精准医学
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【新华社】《科学》杂志发表我国科研新成果 为精准医学提供新工具
Xin Hua She· 2026-01-04 01:23
我国科学家成功构建了能够在单细胞分辨率下,同步实现细胞膜表面标志物发现与靶向核酸适体探 针获取的一体化平台——SPARK-seq。该技术为分子识别、原创靶点发现及精准医学研究提供了全新工 具,有望为众多因靶点不明而缺乏有效疗法的疾病开辟新的治疗途径。 由中国科学院杭州医学研究所谭蔚泓院士与吴芩研究员团队合作取得的这项研究成果,2026年1月1 日在国际学术期刊《科学》上线。 细胞膜蛋白是药物作用的关键靶点,而核酸适体是一类能够高特异性、高亲和力结合靶标分子的寡 核苷酸。然而,传统的核酸适体筛选方法效率低、过程繁杂,且难以在生理相关环境下系统发现全新的 疾病标志物。 2026 年 1 月 1 日,论文在《科学》期刊上线。(资料图) 研究团队在实验室讨论。(资料图) SPARK-seq平台的诞生解决了这一挑战。相较于传统方法,其筛选效率提升达百倍以上,并能高精 度锁定潜在的癌症标志物与治疗靶点。"我们研发SPARK-seq的初衷,是为了攻克像三阴性乳腺癌这类 缺乏明确治疗靶点的临床难题。"吴芩表示,"该平台在单细胞层面部署的'分子雷达',能够大规模、并 行地精准识别细胞表面与疾病相关的靶标,并同步获取能特异性结合 ...
我国科学家构建高通量核酸适体筛选与动力学分析平台
Xin Lang Cai Jing· 2026-01-03 16:08
中国青年报客户端讯(中青报·中青网记者 张渺)近日,中国科学院杭州医学研究所谭蔚泓院士与吴芩 研究员团队合作取得重要突破,成功构建了全球首个能够在单细胞分辨率下,同步实现细胞膜表面标志 物发现与靶向核酸适体探针获取的一体化平台——SPARK-seq。该技术为分子识别、原创靶点发现及精 准医学研究提供了全新的强大工具,有望为众多因靶点不明而缺乏有效疗法的疾病开辟全新的治疗途 径。相关成果于1月1日在线发表于国际学术期刊《科学》。 来源:中国青年报客户端 据介绍,SPARK-seq平台的工作原理清晰高效,相较于传统方法,其筛选效率提升达百倍以上,并能以 前所未有的精度锁定潜在的癌症标志物与治疗靶点。通过自主研发的新算法和大数据分析,SPARK-seq 可以将细胞膜表面标志物的发现从"间接推测"转变为可"直观解读"的数字信号。 未来,该技术有望推动一种"按图索骥"式的精准医疗新模式形成,快速为不同疾病匹配或定制治疗分 子,提升药物研发的效率和治疗方案的精准度。 ...
《科学》杂志发表我国科研新成果 为精准医学提供新工具
Xin Hua She· 2026-01-02 09:07
研究团队在实验室讨论。(资料图) SPARK-seq作为自主可控的原创性平台,为"无药可靶"疾病的靶点研究与治疗开发提供了全新范式,使 得科研与临床工作者能够基于我国独特的临床资源,系统性地发现具有自主知识产权的新靶点与新工 具。未来,该技术有望推动形成一种"按图索骥"式的精准医疗新模式,快速为不同疾病匹配或定制治疗 分子,提升药物研发的效率和治疗方案的精准度。 细胞膜蛋白是药物作用的关键靶点,而核酸适体是一类能够高特异性、高亲和力结合靶标分子的寡核苷 酸。然而,传统的核酸适体筛选方法效率低、过程繁杂,且难以在生理相关环境下系统发现全新的疾病 标志物。 SPARK-seq平台的诞生解决了这一挑战。相较于传统方法,其筛选效率提升达百倍以上,并能高精度锁 定潜在的癌症标志物与治疗靶点。"我们研发SPARK-seq的初衷,是为了攻克像三阴性乳腺癌这类缺乏 明确治疗靶点的临床难题。"吴芩表示,"该平台在单细胞层面部署的'分子雷达',能够大规模、并行地 精准识别细胞表面与疾病相关的靶标,并同步获取能特异性结合它的核酸适体探针。" 我国科学家成功构建了能够在单细胞分辨率下,同步实现细胞膜表面标志物发现与靶向核酸适体探针获 ...
