基因编辑疗法
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《自然》:2025年,这七大科学故事令人振奋
Ke Ji Ri Bao· 2025-12-22 01:02
2025年,尽管全球科研事业面临资金紧缩与机构裁撤的冲击,但科学家们仍在逆境中作出令人瞩目 的成就。这些发现如同镶嵌于乌云边缘的金辉,彰显着科学向善的韧性力量。在12月18日的报道中, 《自然》网站梳理了本年度7个鼓舞人心的科学故事。 部分濒危种群恢复 强有力的保护措施让部分濒危物种的命运迎来转机。 得益于人们长期对龟卵的保护以及减少渔业误捕的努力,一度濒危的绿海龟,如今在世界自然保护 联盟红色名录中已被调整为"无危"级别。 在澳大利亚,体型似鼠的捕食性有袋动物袋鼬展现出顽强的生命力。2015—2021年间,即便在持续 干旱与食物短缺的极端条件下,其分布范围也扩大了超过4.8万平方公里。 今年9月,《联合国公海条约》已获61个国家批准,达到生效门槛。该条约旨在保护国际水域生物 多样性,将于明年1月17日生效,目标是到2030年保护至少30%的陆地和海洋生物。 可再生能源跃居主导地位 清洁能源的崛起已势不可挡。今年,可再生能源首次超越煤炭,成为全球最大电力来源。其中,中 国于5月成为首个光伏发电累计装机规模突破10亿千瓦的国家,仅上半年新增装机就达256吉瓦,占全球 过半。中国计划在下一个5年中,再增200—3 ...
梁文锋,Nature全球年度十大科学人物!
量子位· 2025-12-09 01:21
Core Points - Liang Wenfeng has been recognized as one of the top ten scientists of 2025 by the prestigious journal Nature for his significant contributions to the AI field through the DeepSeek model [1][3] - DeepSeek's model has disrupted the AI industry by achieving remarkable cost-effectiveness and enhancing the global visibility of domestic large models [9][10] - The recent release of DeepSeek's V3.2 model has set a new benchmark in the Agent evaluation, marking a significant advancement in open-source models [11][12] Group 1: Recognition and Impact - Liang Wenfeng is described as a "Tech disruptor" by Nature, highlighting his dual identity as a financial expert and a pioneer in AI [4][5] - The introduction of DeepSeek has been a game-changer for the AI sector, proving that high-performance models can be developed without excessive data or resources [10][21] - The model's cost efficiency has positioned it as a competitive player in the global AI landscape [9] Group 2: Background of Liang Wenfeng - Liang Wenfeng was born in 1985 in Guangdong and excelled academically, earning a place at Zhejiang University [14][15] - He transitioned into quantitative investment in 2008, capitalizing on the emerging trend of quantitative trading in China [17][18] - In 2021, his firm became one of the largest quantitative private equity firms in China, prompting him to explore opportunities in large models [19][20] Group 3: Other Recognized Scientists - Mengran Du, another Chinese researcher, was also recognized for her groundbreaking work in deep-sea ecology [6][22] - Du's research led to the discovery of the deepest known animal ecosystems, challenging existing models of extreme life and carbon cycling [25][26] - Her academic journey includes significant contributions to deep-sea science and technology, with multiple publications in prestigious journals [33]
基因编辑疗法连破纪录,国产器械引领全球创新 | Healthcare View
红杉汇· 2025-11-28 00:04
Group 1 - The first successful clinical treatment of hyperlipidemia using APOC3 gene editing was announced by Zhengxu Biotech, marking a significant breakthrough in gene therapy [2][3] - The CS-121 injection utilizes transformer Base Editor technology to achieve precise single-base correction without damaging the DNA double helix, simulating beneficial mutations found in nature [2] - Clinical data showed a significant decrease in fasting triglyceride levels within three days post-treatment, with no adverse events reported, indicating a promising safety profile [2] Group 2 - New芽基因 reported positive results from a 52-week follow-up of GEN6050X, the first FDA-approved gene editing therapy for Duchenne Muscular Dystrophy (DMD), showing significant improvements in cardiac and pulmonary function [6] - The therapy employs a dual mechanism to restore dystrophin expression and enhance cell stability, providing new hope for DMD patients [6] Group 3 - 精锋医疗's 精锋云® remote surgery system received NMPA approval, representing a leap in domestic surgical robotics technology [8] - The system has successfully assisted over 500 remote surgeries globally with a 100% success rate, showcasing its potential in enhancing healthcare accessibility [8] Group 4 - 凌意生物's LY-M003 injection for Wilson's disease received FDA approval to directly enter Phase II clinical trials, utilizing a novel copper ion dynamic regulation technology [11][12] - Initial clinical observations indicated good safety and positive treatment effects, with no serious adverse events reported [11] Group 5 - 瑞吉生物's freeze-dried mRNA vaccine for tuberculosis received CDE approval for clinical trials, marking a significant advancement in mRNA vaccine technology [13] - This vaccine aims to address a major public health challenge and represents a new technological pathway for tuberculosis prevention [13] Group 6 - 启函生物's QT-019B, a universal dual-target CAR-T cell product for refractory systemic lupus erythematosus, received CDE approval, highlighting its innovative design and potential applications in various autoimmune diseases [15] - The product aims to reduce the risk of graft-versus-host disease and improve clinical outcomes for patients [15] Group 7 - 心泰医疗's interventional guidewire received market approval, enhancing precision and