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Nature Reviews. Drug Discovery最新文献

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Translating base editors: a decade of progress from the bench to the clinic. 翻译碱基编辑器：从实验室到临床的十年进步。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-10-16 DOI: 10.1038/s41573-026-01548-7
Nicole M Gaudelli, Alexis C Komor
{"title":"Translating base editors: a decade of progress from the bench to the clinic.","authors":"Nicole M Gaudelli, Alexis C Komor","doi":"10.1038/s41573-026-01548-7","DOIUrl":"https://doi.org/10.1038/s41573-026-01548-7","url":null,"abstract":"<p><p>A decade has passed since the initial description of cytosine base editors in 2016. This new class of gene editors, utilizing DNA damage intermediates beyond double-stranded DNA breaks, fundamentally shifted the paradigm of gene editing from stochastic disruption to precise chemical correction. Since then, the field has rapidly matured, with the development of optimized base editors with improved editing efficiency, targeting scope, precision and specificity, as well as the subsequent generation of adenine base editors and prime editors. Base editors have not only become widely used research tools but have served as foundational platforms for biotechnology companies developing human therapies. Today, base editors are being deployed in trials for a diverse list of indications including sickle cell disease, alpha-1 antitrypsin deficiency, inborn errors of metabolism, leukaemias and cardiovascular indications. This Review critically examines the 'bench-to-bedside' trajectory of base editors, detailing their mechanistic evolution and expanding role in treating human disease.</p>","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149207551","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
One year of Commissioner's National Priority Vouchers. 一年的局长国家优先券。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-10-15 DOI: 10.1038/d41573-026-00165-8
Asher Mullard
{"title":"One year of Commissioner's National Priority Vouchers.","authors":"Asher Mullard","doi":"10.1038/d41573-026-00165-8","DOIUrl":"https://doi.org/10.1038/d41573-026-00165-8","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149195599","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The cellular landscape of druggable RNA-binding proteins. 可用药rna结合蛋白的细胞景观。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-29 DOI: 10.1038/s41573-026-01531-2
Gaia Di Timoteo, Benson M George, Omar Abdel-Wahab, Ryan A Flynn, Konstantinos Tzelepis
{"title":"The cellular landscape of druggable RNA-binding proteins.","authors":"Gaia Di Timoteo, Benson M George, Omar Abdel-Wahab, Ryan A Flynn, Konstantinos Tzelepis","doi":"10.1038/s41573-026-01531-2","DOIUrl":"https://doi.org/10.1038/s41573-026-01531-2","url":null,"abstract":"<p><p>RNA-binding proteins (RBPs) are involved in every step of RNA metabolism, from RNA processing and modification to translation and decay, and are also important for the function of non-coding RNAs. As such, RBPs are crucial in shaping cell identity and are central to the development of numerous diseases. Over the past decade, systematic efforts to explore the RNA interactome have expanded the RBP census as well as the landscape of potentially therapeutically targetable RBPs. More recently, certain RBPs have been shown to be present on the cell surface with cell-type and disease-associated specificities. These cell surface RBPs could potentially serve as biomarkers and immunotherapy targets in a variety of diseases. Here, we summarize established intracellular roles for RBPs, as well as their connections to disease with an emphasis on cancer. Moreover, we review the nascent field of cell surface RBPs and describe strategies to harness the cell surface localization of RBPs for the design and development of novel immunotherapeutic modalities.</p>","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149081694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Psychedelic protects against chemotherapy-induced nerve damage. 迷幻药可以防止化疗引起的神经损伤。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-29 DOI: 10.1038/d41573-026-00158-7
Katie Kingwell
