基因治疗
Search documents
公司互动丨这些公司披露在医药生物、机器人等方面最新情况
Di Yi Cai Jing· 2026-02-26 14:42
2月26日,多家上市公司通过互动平台、披露投资者关系活动记录表等渠道披露公司在医药生物、机器 人等方面最新情况: 利亚德:公司已与国内外诸多知名机器人厂家建立合作 灿瑞科技:公司的智能传感器芯片没有应用于人形机器人 【电池】 万顺新材:高达因电池铝箔已进入小批量供应阶段 【化工】 【医药生物】 长春高新"治疗男童发育不良新药"引爆股价,公司回应:项目还处于早期阶段 博雅生物:未来将围绕基因治疗、抗体药物等领域开展研究 【机器人】 利亚德:公司机器人业务属于AI与空间计算板块,该板块营收占总营收比重约5个百分点 雅化集团:目前津巴布韦出口禁令不会对公司正常生产经营造成影响 东岳硅材:电力液体胶产品正积极对接市场 (本文来自第一财经) ...
一日内两成果同登《自然》,新华医院医工交叉与基因治疗领域迎来重大突破
Xin Lang Cai Jing· 2026-02-19 11:42
转自:新华财经 新华财经上海2月19日电 (谷青竹)19日,上海交通大学医学院附属新华医院(以下简称"新华医院") 传来重大科研消息:该院两项原创性研究成果登上国际顶级学术期刊《自然》(Nature),分别在罕见 病AI诊断、神经发育疾病基因治疗领域实现重大突破。 研究团队首先构建了复刻患者核心症状的人源化突变小鼠模型,为治疗研究搭建精准的"疾病模拟平 台";并创新性设计出新型腺嘌呤碱基编辑器TeABE,与传统技术相比,全程无DNA双链断裂风险,大 幅降低基因组紊乱风险。实验显示,编辑器成功抵达突变小鼠多个脑区实现靶向修复,其社交回避、认 知迟钝、运动不协调等行为异常得到显著改善。更重要的是,团队在非人灵长类(猕猴)模型中开展实 验,也成功检测到明确的碱基编辑活性,验证了该技术的跨物种应用可行性,为后续临床试验积累了充 足前期数据。 据悉,新华医院团队深耕儿童神经发育障碍领域二十余年,致力于其遗传及环境病因的基础到临床转化 研究。李斐团队聚焦基因精准编辑,在《自然》发表重磅成果的同时,还在环境病因研究方面有所突 破,近期牵头完成的"神经发育障碍的生命早期环境风险因素识别和防控体系建立与推广"项目,在2025 ...
杭企嘉因生物冲刺港股IPO
Mei Ri Shang Bao· 2026-02-05 22:25
Group 1 - The core viewpoint of the article is that gene therapy is becoming a central focus for companies, with Hangzhou Jiayin Biotechnology Co., Ltd. officially submitting its prospectus to the Hong Kong Stock Exchange [1] - Jiayin Biotechnology, established in 2019, specializes in the development of gene therapies and oligonucleotide drugs, utilizing a dual technology platform system consisting of AAVarta and SODA [1] - The company’s research covers a wide range of diseases, including neurological disorders, ophthalmic diseases, and various hereditary and chronic conditions, resulting in a diversified product pipeline [1] Group 2 - Jiayin Biotechnology currently has 10 candidate products in development, with 4 of them already in clinical stages [2] - The core product EXG001-307, targeting SMA1 type, is set to enter Phase III clinical trials in the second half of this year and is considered the potential best-in-class candidate for SMA1 treatment in China [2] - The funds raised from the IPO will primarily be used for the further development of core products EXG001-307, key products EXG102-031 and EXG202, as well as to enhance the core technology platform and support daily operations and business expansion [2]
Vita:马丽佳/李颜团队开发新型AAV,打一针,在体内自动生成CAR-T细胞,治疗系统性红斑狼疮
生物世界· 2026-02-04 04:11
Core Viewpoint - CAR-T cell therapy is a revolutionary cancer treatment that utilizes genetically modified T cells to target and kill cancer cells, and it has shown promise in treating autoimmune diseases as well [2]. Group 1: Challenges of Traditional CAR-T Therapy - The preparation of CAR-T therapy is complex and expensive, involving blood extraction, genetic modification, and expansion, which can take several weeks and incur high costs [3]. - A pre-treatment with chemotherapy is required to clear the patient's existing lymphocytes before reinfusion, leading to significant side effects [3]. Group 2: Innovations in CAR-T Therapy - Researchers have proposed in vivo CAR-T cell therapy, which allows for the direct generation of CAR-T cells in the body through injection, potentially simplifying and broadening access to CAR-T therapy [4]. - A study published on February 3, 2026, introduced a novel adeno-associated virus variant, AAV6-M2, which can specifically target human T cells and generate CAR-T cells effectively in a mouse model of systemic lupus erythematosus (SLE) [4][5]. Group 3: Mechanism and Efficacy - The AAV6-M2 variant was developed