基因递送
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频登顶刊!西湖大学首创凝聚体递送系统,助力全球科学研究
生物世界· 2025-12-17 04:21
在生命科学研究与基因治疗转化的赛道上, 基因递送 是当之无愧的核心环节 —— 它就像一座 " 桥梁 " , 负责将核酸分子 (例如 DNA 、 RNA 、 RNP 等) 精准送进细胞内,实现基因的过表达、敲低或编辑,从 而帮助科研人员解析基因功能、探索疾病机制。但长久以来,这座 " 桥梁 " 却一直存在诸多 " 通行障碍 "。 基因递送的 " 老难题 " :传统工具的局限性 一条不同的技术路线:从基础生物学出发的重新思考 针对以上困境, 西湖大学 联合 西湖凝聚体 团队,自主研发出全球首创的 ProteanFect 基因递送系统 , 构建了 非病毒、非脂质体、非电转 的全新递送路径 。其核心创新点在于 " 仿生内源蛋白凝聚体 " 的设计 思路:该系统模拟细胞内源性蛋白液液相分离特性,通过仿生机制实现核酸分子的高效包裹与递送。这种 独特的递送模式,既规避了病毒载体的免疫原性风险,又摆脱了脂质体对细胞类型的适配限制,同时避免 了物理手段对细胞的损伤,为基因递送搭建起了一条更安全、更通畅的 " 新桥梁 " 。 从 递送类型 来看, ProteanFect 可实现 mRNA/ 质粒过表达、 siRNA 基因敲低、 C ...
从实验室痛点到全球科研赋能,科学家如何用科研逻辑创业?
财富FORTUNE· 2025-11-05 13:29
Core Viewpoint - The article highlights the innovative approach of Yunzhou Biotech in revolutionizing the production and design of gene vectors, addressing significant inefficiencies in the life sciences research sector and extending its services from research to clinical applications [1][10][12]. Group 1: Background and Challenges - The inception of Yunzhou Biotech stemmed from a challenge faced by researchers in constructing gene vectors, which often took months to ensure quality [2][4]. - The founder, Dr. Lantian, recognized that the lack of reliable suppliers for gene vectors led to a systemic inefficiency in the industry, consuming valuable time and resources [4][6]. Group 2: Innovative Solutions - Dr. Lantian proposed a modular and standardized approach to gene vector construction, akin to assembling with LEGO blocks, allowing researchers to select components from a library of over ten thousand functional elements [6][9]. - The launch of the VectorBuilder platform in 2015 marked a significant advancement, enabling users to design and order custom gene vectors with minimal expertise, thus freeing them from repetitive tasks and enhancing research efficiency [9][10]. Group 3: Business Expansion and Clinical Applications - Yunzhou Biotech has expanded its services from basic research to include Contract Research Organization (CRO) and Contract Development and Manufacturing Organization (CDMO) services, creating a comprehensive service chain from research to commercialization [11][12]. - The company has successfully delivered over 300 batches of customized clinical-grade plasmids and viral vectors, with more than 70 for large-scale clinical production, and has achieved FDA DMF registration for its products [12]. Group 4: Commitment to Research and Development - Dr. Lantian continues to prioritize research, leading his team in publishing multiple preprints and addressing common design flaws in laboratory-made plasmids [14][16]. - The introduction of the miniVec™ plasmid system represents a significant innovation, enhancing safety and reducing production costs while being more suitable for clinical applications [16]. Group 5: Social Responsibility and Vision - Yunzhou Biotech actively contributes to the scientific community by providing free research tools, such as the VectorBee software, and engaging in collaborations to support rare disease treatments [18]. - The company's vision, as articulated by Dr. Lantian, is to leverage scientific advancements to improve the world, reflecting a commitment to both innovation and social impact [18].