{"title":"Psychedelic protects against chemotherapy-induced nerve damage.","authors":"Katie Kingwell","doi":"10.1038/d41573-026-00158-7","DOIUrl":"https://doi.org/10.1038/d41573-026-00158-7","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149081671","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Drug conjugate allows cell surface payload release. 药物偶联物允许细胞表面有效载荷释放。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-28 DOI: 10.1038/d41573-026-00157-8
Alex Eccleston
{"title":"Drug conjugate allows cell surface payload release.","authors":"Alex Eccleston","doi":"10.1038/d41573-026-00157-8","DOIUrl":"https://doi.org/10.1038/d41573-026-00157-8","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Two trials or not two trials? That should not be the question. 两次试验还是不两次试验？这不应该是问题所在。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-21 DOI: 10.1038/s41573-026-01537-w
Peter Arlett, Steffen Thirstrup, Francesca Day, Juan Garcia Burgos, Nacho Mbaeliachi, Paolo Foggi, Kit Roes, Emer Cooke, Bruno Sepodes
{"title":"Two trials or not two trials? That should not be the question.","authors":"Peter Arlett, Steffen Thirstrup, Francesca Day, Juan Garcia Burgos, Nacho Mbaeliachi, Paolo Foggi, Kit Roes, Emer Cooke, Bruno Sepodes","doi":"10.1038/s41573-026-01537-w","DOIUrl":"https://doi.org/10.1038/s41573-026-01537-w","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The evolving landscape of drug targets. 不断发展的药物靶点。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-21 DOI: 10.1038/s41573-026-01530-3
Liliana Halip, Sorin Avram, John P Overington, Bissan Al-Lazikani, Ramona Curpan, Suman Sirimulla, Alexei Pushechnikov, Andrea Rosario Beccari, Nikolay Savchuck, Tudor I Oprea
{"title":"The evolving landscape of drug targets.","authors":"Liliana Halip, Sorin Avram, John P Overington, Bissan Al-Lazikani, Ramona Curpan, Suman Sirimulla, Alexei Pushechnikov, Andrea Rosario Beccari, Nikolay Savchuck, Tudor I Oprea","doi":"10.1038/s41573-026-01530-3","DOIUrl":"https://doi.org/10.1038/s41573-026-01530-3","url":null,"abstract":"<p><p>Since the turn of the century, drug discovery has been transformed by advances in genetics, genomics and proteomics, as well as automation, data science and the diversification of therapeutic modalities beyond synthetic small molecules and natural product derivatives. This transformation has led to the expansion of the drug target landscape, enabling advances in the treatment of many diseases, including some that previously lacked pharmacotherapies. In this Review, we map and quantify key elements of these changes over the past 25 years, such as trends in the number and type of targets through which drugs mediate their therapeutic effects, which now include 686 biomolecules modulated by 1,702 drugs. We also discuss trends we believe will continue or change, as target druggability continues to evolve with the emergence of new therapeutic modalities.</p>","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149017376","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
First myostatin-targeted antibody approved for spinal muscular atrophy. 首个肌抑制素靶向抗体获批治疗脊髓性肌萎缩症。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-18 DOI: 10.1038/d41573-026-00152-z
Asher Mullard
{"title":"First myostatin-targeted antibody approved for spinal muscular atrophy.","authors":"Asher Mullard","doi":"10.1038/d41573-026-00152-z","DOIUrl":"https://doi.org/10.1038/d41573-026-00152-z","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149002273","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The value of speed for drug developers. 速度对药物开发人员的价值。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-18 DOI: 10.1038/d41573-026-00149-8
David B Ridley, Chenxi Xu
{"title":"The value of speed for drug developers.","authors":"David B Ridley, Chenxi Xu","doi":"10.1038/d41573-026-00149-8","DOIUrl":"10.1038/d41573-026-00149-8","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149002270","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Upcoming FDA approval decisions in Q4 2026 FDA将于2026年第四季度做出批准决定。
IF 91.2 1区 医学
Nature Reviews. Drug Discovery Pub Date : 2026-09-09 DOI: 10.1038/d41573-026-00150-1
Kaniaw Dilzer
{"title":"Upcoming FDA approval decisions in Q4 2026","authors":"Kaniaw Dilzer","doi":"10.1038/d41573-026-00150-1","DOIUrl":"10.1038/d41573-026-00150-1","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 10","pages":"749-749"},"PeriodicalIF":91.2,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920681","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","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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