using AI-assisted design and high-throughput screening, allowing it to efficiently target resting T cells without prior activation, which is crucial for in vivo CAR-T cell generation [8]. - In tests on humanized mouse models, a single injection of AAV6-M2-CD19CAR led to a 77.5% conversion rate of CD8+ T cells to CAR-T cells, effectively eliminating B cells and improving symptoms of lupus [10]. Group 4: Safety and Advantages - AAV6-M2 demonstrated significantly reduced liver tropism compared to wild-type AAV, lowering the risk of liver toxicity and enhancing the cost-effectiveness of the treatment [13]. - The study indicates that AAV-mediated CAR delivery can generate functional human CAR-T cells in vivo, marking a shift from personalized treatments to more universal, off-the-shelf products [5][14]. Group 5: Future Prospects - This research signifies a platform innovation, proving that a single systemic injection of an AAV vector can generate durable, functional human CAR-T cells, paving the way for rapid clinical translation [14]. - The application of AAV-mediated gene therapy is expanding from genetic diseases to cancer and autoimmune diseases, opening new avenues for accessible cell and gene therapies [15].
Cell:AAV基因治疗新突破,路中华/姜玉武/刘太安等开发AAVLINK技术,实现大基因高效递送
生物世界· 2026-01-31 03:05
Core Viewpoint - The article discusses the revolutionary AAVLINK technology, which overcomes the limitations of adeno-associated virus (AAV) in gene therapy by enabling the delivery of large genes, thus providing new treatment possibilities for genetic diseases such as autism and epilepsy [2][27]. Group 1: AAVLINK Technology Overview - AAVLINK stands for "AAV with translocation linkage," utilizing the Cre/lox system for DNA recombination, allowing large genes to be split into smaller segments and delivered via multiple AAVs [4]. - The technology enables precise reassembly of these gene segments within cells, leading to the expression of functional proteins [4]. Group 2: Advantages of AAVLINK - AAVLINK demonstrates significant advantages over existing methods for delivering large genes, such as protein splicing and RNA splicing, which often have low efficiency and produce many by-products [6]. - In experiments, AAVLINK achieved over 25 times higher efficiency in reconstructing fluorescent proteins compared to the intein method, and up to 245 times higher in triple vector delivery [12]. - AAVLINK produces minimal by-products, ensuring safety by avoiding truncated proteins that could interfere with normal functions [12]. Group 3: Application in Disease Models - AAVLINK was tested in two disease models: Phelan-McDermid syndrome (PMS) and Dravet syndrome, successfully delivering and reconstructing the SHANK3 and SCN1A genes, respectively, leading to improved behavioral and survival outcomes in mouse models [8][11]. Group 4: CRISPR Delivery Capability - AAVLINK can also deliver large CRISPR-Cas systems, enabling gene editing and regulation, which opens new avenues for treating genetic disorders, such as lowering cholesterol levels by editing the PCSK9 gene [11]. Group 5: Safety Enhancements and Resource Availability - The development of AAVLINK 2.0 addresses potential safety risks associated with the long-term presence of Cre enzyme in the body, paving the way for clinical applications [15][17]. - AAVLINK has established a resource library containing 198 large disease-related genes and 5 CRISPR tools, allowing researchers to accelerate therapy development [19][21].