上海专家团队自研试剂,为胰腺癌精准医疗贡献“中国方案”
Xin Lang Cai Jing· 2025-12-31 14:51
Core Viewpoint - The "Human Pancreatic Cancer 6 Gene Mutation Detection Kit," developed by Professor Yu Xianjun's team at Fudan University Affiliated Tumor Hospital, has been officially registered with the Shanghai Municipal Drug Administration, marking it as the first self-developed in vitro diagnostic reagent for pancreatic cancer in China and the world [1][3]. Group 1: Product Development and Approval - The detection kit is the first of its kind to be approved for use by a medical institution in China, contributing to global precision medicine for pancreatic cancer [1][3]. - The kit addresses the urgent need for effective gene testing methods in clinical settings, particularly for pancreatic cancer patients who often miss surgical opportunities due to late diagnosis [3][4]. Group 2: Clinical Significance and Technology - Pancreatic cancer is known for its high malignancy and rapid progression, with most patients diagnosed at advanced stages, leading to low five-year survival rates [3]. - Traditional gene testing methods are limited in scope and sensitivity, often missing critical mutations, which can hinder personalized treatment plans [3][4]. - The new kit integrates next-generation sequencing technology and ultra-deep capture techniques, significantly improving mutation detection rates and providing a comprehensive view of gene variations for personalized treatment [6]. Group 3: Research and Development Team - A cross-disciplinary research team was formed to focus on the molecular characteristics of pancreatic cancer in the Chinese population, led by Professor Hu Xin from the Precision Tumor Center at Fudan University Affiliated Tumor Hospital [4]. - The team has spent three years overcoming key technical challenges to achieve breakthroughs in precision and efficiency in mutation detection [6].
万众一芯精准医学产业智造中心签约
Su Zhou Ri Bao· 2025-12-23 22:19
万众一芯生物科技有限公司由张家港市本土孵化,是一家专注于生物芯片技术研发与应用,为精准 医学领域提供创新解决方案的高技术企业。公司具备分子诊断试剂、分子医疗器械、微纳生物芯片/微 流控芯片生产制造能力,赋能肿瘤、慢性病等多组学精准诊疗场景,其开发的"基因电子芯片"及相关技 术路线属国际首创。此次签约的精准医学产业智造中心项目总投资5亿元,集总部大脑、智能制造、产 业生态于一体,将建设芯片、仪器、试剂三条核心产线,实现核心技术的自主可控与规模化输出。其 中,研发中试与总部运营中心将承载芯片制造、仪器装配、试剂生产等功能。 铸强精准医学"中国芯"。昨天(12月23日),张家港万众一芯精准医学产业智造中心项目正式签 约。苏州市委副书记、市长吴庆文出席活动。 张家港万众一芯生物科技有限公司董事长胡文闯;苏州市政府秘书长徐本,张家港市主要负责同志 等参加活动。 活动中,万众一芯关键产品及技术成果发布,战略投资协议签约。 ...
卵巢癌FZOCUS-1研究登顶全球肿瘤学顶级期刊
Huan Qiu Wang· 2025-12-15 03:38
Core Viewpoint - The FZOCUS-1 study has been published in the prestigious journal "CA: A Cancer Journal for Clinicians," marking a significant milestone for Chinese clinical oncology research and demonstrating China's leadership in gynecological oncology research on a global scale [1][6]. Group 1: Study Overview - The FZOCUS-1 study is a randomized, double-blind, placebo-controlled Phase III clinical trial conducted over six years across 54 centers in China, involving 674 patients [4]. - The study aims to evaluate the efficacy and safety of the first domestically developed PARP inhibitor, Fluorouracil, either alone or in combination with the anti-angiogenic drug Apatinib, for first-line maintenance treatment of advanced ovarian cancer [4]. Group 2: Clinical Context - Ovarian cancer, known as the "king of gynecological cancers," is characterized by late-stage diagnosis, high recurrence rates, and poor long-term prognosis, with less than 30% five-year survival rate for advanced cases [3]. - Traditional treatment methods have limited effectiveness, with up to 70% of patients facing recurrence within two years, leading to a cycle of treatment and relapse [3]. Group 3: Implications and Accessibility - The successful publication of the FZOCUS-1 study provides critical evidence for clinical practice in advanced ovarian cancer, contributing both breakthrough advancements and precision treatment options [4][6]. - The PARP inhibitor Fluorouracil has been included in the medical insurance directory, significantly improving drug accessibility for Chinese ovarian cancer patients [6].