safety in structural heart disease interventions [18] - The guidewire's innovative design allows for multiple functions, improving surgical efficiency and reducing risks [18] Group 8 - 同心医疗's BrioVAD system successfully completed its first clinical enrollment in the US, aiming to provide long-term support for patients with advanced heart failure [19][20] - The system's design minimizes surgical trauma and infection risk, enhancing patient experience and quality of life [19] Group 9 - 唯柯医疗's transcatheter mitral valve implantation project was approved for the 2025 National Key R&D Program, focusing on developing a durable and low-complication valve replacement system [23] - This project represents a significant step in addressing clinical challenges in heart valve diseases and enhancing domestic medical device capabilities [23] Group 10 - 微光医疗's project on coronary atherosclerosis diagnosis and treatment technology won the Beijing Science and Technology Progress Award, showcasing its innovative contributions to medical imaging [26] - The project has established a comprehensive evaluation system and achieved significant technological breakthroughs, enhancing the quality of care in cardiovascular diseases [26] Group 11 - 迪必尔生物工程 was recognized as a "Little Giant" enterprise, reflecting its innovation in high-performance bioreactor technology [30] - The recognition underscores the company's role in advancing the biomanufacturing industry and addressing global challenges in process scaling [30] Group 12 - 重庆壹永科技 was also recognized as a "Little Giant" enterprise, highlighting its capabilities in tumor big data and artificial intelligence [32] - This acknowledgment supports the company's ongoing innovation and market competitiveness in the healthcare sector [32] Group 13 - 红杉中国 has invested over 200 healthcare companies with distinctive technological features and high growth potential, covering various segments including innovative drugs and medical devices [33] - The investment strategy reflects a commitment to fostering innovation and supporting the growth of the healthcare industry [33]
西藏药业:前三季度营业收入近21亿元 加码创新药新赛道投资布局
Zheng Quan Shi Bao Wang· 2025-10-30 11:16
Core Viewpoint - Tibet Pharmaceutical (600211.SH) reported a steady performance in Q3 2025, with revenue of 2.089 billion yuan and net profit of 722 million yuan, while actively seeking new growth points through investments in innovative drug development [1][2]. Group 1: Financial Performance - In the first three quarters of 2025, Tibet Pharmaceutical achieved operating revenue of 2.089 billion yuan and a net profit attributable to shareholders of 722 million yuan, indicating stable overall performance [1]. - The company reported a non-recurring net profit of 668 million yuan, reflecting its solid financial foundation [1]. Group 2: Strategic Investments - The company made a significant investment of 60 million USD in Suzhou Ruizheng Gene Co., Ltd., marking a key highlight in its management strategy for entering the innovative drug sector [1]. - In August 2025, Tibet Pharmaceutical announced a 60 million USD equity investment in Accuredit Therapeutics Limited, acquiring a 40.82% stake, which aligns with its strategic focus on innovative drug development [1][2]. Group 3: Innovative Drug Pipeline - Ruizheng Gene is developing therapies targeting metabolic diseases, particularly in the liver disease area, addressing significant unmet clinical needs [2]. - The company is the first in China to enter exploratory clinical research for an LNP-based in vivo gene editing product and has received FDA clinical approval in the U.S. for its pipeline [2]. - The ART001 pipeline targets rare diseases, currently in Phase I/IIa clinical trials in China, and has received orphan drug designation from the FDA [2]. Group 4: Product Development and Market Strategy - The company reported sales of its product "New活素" reaching approximately 4.17 million units, generating revenue of 1.456 billion yuan, with a low market penetration rate despite a large annual incidence of heart failure patients in China [3]. - The company is actively promoting "New活素" sales and has initiated exploratory research to expand sales channels [3]. - Other products like "诺迪康" and "金罗汉" are performing well in the market, with expectations for gradual growth in market share [3]. Group 5: Additional Investments - In March 2025, the company completed a 300 million yuan equity investment in Jiangsu Chentai Pharmaceutical Technology Co., Ltd., acquiring a 13.04% stake [4]. - The drug "Zorifertinib" developed by Chentai is the first globally to conduct registration clinical trials for advanced non-small cell lung cancer with CNS metastasis, showing significant clinical value [4]. - The commercialization of Zorifertinib is underway, with preparations for negotiations regarding medical insurance, which could provide stable cash flow for the company [4].