【央视新闻】我国新型基因治疗策略有望推动孤独症、癫痫等疾病治疗
Yang Shi Xin Wen· 2026-01-29 03:44
为此,研究团队创新性地提出了一种名为" AAVLINK"的新方法。该方法将长基因分成两段,分别 装进两个AAV中,一个AAV携带的基因片段装上特殊的"分子魔术贴"——lox位点,另一个AAV除了携 带另一半基因片段、lox位点外,还携带Cre重组酶基因。这两个AAV载体进入细胞后,Cre重组酶会精 准识别"魔术贴",使拆分的两段基因精准重组,使其表达出完整的功能。另外,科研人员还开发了该技 术的2.0版本,解决了潜在的基因重排和免疫反应等生物安全问题,提升了该技术临床应用的安全性。 研究表明,该技术在多种细胞中能够高效重构大片段基因,且不产生截断蛋白,重组效率显著优于 传统方法。动物实验显示,该技术可以有效改善相关模型小鼠的行为和癫痫表型。 未来科研团队还将进一步探索该技术的全身递送效率、机制研究和疾病模型的建立,开展灵长类动 物模型的系统验证和临床前研究,推动该技术的转化落地。 记者从中国科学院深圳先进技术研究院获悉,该院科研团队历时五年,成功攻克了基因治疗领域利 用AAV(腺相关病毒载体)高效递送长基因的难题,提出了一种名为"AAVLINK"的新型基因治疗策 略,有望推动针对孤独症、癫痫等神经系统疾病和其 ...
新型基因治疗策略为孤独症、癫痫等疾病带来新希望
Xin Lang Cai Jing· 2026-01-28 09:28
转自:中国科学报 2020年,路中华团队联合姜玉武团队围绕这一痛点难题展开攻关。他们提出了一种名为"AAVLINK"的 新方法。该方法将长基因分成两段,分别装进两个AAV中,一个AAV携带的基因片段装上特殊的"分子 魔术贴"—lox位点,另一个AAV除了携带另一半基因片段、lox位点外,还携带Cre重组酶基因。两个装 载着基因的AAV载体进入细胞后,Cre重组酶会精准识别"魔术贴",使拆分的两段基因精准重组,使其 表达出完整的功能。 目前,世界范围内已发现超过7000种罕见病。这类疾病大多由基因突变引发且缺乏有效治疗手段,构成 人类医学面临的重要挑战之一。近年来,基因治疗作为一种新型治疗技术,通过修复、替换或抑制致病 基因等方法,为治疗罕见病等遗传性疾病提供了新希望。 北京时间1月27日,一项发表于《细胞》的最新研究,首次提出了一种名为"AAVLINK"的新型基因治疗 策略,研究团队成功攻克了基因治疗领域利用AAV(腺相关病毒载体)高效递送长基因的难题,有望 显著推动针对孤独症、癫痫等神经系统疾病和其他遗传病的基因治疗技术的临床应用。 该研究由中国科学院深圳先进技术研究院(简称"深圳先进院")和北京大学第一医 ...
我国新型基因治疗策略有望推动孤独症、癫痫等疾病治疗
Yang Shi Xin Wen· 2026-01-28 04:32
记者从中国科学院深圳先进技术研究院获悉,该院科研团队历时五年,成功攻克了基因治疗领域利用 AAV(腺相关病毒载体)高效递送长基因的难题,提出了一种名为"AAVLINK"的新型基因治疗策略, 有望推动针对孤独症、癫痫等神经系统疾病和其他遗传病的基因治疗技术的临床应用。相关成果1月28 日在国际学术期刊《细胞》发表。 当前,世界范围内已发现超过7000种罕见病。这类疾病大多由基因突变引发且缺乏有效治疗手段,是人 类医学面临的重要挑战之一。近年来,基因治疗作为一种新型治疗技术,通过修复、替换或抑制致病基 因等方法,为治疗罕见病等遗传性疾病提供了新希望。 (文章来源:央视新闻) 为此,研究团队创新性地提出了一种名为" AAVLINK"的新方法。该方法将长基因分成两段,分别装进 两个AAV中,一个AAV携带的基因片段装上特殊的"分子魔术贴"——lox位点,另一个AAV除了携带另 一半基因片段、lox位点外,还携带Cre重组酶基因。这两个AAV载体进入细胞后,Cre重组酶会精准识 别"魔术贴",使拆分的两段基因精准重组,使其表达出完整的功能。另外,科研人员还开发了该技术的 2.0版本,解决了潜在的基因重排和免疫反应等生物 ...
