designed to efficiently deliver various types of nucleic acids, including plasmid DNA, mRNA, and CRISPR-Cas9 tools [1][5]. - The system is inspired by natural cellular coacervates, which are membrane-less organelles formed through liquid-liquid phase separation, playing crucial roles in various biological processes [6]. Group 2: Mechanism and Advantages - The core of the EASY system is the engineered endogenous protein, ProteanFect™, which self-assembles with nucleic acids to form highly ordered nanoparticle structures [7]. - The delivery process involves the formation of stable nanoparticles, efficient cellular uptake through active endocytosis, intracellular release of nucleic acids, and safe degradation of carrier proteins via natural cellular pathways [8]. - The EASY system offers revolutionary advantages, including: 1. Ultra-high loading capacity, approximately 50 times that of lipid nanoparticles (LNP) [9]. 2. Compatibility with various nucleic acids, enabling the delivery of plasmid DNA, mRNA, siRNA, sgRNA, and Cas9 mRNA [10]. 3. High efficiency and low toxicity, achieving comparable or higher gene delivery efficiency while significantly reducing cell mortality [10]. 4. Broad applicability to various primary cells, including NK cells, B cells, and hematopoietic stem cells [10]. Group 3: Commercialization and Clinical Relevance - The ProteanFect series of kits represents the first commercial product of the EASY system, enabling efficient gene delivery and precise gene editing in various primary and hard-to-transfect cells [13]. - The system has demonstrated high gene editing efficiency in primary T cells, with results showing a range of 67%-88% efficiency for gene marking and over 90% efficiency for gene editing [18]. Group 4: Future Outlook - As gene therapy approaches critical clinical applications, the safety and efficacy of delivery systems are paramount. The EASY system provides a new solution by leveraging the natural biological mechanisms of coacervates [19]. - The international recognition of this technology not only affirms China's scientific innovation but also brings new hope to the global gene therapy field, with expectations for the EASY system to play an increasingly important role in clinical applications [19].

沈茜告诉第一财经记者，除了细胞治疗之外，在复旦儿科医院肾脏科和风湿科学科带头人徐虹教授的带 领下，一种目前用于法布雷病的基因疗法未来也有望用于肾病治疗。目前该疗法已经进入IIT（研究者 发起研究）阶段。 除了细胞治疗之外，在复旦儿科医院肾脏科和风湿科学科带头人徐虹教授的带领下，一种目前用于法布 雷病的基因疗法未来也有望用于肾病治疗。目前该疗法已经进入IIT阶段。 近日，浙江大学医学院附属儿童医院肾脏内科公布消息称，一名多药耐药型肾病综合征患儿接受了一种 靶向BCMA/CD70自体CAR-T细胞治疗后，在回输的第3个月，尿蛋白11年来首次转阴。 这一病例的救治成功意味着CAR-T细胞治疗在治疗儿童肾病方面迈出重要的一步，有望帮助更多疑难疾 病患儿实现治愈。 此前，细胞治疗已经用于儿童系统性红斑狼疮以及难治性幼年皮肌炎等疾病。 5月22日，在复旦大学附属儿科医院肾脏科的一场儿童肾病公益项目中，复旦大学附属儿科医院肾脏科 主任沈茜教授对第一财经记者表示："系统性红斑狼疮会导致狼疮性肾炎，肾炎会影响肾功能，如果不 及时治疗就会发展成为尿毒症。" "法布雷病是一种基因遗传性疾病，会导致包括肾衰竭在内的多种脏器的功能损伤 ...