基因编辑疗法

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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].