Lexeo Therapeutics (NasdaqGM:LXEO) Update / Briefing Transcript
2025-12-09 21:02
Lexeo Therapeutics Conference Call Summary Company Overview - **Company**: Lexeo Therapeutics (NasdaqGM:LXEO) - **Focus**: Development of gene therapies for genetically mediated cardiovascular diseases, specifically targeting Arrhythmogenic Cardiomyopathy (ACM) Key Points Discussed Industry and Disease Context - **Disease**: Arrhythmogenic Cardiomyopathy (ACM), particularly related to mutations in the PKP2 gene - **Prevalence**: Approximately 125,000 individuals in the U.S. are diagnosed with ACM, with about 20% presenting due to sudden cardiac arrest [9][10] - **Genetic Basis**: Mutations in the PKP2 gene account for over 50% of ARVC ACM cases, leading to significant electrical and structural abnormalities in the heart [10][12] Clinical Insights and Management - **Holistic Approach**: Emphasis on a comprehensive management strategy for ACM, integrating genetic testing and precision medicine [4][8] - **Diagnostic Criteria**: Utilization of Task Force criteria for diagnosing ACM, which includes both structural and electrical evaluations [14][17] - **Management Strategies**: - Initial treatment often involves beta-blockers for arrhythmias [28] - Implantable cardioverter-defibrillators (ICDs) are considered for high-risk patients [29] - Antiarrhythmic medications like flecainide and sotalol may be used as the disease progresses [30] - Ablation procedures are an option if medical management fails, though they do not address the underlying disease progression [32][33] Gene Therapy Potential - **Gene Therapy Candidate**: LX2020, currently in clinical trials for treating PKP2-associated ACM [4][42] - **Patient Selection**: Ideal candidates for gene therapy are those with early signs of disease, particularly those experiencing symptomatic arrhythmias [36][70] - **Expected Outcomes**: Aiming to reduce arrhythmias and prevent progression to heart failure, with a focus on improving electrical stability in the heart [39][40] Future Directions - **Clinical Trials**: Anticipation of data updates on LX2020 in early 2026, with a focus on clinical endpoints and pathological studies [78] - **Adoption of Gene Therapy**: High expected uptake among patients who have experienced severe arrhythmias, with estimates suggesting 70-90% readiness for gene therapy [71][72] Additional Considerations - **Family Screening**: Importance of genetic testing for family members of diagnosed patients to identify at-risk individuals [20][62] - **Disease Progression**: Variability in disease progression rates among patients, with those experiencing sudden cardiac arrest likely to progress more rapidly [86][87] Conclusion - Lexeo Therapeutics is at the forefront of developing innovative gene therapies for ACM, with a strong emphasis on precision medicine and holistic patient care. The upcoming clinical data on LX2020 is highly anticipated and could significantly impact treatment paradigms for ACM patients.