基因编辑疗法又一安全危机
3 6 Ke· 2025-10-28 23:29
Core Viewpoint - The gene editing sector experienced a significant setback following a severe liver injury incident in a patient treated with Intellia Therapeutics' NEX-z therapy, leading to a 42.23% drop in the company's stock price and a nearly 50% loss in market capitalization [1][2]. Group 1: Incident Details - A patient with a rare heart disease undergoing NEX-z treatment reported severe liver damage, prompting the suspension of two Phase III clinical trials [1][3]. - This incident marks the second occurrence of Grade 4 liver toxicity related to NEX-z this year, raising concerns about the safety of gene editing therapies [1][4]. - The first incident in May was asymptomatic and self-resolving, contrasting sharply with the current case, which required hospitalization [1][3]. Group 2: Market Impact - The safety concerns surrounding NEX-z have negatively affected the entire gene editing sector, causing stock declines in other companies such as Beam Therapeutics and Prime Medicine [1][4]. - Analysts had previously viewed the risk/reward balance of NEX-z favorably, but the recent events have led to increased scrutiny and skepticism regarding its safety profile [3][4]. Group 3: Market Potential and Competition - The market for ATTR-CM, the condition treated by NEX-z, is projected to exceed $11.2 billion by 2030, indicating significant commercial potential [2]. - Other treatments for ATTR-CM, such as Alnylam's Amvuttra, have shown strong sales growth, highlighting the competitive landscape and the urgency for Intellia to address safety concerns [8][9]. Group 4: Future Considerations - Intellia is collaborating with liver disease experts and regulatory bodies to investigate the recent adverse event and explore potential risk management strategies [6][7]. - The company faces financial pressure, with cash flow projected to last until 2027, making the resolution of these safety issues critical for its future [7][8].
颠覆未来20年的科技是什么?“中国诺奖”得主们这样说
第一财经· 2025-07-10 02:01
Core Viewpoint - The article discusses the significance of the "Future Science Prize" in promoting groundbreaking scientific research in China, highlighting its role in encouraging young scientists and recognizing impactful contributions in various fields [1][10]. Group 1: Future Science Prize - The "Future Science Prize," often referred to as the "Nobel Prize of China," awards up to $1 million for significant scientific achievements in life sciences, material sciences, and mathematics and computer sciences [1]. - Since its inception, 39 scientists have received the Future Science Prize, emphasizing its growing importance in the scientific community [1]. Group 2: Biomedical Research - The integration of basic, applied, and clinical research in biomedicine is advancing, with potential therapies emerging that could revolutionize disease treatment, such as gene editing for heart disease and cancer [2]. - The scarcity of physician-scientists globally is a challenge that needs to be addressed to bridge the gap between basic research and clinical application [2]. Group 3: Energy Solutions - Controlled nuclear fusion is presented as a clean and safe solution to the current energy crisis, with advancements in laser-driven inertial confinement fusion being a key focus [3]. - The Double-cone ignition scheme (DCI) proposed by Chinese researchers aims to improve the efficiency of energy conversion in fusion processes, potentially leading to higher energy gains with lower laser energy [3]. Group 4: Room Temperature Superconductivity - Achieving room temperature superconductivity could significantly impact energy breakthroughs and support advancements in various fields, including artificial intelligence and quantum computing [4]. - The development of core materials is crucial for progress in AI, quantum computing, and energy sectors, with room temperature superconductors being a transformative material for the next two decades [4]. Group 5: Artificial Intelligence in Science - AI is influencing scientific research across various domains, with potential breakthroughs in aging mechanisms and brain health prediction through AI-driven gene analysis [6]. - The combination of AI and quantum computing is seen as a future direction for scientific development, with the potential to create new algorithms and systems [7]. Group 6: Support for Scientists - There is a growing trend in China to establish private scientific awards to encourage original research, with significant funding initiatives like Tencent's "New Cornerstone Researcher Project" providing substantial financial support to scientists [10]. - The emphasis is on supporting scientists who are ambitious, innovative, and willing to explore uncharted territories in their research [11].
