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The rise of China’s pharmaceutical industry from 2015–2024: a decade of innovation 2015-2024年中国医药产业崛起：创新的十年
Nature Reviews Drug Discovery Pub Date : 2025-07-21 DOI: 10.1038/d41573-025-00102-1
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引用次数: 0
The non-small-cell lung cancer drug market 非小细胞肺癌药物市场
Nature Reviews Drug Discovery Pub Date : 2025-07-18 DOI: 10.1038/d41573-025-00123-w
{"title":"The non-small-cell lung cancer drug market","authors":"","doi":"10.1038/d41573-025-00123-w","DOIUrl":"https://doi.org/10.1038/d41573-025-00123-w","url":null,"abstract":"Discover the world’s best science and medicine | Nature.com","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"37 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144652662","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advances in the treatment of systemic lupus erythematosus 系统性红斑狼疮的治疗进展
Nature Reviews Drug Discovery Pub Date : 2025-07-17 DOI: 10.1038/s41573-025-01242-0
Marc Scherlinger, Antonios G. A. Kolios, Vasileios C. Kyttaris, George C. Tsokos
{"title":"Advances in the treatment of systemic lupus erythematosus","authors":"Marc Scherlinger, Antonios G. A. Kolios, Vasileios C. Kyttaris, George C. Tsokos","doi":"10.1038/s41573-025-01242-0","DOIUrl":"https://doi.org/10.1038/s41573-025-01242-0","url":null,"abstract":"<p>Systemic lupus erythematous is a clinically and pathogenetically heterogeneous disease that has long challenged researchers and clinicians aiming to improve its treatment. Advances over the past 75 years have revealed a number of key immune mechanisms that drive clinical manifestations, paving the way for the development of therapies that go beyond broad immunosuppression to improve clinical efficacy and reduce side effects. These include approaches aimed at specific immune pathways, and emerging efforts to restore immune homeostasis, such as chimeric antigen receptor T cell therapies to eliminate pathogenic B cells, low-dose interleukin 2 or regulatory T cell therapies. Although hopes for durable remissions or cure are rising, major obstacles remain owing to the complex nature of the disease. In this Review, we discuss emerging therapeutic strategies designed to address these challenges.</p>","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"84 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144652618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Biased adrenergic receptor agonist tackles metabolic disease 偏肾上腺素能受体激动剂治疗代谢性疾病
Nature Reviews Drug Discovery Pub Date : 2025-07-17 DOI: 10.1038/d41573-025-00125-8
{"title":"Biased adrenergic receptor agonist tackles metabolic disease","authors":"","doi":"10.1038/d41573-025-00125-8","DOIUrl":"https://doi.org/10.1038/d41573-025-00125-8","url":null,"abstract":"Discover the world’s best science and medicine | Nature.com","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"13 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645580","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Building on the momentum of N-of-1 base editor therapies for rare diseases 以N-of-1碱基编辑器治疗罕见疾病的势头为基础
Nature Reviews Drug Discovery Pub Date : 2025-07-15 DOI: 10.1038/d41573-025-00119-6
{"title":"Building on the momentum of N-of-1 base editor therapies for rare diseases","authors":"","doi":"10.1038/d41573-025-00119-6","DOIUrl":"https://doi.org/10.1038/d41573-025-00119-6","url":null,"abstract":"Rebecca Ahrens-Nicklas, a physician-scientist at the Children’s Hospital of Philadelphia, discusses her groundbreaking application of a gene-editing therapy for an ultra-rare urea cycle disorder.","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"34 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
FDA new drug approvals in Q2 2025 2025年第二季度FDA新药批准
Nature Reviews Drug Discovery Pub Date : 2025-07-15 DOI: 10.1038/d41573-025-00124-9
{"title":"FDA new drug approvals in Q2 2025","authors":"","doi":"10.1038/d41573-025-00124-9","DOIUrl":"https://doi.org/10.1038/d41573-025-00124-9","url":null,"abstract":"Discover the world’s best science and medicine | Nature.com","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"88 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CRISPR-based therapeutic genome editing for inherited blood disorders 基于crispr的遗传性血液疾病治疗性基因组编辑
Nature Reviews Drug Discovery Pub Date : 2025-07-14 DOI: 10.1038/s41573-025-01236-y
