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FDA new drug approvals in Q3 2024 美国食品和药物管理局 2024 年第三季度批准的新药
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-10 DOI: 10.1038/d41573-024-00166-5
Paul Verdin
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引用次数: 0
Reply to ‘Bayesian approaches in drug development: continuing the virtuous cycle’ 对 "药物开发中的贝叶斯方法:继续良性循环 "的答复
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-10 DOI: 10.1038/s41573-024-01054-8
Stephen J. Ruberg
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引用次数: 0
Bayesian approaches in drug development: continuing the virtuous cycle 药物开发中的贝叶斯方法:继续良性循环
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-10 DOI: 10.1038/s41573-024-01052-w
Wilmar Igl, John Constant
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引用次数: 0
Tumour cells get a dendritic cell makeover 树突状细胞改造肿瘤细胞
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-08 DOI: 10.1038/d41573-024-00168-3
M. Teresa Villanueva
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引用次数: 0
mRNA vaccines for infectious diseases — advances, challenges and opportunities 用于传染病的 mRNA 疫苗--进展、挑战和机遇
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-04 DOI: 10.1038/s41573-024-01042-y
Norbert Pardi, Florian Krammer
The concept of mRNA-based vaccines emerged more than three decades ago. Groundbreaking discoveries and technological advancements over the past 20 years have resolved the major roadblocks that initially delayed application of this new vaccine modality. The rapid development of nucleoside-modified COVID-19 mRNA vaccines demonstrated that this immunization platform is easy to develop, has an acceptable safety profile and can be produced at a large scale. The flexibility and ease of antigen design have enabled mRNA vaccines to enter development for a wide range of viruses as well as for various bacteria and parasites. However, gaps in our knowledge limit the development of next-generation mRNA vaccines with increased potency and safety. A deeper understanding of the mechanisms of action of mRNA vaccines, application of novel technologies enabling rational antigen design, and innovative vaccine delivery strategies and vaccination regimens will likely yield potent novel vaccines against a wide range of pathogens. Following the success of the COVID-19 vaccines, mRNA vaccines have now entered development for a wide range of infectious diseases. This Review discusses mRNA vaccine design considerations, delivery strategies and mechanisms of action, assessing mRNA vaccines currently in development for various viruses, bacteria and parasites. The challenges, limitations and opportunities facing next-generation mRNA vaccines are considered.
基于 mRNA 的疫苗概念出现于三十多年前。过去 20 年中的突破性发现和技术进步解决了最初阻碍这种新型疫苗应用的主要障碍。核苷修饰的 COVID-19 mRNA 疫苗的快速开发表明,这种免疫平台易于开发,具有可接受的安全性,并且可以大规模生产。抗原设计的灵活性和简易性使 mRNA 疫苗能够用于多种病毒以及各种细菌和寄生虫的开发。然而,我们的知识空白限制了效力和安全性更高的下一代 mRNA 疫苗的开发。深入了解 mRNA 疫苗的作用机制,应用新技术合理设计抗原,以及创新的疫苗递送策略和接种方案,将有可能开发出针对多种病原体的强效新型疫苗。
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引用次数: 0
A viral–lipid hybrid delivery system for genetic therapies 用于基因疗法的病毒-脂质混合输送系统
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-03 DOI: 10.1038/d41573-024-00165-6
Katie Kingwell
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引用次数: 0
Two decades of new drug approvals in Japan 二十年来日本新药审批情况
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-02 DOI: 10.1038/d41573-024-00153-w
Ryosuke Kuribayashi,  Aya Hariu,  Yasuhiro Kishioka,  Akira Sakurai
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引用次数: 0
FDA approves first monoclonal antibody for COPD 美国食品和药物管理局批准首个用于治疗慢性阻塞性肺病的单克隆抗体
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-10-01 DOI: 10.1038/d41573-024-00164-7
Asher Mullard
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引用次数: 0
Therapeutic targeting of senescent cells in the CNS 中枢神经系统衰老细胞的治疗靶点
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-09-30 DOI: 10.1038/s41573-024-01033-z
Markus Riessland, Methodios Ximerakis, Andrew A. Jarjour, Bin Zhang, Miranda E. Orr
Senescent cells accumulate throughout the body with advanced age, diseases and chronic conditions. They negatively impact health and function of multiple systems, including the central nervous system (CNS). Therapies that target senescent cells, broadly referred to as senotherapeutics, recently emerged as potentially important treatment strategies for the CNS. Promising therapeutic approaches involve clearing senescent cells by disarming their pro-survival pathways with ‘senolytics’; or dampening their toxic senescence-associated secretory phenotype (SASP) using ‘senomorphics’. Following the pioneering discovery of first-generation senolytics dasatinib and quercetin, dozens of additional therapies have been identified, and several promising targets are under investigation. Although potentially transformative, senotherapies are still in early stages and require thorough testing to ensure reliable target engagement, specificity, safety and efficacy. The limited brain penetrance and potential toxic side effects of CNS-acting senotherapeutics pose challenges for drug development and translation to the clinic. This Review assesses the potential impact of senotherapeutics for neurological conditions by summarizing preclinical evidence, innovative methods for target and biomarker identification, academic and industry drug development pipelines and progress in clinical trials. With ageing global populations, cellular senescence is gaining increasing attention as a therapeutic target. In their Review, Orr and colleagues discuss ongoing research into senescence in the central nervous system, including promising targets and drugs in development that aim to clear senescent cells or modulate their senescence-associated secretory phenotype.
随着年龄的增长、疾病和慢性病的发生,衰老细胞在人体内不断积累。它们对包括中枢神经系统(CNS)在内的多个系统的健康和功能产生负面影响。针对衰老细胞的疗法(广义上称为衰老疗法)最近成为中枢神经系统潜在的重要治疗策略。前景看好的治疗方法包括使用 "衰老分解剂 "解除衰老细胞的促生存途径,从而清除衰老细胞;或使用 "衰老形态学药物 "抑制衰老相关分泌表型(SASP)的毒性。在率先发现第一代衰老分解剂达沙替尼和槲皮素之后,又发现了数十种其他疗法,还有几个前景看好的靶点正在研究中。尽管衰老疗法可能带来变革,但目前仍处于早期阶段,需要进行全面测试,以确保可靠的靶点参与、特异性、安全性和有效性。中枢神经系统作用的衰老治疗药物对大脑的穿透力有限,并有潜在的毒副作用,这给药物开发和临床转化带来了挑战。本综述通过总结临床前证据、靶点和生物标记物鉴定的创新方法、学术界和业界的药物开发管线以及临床试验进展,评估了老年疗法对神经系统疾病的潜在影响。
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引用次数: 0
Targeting undruggable GTPases 靶向不可药用 GTPases
IF 122.7 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Pub Date : 2024-09-27 DOI: 10.1038/d41573-024-00158-5
Sarah Crunkhorn
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引用次数: 0
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