13亿美元!礼来收购Verve公司,为人类头号致死原因开发基因编辑疗法
生物世界· 2025-06-18 09:08
Core Viewpoint - Eli Lilly announced the acquisition of Verve Therapeutics for up to $1.3 billion to enhance its cardiovascular disease treatment pipeline through Verve's gene editing therapies [2]. Group 1: Acquisition Details - Eli Lilly will pay an upfront payment of $1 billion, equivalent to $10.50 per share, plus up to $300 million in potential contingent value rights (CVR) based on the progress of VERVE-102 in clinical trials [12]. - The acquisition aims to leverage Verve's innovative gene editing technology to provide a one-time treatment for cardiovascular diseases, shifting the treatment paradigm from chronic care to a single intervention [9][11]. Group 2: Clinical Trial Insights - Verve's VERVE-102 therapy showed promising results in a Phase Ib clinical trial, with a single infusion leading to a dose-dependent reduction in plasma PCSK9 protein levels and LDL-C, with a 53% average reduction in LDL-C at a 0.6 mg/kg dose [2]. - The therapy targets the PCSK9 gene to lower LDL-C levels, which is crucial for preventing and treating conditions like heterozygous familial hypercholesterolemia (HeFH) [2][13]. Group 3: Company Background and Future Plans - Verve Therapeutics was founded in 2019 by renowned cardiologist Sekar Kathiresan and went public in June 2021, focusing on gene editing technologies for cardiovascular disease prevention and treatment [6]. - The company plans to initiate dosing for the first patient in the Phase II trial of VERVE-102 in the second half of the year and will provide updates on its second clinical project, VERVE-201, aimed at lowering LDL-C [9][12].
礼来超10亿美元押注基因疗法,“心脏疫苗”问世还有多远?
Di Yi Cai Jing· 2025-06-18 04:53
Core Viewpoint - Eli Lilly announced the acquisition of Verve Therapeutics for up to $1.3 billion, including nearly $1 billion upfront payment, which significantly boosted Verve's stock price by over 80% on the announcement day [1][3]. Group 1: Acquisition Details - Eli Lilly's acquisition of Verve Therapeutics aims to enhance its portfolio in the cardiovascular disease treatment sector, alongside its existing GLP-1 weight loss drug, expected to generate over $30 billion in total sales this year [3]. - Verve Therapeutics is developing a gene-editing therapy, Verve-102, targeting the PCSK9 protein to lower cholesterol levels, potentially transforming chronic cardiovascular disease treatment into a one-time therapy [1][3]. Group 2: Market Potential and Clinical Trials - Verve-102 is currently undergoing a Phase 1b clinical trial and has received Fast Track designation from the FDA, indicating its potential for significant market impact if proven effective [3]. - The gene therapy market for cardiovascular diseases may face skepticism regarding demand, as existing PCSK9-targeting drugs already meet a substantial portion of market needs [4]. Group 3: Competitive Landscape - The PCSK9 monoclonal antibodies have shown to reduce LDL cholesterol levels by over 50% compared to statins, with several products already available in the market, including a long-acting PCSK9 monoclonal antibody recently launched in China [4][5]. - Oral PCSK9 inhibitors from major pharmaceutical companies like Merck and AstraZeneca are expected to drive sales from approximately $4 billion to $12 billion by 2034, indicating a growing competitive landscape [6].
世界首例!新一代基因编辑技术——“先导编辑”完成首次人体试验,修复患者免疫细胞功能
生物世界· 2025-05-20 03:03
Core Viewpoint - Prime Editing technology, a prominent member of the CRISPR gene editing family, has made its debut in the medical field with the treatment of Chronic Granulomatous Disease (CGD) using the PM359 therapy, showing promising early clinical results [2][5][8]. Group 1: Prime Editing Technology - Prime Editing technology, developed by Professor David Liu, allows for precise gene editing without relying on DNA templates, enabling the correction of 89% of known pathogenic human genetic variations [5][7]. - The technology has been upgraded to improve editing efficiency, and it is particularly suitable for diseases like CGD, where common mutations can be corrected by inserting two missing bases in the DNA sequence [9][10]. Group 2: Clinical Trials and Results - Initial clinical data from Prime Medicine indicates that after one month of treatment with PM359, a teenage patient showed significant recovery in NADPH oxidase activity, with 66% of neutrophils fully restored, exceeding the expected clinical benefit threshold of 20% [2][8]. - The company has reported a 92% editing efficiency in correcting the most common mutation type associated with CGD in preclinical studies [7]. Group 3: Market and Economic Considerations - Prime Medicine announced a strategic restructuring, exploring external partnerships for the clinical development of PM359, highlighting the economic challenges in developing gene editing therapies for rare diseases [8][11]. - The only currently marketed gene editing therapy, Casgevy, has faced slow commercialization despite its approval, with projected sales of less than $10 million in 2024 [8]. Group 4: Future Directions - Prime Medicine plans to focus on developing gene editing therapies for hereditary liver diseases and continue supporting in vivo gene editing for cystic fibrosis, while also collaborating with Bristol-Myers Squibb on CAR-T cell therapies [11][13]. - The company aims to enhance its financial resources and accelerate innovation to ensure the widespread application of Prime Editing technology [11].