迪安诊断西南数字化精准中心签约落户重庆市两江新区
Sou Hu Cai Jing· 2025-12-03 15:09
Core Insights - The establishment of Chongqing Liangjiang Xingkang Medical Equipment Co., Ltd. and the Di'an Southwest Digital Precision Center marks a significant development in the precision medicine sector in Chongqing [1][3] - The collaboration between Di'an Diagnostics and Liangjiang Xinshi Company aims to enhance diagnostic capabilities and expand services in the region [3] Group 1: Company Overview - Liangjiang Xingkang Company is a joint venture established by Di'an Diagnostics from Zhejiang and Liangjiang Xinshi Company [3] - Di'an Diagnostics is recognized as one of the early players in precision diagnostics in China, focusing on molecular diagnostics, pathology diagnostics, and mass spectrometry diagnostics [3] Group 2: Strategic Initiatives - The company plans to upgrade and build a digital regional medical testing center, relying on public hospitals in the area [3] - It will create a centralized procurement platform for medical reagents, consumables, and equipment, while also offering quality system construction, business training, technical support, and talent and research support [3] Group 3: Impact on the Industry - The Di'an Southwest Digital Precision Center aims to serve as a headquarters for precision medicine in the western region, integrating high-quality resources from industry, academia, and research [3] - The initiative will enhance diagnostic technology levels and promote the application of precision medicine in clinical settings, enriching the ecosystem of the biomedicine and medical device industries in Liangjiang New Area [3]
IDEAYA Biosciences(IDYA) - 2025 FY - Earnings Call Transcript
2025-12-02 15:02
Financial Data and Key Metrics Changes - The company is approaching top line results for its lead program, darovasertib, with guidance for an accelerated approval filing in the U.S. by year-end this year to Q1 next year [3][4] - Historical median progression-free survival (PFS) for metastatic uveal melanoma is about 2 to 3 months, while the company has reported a PFS of approximately 7 months in their studies, indicating a significant improvement [6][7] Business Line Data and Key Metrics Changes - The company has eight programs in clinical development, with darovasertib being the lead program in a registrational trial for metastatic uveal melanoma [3] - The company has received breakthrough therapy designation for darovasertib in the neoadjuvant setting, indicating a strong focus on this area [4] - The next key program is DLL3, with recent data presented at the World Conference on Lung Cancer, highlighting its potential in small cell lung cancer [4][55] Market Data and Key Metrics Changes - The market for darovasertib targets approximately 4,000-5,000 patients, with a significant portion being HLA negative, which the company aims to address [9] - The annual incidence of neoadjuvant uveal melanoma is estimated to be 10,000-12,000 patients, indicating a substantial unmet need in this market [12] Company Strategy and Development Direction - The company is focusing on a diversified pipeline, with plans to initiate an adjuvant study in collaboration with Servier in the first half of next year, targeting high-risk metastatic patients [28] - The company aims to implement rational combinations in the MTAP deletion space, positioning itself as a leader in this area with multiple clinical stage assets [37][38] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming data readouts, particularly for eye preservation rates and event-free survival in the neoadjuvant setting [19][13] - The company believes it is well-positioned to address the unmet needs in the neoadjuvant space due to limited competition [15][17] Other Important Information - The company is developing a bispecific ADC targeting B7H3 and PTK7, which is unique in its AND format, aiming to enhance efficacy in dual expression populations [68][69] - The company is also advancing its KAT6/7 inhibitor program, which targets chromatin remodeling, with a focus on breast cancer and potentially other indications [76][77] Q&A Session Summary Question: What is the timeline for the neoadjuvant study? - The company plans to enroll the study in roughly five quarters, with the first eye preservation data expected in about six months [19][20] Question: How does the company plan to differentiate its DLL3 program? - The company believes its linker system allows for higher dosing and reduced toxicity compared to competitors, which could lead to better efficacy and durability [55][56] Question: What is the market potential for the adjuvant study? - The adjuvant market could potentially be the largest of the three indications, with a path to becoming a blockbuster opportunity [30]
儿科用药仍存“掰药猜量”难题,真实世界研究为合理用药提供依据
Xin Jing Bao· 2025-12-02 09:56
Group 1 - The current pediatric clinical medication still relies on guesswork for dosage and administration, highlighting the need for evidence-based practices in children's medication [1] - Pediatricians should manage health from fertilization through to the age of 18-20, emphasizing a comprehensive approach to child development and health [1] - The transition from "empirical medicine" to "precision medicine" in treating conditions like growth disorders is crucial, ensuring treatment is based on guidelines and individual patient needs [1] Group 2 - China has made significant advancements in innovative drug treatments, particularly in cell therapy, gene therapy, and traditional biological treatments, with real-world clinical data derived from local patient practices [2] - The establishment of databases is essential for supporting safe and rational medication use, as long-term clinical data accumulation is necessary to ensure drug safety and efficacy [2] - The "Young and Middle-aged Physician Growth Plan - Starry Sky Science Popularization Project" was launched to enhance pediatric medical talent development and promote awareness of children's medication [2]