加拿大医生携幼女来沪求医,花16万元“保脾”
第一财经· 2026-01-25 11:09
花费16万元切除肿瘤保下脾脏,还是"免费"将肿瘤和脾脏同时切除?这个选择对有一定经济能力的 人来说并不难做。 近日,第一财经记者独家了解到,有加拿大华人医生夫妇携幼女来到上海瑞金医院自费接受了一台高 难度的机器人胰腺肿瘤切除手术治疗,同时保住了女儿的脾脏,目前孩子已顺利康复出院。 他们没有选择在加拿大当地接受免费治疗的原因是被医生告知"这样的手术必须切除脾脏"。但中国医 生给出了一种两全的选项,不让患者做痛苦的"选择题"。 日前,十多位接受第一财经记者采访的专家一致认为,中国医疗远不止"性价比",国内顶尖医疗技术 的发展水平已经不输全球发达国家,并且在一些先进技术应用方面的水平已经开始引领世界。发展国 际医疗业务对于国内医院、医生以及全球患者而言都是好事。 2026.01. 25 本文字数:5018,阅读时长大约8分钟 作者 | 第一财经 钱童心 16万保脾还是"免费"切脾? 在加拿大温哥华执业20年的华人Kevin医生夫妇近期带着10岁的女儿来到上海交通大学医学院附属瑞 金医院胰腺外科中心求医。在成功接受了高难度的机器人胰腺手术治疗后,目前孩子已经顺利康复出 院。 Kevin在准备飞回温哥华的前一天与第一财 ...
“浙”里新声丨温州两会观察:“万亿之城”如何再出发?
Xin Lang Cai Jing· 2026-01-20 13:41
Core Viewpoint - Wenzhou is projected to surpass a GDP of 1 trillion yuan by 2025, becoming the third city in Zhejiang province to achieve this milestone after Hangzhou and Ningbo, marking a significant completion of the "14th Five-Year Plan" [1][25]. Economic Growth - As of 2024, Wenzhou's GDP reached 971.9 billion yuan, needing less than 30 billion yuan to hit the 1 trillion yuan mark in 2025 [3][25]. - The private economy is a key driver, with 1.564 million registered private enterprises and individual businesses, accounting for 90.8% of the industrial output value of above-scale enterprises [4][26]. Industrial Development - Wenzhou plans to supply 15,200 acres of industrial land and 10,200 acres of "data-driven" land by 2025, both setting historical highs [6][28]. - The city's industrial output value increased by 10.3% year-on-year, leading the province and maintaining growth above national and provincial levels for 33 consecutive months [7][30]. Technological Innovation - High-tech industries in Wenzhou are expected to rise from 60.9% to 73% during the "14th Five-Year Plan," with a focus on the new energy sector [9][31]. - Wenzhou has established over 10 million square meters of incubator space, ranking among the top 20 cities in China for talent attraction, with industrial output exceeding 1.5 trillion yuan [9][31]. Future Development Strategy - The city aims to implement an industrial doubling plan, enhancing platforms like Wenzhou High-tech Zone and Wenzhou Economic Development Zone, targeting a manufacturing output value of 430 billion yuan [16][37]. - Wenzhou's future vision includes strengthening its role in global supply chains and enhancing trade networks, with a goal of achieving over 400 billion yuan in import and export volume [19][42]. Urban Development and Quality of Life - Wenzhou is focusing on improving living conditions and urban infrastructure, with plans to integrate 2,622 square kilometers into urban planning and attract 13,000 enterprises to its incubators [22][45]. - The city aims to enhance public services and create a family-friendly environment, with initiatives to promote education, healthcare, and community services [43][45]. Consumer Market - Wenzhou's per capita consumption level ranks among the highest in the country, with efforts to develop a regional consumption center and promote local culture and cuisine [23][46]. - The city plans to implement strategies for consumption transformation, focusing on brand aggregation and service upgrades to enhance the overall consumer experience [23][46].