微滔生物获启明创投领投超亿元天使轮融资,引领体内CAR-T技术变革
IPO早知道· 2025-11-05 06:32
Core Insights - Weitao Biotech has completed over 100 million RMB in angel round financing, led by Qiming Venture Partners, with funds aimed at optimizing core technology platforms and advancing clinical development of its first candidate product in autoimmune diseases [2][3] Company Overview - Weitao Biotech, established on June 25, 2025, is a spin-off from Beijing Shali Bio-pharmaceutical Co., Ltd., focusing on in vivo CAR-T therapy [2] - The company aims to revolutionize treatment for hematological malignancies and autoimmune diseases by enabling CAR expression directly in patient T cells, eliminating the need for traditional autologous CAR-T therapy processes [2] Technology and Innovation - Weitao Biotech's in vivo CAR-T therapy utilizes a targeted LNP delivery system, which enhances targeting efficiency, safety, and production compatibility, effectively reducing off-target risks [3] - The proprietary technology allows for efficient T cell activation and long-lasting CAR expression, demonstrating excellent B cell clearance effects in preclinical models and good safety for repeated dosing [3] Investment and Future Plans - Qiming Venture Partners, as the lead investor, emphasizes the global leading potential of Weitao Biotech's targeted LNP delivery platform in addressing traditional CAR-T therapy challenges [4] - The CEO of Weitao Biotech expresses commitment to filling unmet clinical needs through technological innovation, with the recent financing expected to accelerate clinical validation of its in vivo CAR-T pipeline [4]
自带导航,主动敲门,CRISPR有了新型“快递车”
Ke Ji Ri Bao· 2025-09-23 08:52
Core Insights - CRISPR technology represents a revolutionary gene-editing method that offers unprecedented hope for treating genetic disorders, cancer, and rare diseases by precisely modifying disease-causing genes [1] - A significant breakthrough has been achieved by a team from Northwestern University, which has developed a new delivery system for CRISPR tools, enhancing efficiency and safety in gene therapy applications [1][3] Delivery Mechanisms - Current methods for delivering CRISPR into cells primarily rely on modified viruses and lipid nanoparticles (LNPs), each with distinct limitations [2] - Modified viruses are efficient at entering cells but pose safety risks due to immune responses and limited cargo capacity [2] - LNPs are safer but have low delivery efficiency, often getting trapped in cellular compartments, which hinders the effectiveness of gene tools [2] New Delivery System - The new system, termed "Lipid Nanoparticle Spherical Nucleic Acids" (LNP-SNA), features a special DNA shell that enhances visibility and acceptance by cells, significantly improving delivery efficiency [3] - This innovative delivery vehicle has been shown to enter cells over three times more efficiently than traditional lipid particles, with reduced toxicity and a threefold increase in successful gene editing probability [3] - The accuracy of gene repair has improved by over 60%, which is crucial for minimizing health risks associated with erroneous edits [3] Versatility and Future Applications - The LNP-SNA technology is modular, allowing for tailored delivery to specific cell types, such as liver, brain, or cancer cells, enhancing precision in treatment [4] - This new system has demonstrated excellent delivery results across various human cell types, including skin, immune, kidney, and bone marrow stem cells [4] - Seven drugs based on similar spherical nucleic acid technology are currently in human clinical trials, with some targeting cancer treatment [4] - The advancement in delivery mechanisms is critical for the future of gene editing therapies, potentially enabling the treatment of previously untreatable diseases [4]
广州黄埔生物医药产业的弯道超车:培育土壤、打造真创新
2 1 Shi Ji Jing Ji Bao Dao· 2025-09-02 11:27
Core Insights - The competitiveness of the biopharmaceutical industry ultimately reflects the cluster effect [1][11] - The biopharmaceutical industry is a core track of global technological revolution and industrial transformation, serving as a key indicator of regional economic high-quality development [2] Industry Development - The Yangtze River Delta region, centered around Shanghai Zhangjiang and Suzhou BioBAY, has established a first-mover advantage in the biopharmaceutical field due to early policy layouts, dense research resources, and a complete industrial chain [2] - Guangzhou Huangpu District has not lagged despite being a latecomer; it has optimized its policy system, built talent aggregation platforms, and focused on breakthroughs in niche markets, gradually compensating for industrial chain shortcomings [2][12] Key Companies and Innovations - Yunzhou Biotech, founded by Dr. Lan Tian in Huangpu District in 2014, focuses on gene delivery, a critical link between basic research and clinical application [3] - The company developed the world's first intelligent design and ordering platform for gene carriers, integrating over 120 carrier systems and more than 2 million carrier components, significantly lowering the barriers for researchers [4][5] - Yunzhou Biotech has become the world's largest custom gene carrier supplier, serving over 7,000 institutions in more than 130 countries, with a cumulative delivery of over 1 million carriers by September 2024 [5][6] Market Position and Achievements - In 2023, Yunzhou Biotech achieved a valuation of 7 billion, becoming a unicorn and the first biopharmaceutical company in Guangzhou to reach this status [6] - The company has established a full-service capability in the gene drug development chain, including CRO and CDMO services, with its products approved for clinical trials in the U.S. [6][7] Competitive Landscape - Innovation in drug development is a core competitive advantage in the biopharmaceutical industry, with companies like Kangfang Biopharma emerging as leaders in the field [8] - Kangfang Biopharma has developed over 50 innovative drug candidates, with 24 products undergoing clinical trials, and has successfully commercialized 7 first-class new drugs [10] Ecosystem and Collaboration - Huangpu District's "bending overtaking" is not merely the success of a single enterprise but a result of creating a quality industrial ecosystem that fosters collaboration between leading external companies and local enterprises [12] - The presence of major companies like Hengrui Medicine and Nuocheng Jianhua in Huangpu has attracted a network of supporting enterprises, enhancing the regional industrial chain [13][14]
西湖大学开发基因递送黑科技,全球首个哺乳动物蛋白凝聚体递送系统丨ASGCT 口头报告精选
生物世界· 2025-05-26 02:52
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].