Sébastien Levesque, Daniel E. Bauer
{"title":"CRISPR-based therapeutic genome editing for inherited blood disorders","authors":"Sébastien Levesque, Daniel E. Bauer","doi":"10.1038/s41573-025-01236-y","DOIUrl":"https://doi.org/10.1038/s41573-025-01236-y","url":null,"abstract":"<p>Therapeutic genome editing promises to transform medicine. Pivotal discoveries have provided a diverse and versatile set of tools to correct pathogenic mutations or produce protective alleles using CRISPR-based technologies. These innovative therapies are especially adaptable for blood and immune disorders, where clinical methods allow haematopoietic stem cells (HSCs) to be mobilized, harvested, engineered ex vivo and transplanted back into a patient to permanently replace their blood system. This paradigm has been exemplified with the first US Food and Drug Administration (FDA)-approved CRISPR–Cas9 therapy for sickle cell disease and β-thalassaemia, exa-cel (Casgevy). Although promising, efficient delivery of gene edits involves complicated ex vivo manipulation and toxic myeloablative conditioning. The quiescent and elusive nature of HSCs also brings associated challenges. In this Review, we explore the state-of-the-art genome editing technologies of nucleases, base editors and prime editors, which hold promise to address unmet clinical needs for patients with inherited haematological disorders. We highlight the progress made for several disorders and discuss the challenges that remain for ex vivo and in vivo targeting of HSCs for next-generation gene therapies.</p>","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"14 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
RNA chemistry and therapeutics RNA化学和治疗学
Nature Reviews Drug Discovery Pub Date : 2025-07-14 DOI: 10.1038/s41573-025-01237-x
Siyu Wang, Drew Weissman, Yizhou Dong
{"title":"RNA chemistry and therapeutics","authors":"Siyu Wang, Drew Weissman, Yizhou Dong","doi":"10.1038/s41573-025-01237-x","DOIUrl":"https://doi.org/10.1038/s41573-025-01237-x","url":null,"abstract":"<p>RNA-based therapeutics have made substantial clinical advances, primarily due to the unique chemical and biological profiles of RNA molecules. As evidenced by the approval of various RNA drugs, some initial challenges related to RNA-based therapeutics, including issues associated with large-scale production, effective delivery and immunogenicity properties, are now being addressed. Extensive efforts have focused on chemically modifying RNA molecules to enhance their stability, increase protein production, extend circulation time and improve target specificity. Three RNA categories — small RNA, translatable RNA and CRISPR guide RNA — are now being extensively developed for therapeutic applications. This Review summarizes the synthetic methods applied to these three RNA categories, describes key chemical modification strategies being used to enhance their properties and highlights current therapeutic applications and future opportunities.</p>","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"51 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Unmet needs in vaccine development 疫苗开发方面未满足的需求
Nature Reviews Drug Discovery Pub Date : 2025-07-10 DOI: 10.1038/d41573-025-00100-3
{"title":"Unmet needs in vaccine development","authors":"","doi":"10.1038/d41573-025-00100-3","DOIUrl":"https://doi.org/10.1038/d41573-025-00100-3","url":null,"abstract":"Discover the world’s best science and medicine | Nature.com","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"4 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144593866","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Lilly buys Verve and its in vivo gene editors for up to US$1.3 billion 礼来以高达13亿美元的价格收购了Verve及其体内基因编辑器
Nature Reviews Drug Discovery Pub Date : 2025-07-09 DOI: 10.1038/d41573-025-00120-z
{"title":"Lilly buys Verve and its in vivo gene editors for up to US$1.3 billion","authors":"","doi":"10.1038/d41573-025-00120-z","DOIUrl":"https://doi.org/10.1038/d41573-025-00120-z","url":null,"abstract":"Discover the world’s best science and medicine | Nature.com","PeriodicalId":18847,"journal":{"name":"Nature Reviews Drug Discovery","volume":"3 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144587018","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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