{"title":"A translational framework for extrahepatic oligonucleotide delivery.","authors":"Łukasz Jan Kiełpiński, Felix Franz Schumacher","doi":"10.1038/d41573-026-00125-2","DOIUrl":"https://doi.org/10.1038/d41573-026-00125-2","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148920698","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}
{"title":"Un-siloed biomedical opportunities","authors":"Asher Mullard","doi":"10.1038/d41573-026-00148-9","DOIUrl":"10.1038/d41573-026-00148-9","url":null,"abstract":"Edith Heard, CEO of the Francis Crick Institute, discusses the need to break down biomedical boundaries.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 10","pages":"750-751"},"PeriodicalIF":91.2,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148906890","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}
Sara Ancel, Emmeran Le Moal, Yu Xin Wang, Helen M Blau
{"title":"Strengthening muscle for healthy ageing: innovative treatments for sarcopenia.","authors":"Sara Ancel, Emmeran Le Moal, Yu Xin Wang, Helen M Blau","doi":"10.1038/s41573-026-01514-3","DOIUrl":"https://doi.org/10.1038/s41573-026-01514-3","url":null,"abstract":"<p><p>Millions of people worldwide suffer from sarcopenia, a clinically recognized syndrome that is defined as the age-related loss of skeletal muscle mass and strength. Sarcopenia reduces mobility, leads to falls and fractures, and increases the risk of death, posing an ever-increasing health-care and economic burden to society. There are currently no approved therapies for the syndrome, partly owing to its multifactorial aetiology, highlighting the need for therapies with pleiotropic beneficial effects on muscle function. Increased understanding of the cellular, metabolic and molecular mechanisms that drive muscle wasting with ageing is informing pharmacological interventions to increase muscle strength by promoting mitochondrial function (NAD<sup>+</sup>-related molecules, urolithin A), restoring impaired autophagy (mTORC1 inhibitors), promoting anabolic signalling (myostatin inhibitors), acting as selective androgen receptor modulators, maintaining vascularization (VEGF, apelin), reducing inflammageing (inhibitors of cytokine signalling), and restoring innervation, anabolism and regeneration (inhibitors of the gerozyme 15-PGDH). The recent surge in clinical trials combining weight loss-inducing GLP1RA therapies with drugs that preserve muscle tissue suggests that treatments that promote both healthspan and lifespan, or 'healthy ageing', are on the horizon.</p>","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148906810","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}
Yee Kwan Law, Selene Glück, Enrico Girardi, Ariel Bensimon, S Andreas Angermayr, Giulio Superti-Furga
{"title":"Solute carrier membrane transporters: emerging targets in CNS disorders.","authors":"Yee Kwan Law, Selene Glück, Enrico Girardi, Ariel Bensimon, S Andreas Angermayr, Giulio Superti-Furga","doi":"10.1038/s41573-026-01513-4","DOIUrl":"https://doi.org/10.1038/s41573-026-01513-4","url":null,"abstract":"<p><p>Prominent members of the solute carrier (SLC) superfamily of membrane transporters are established targets of central nervous system (CNS) drugs, including the blockbuster neurotransmitter reuptake inhibitors. Numerous other SLC transporters exhibit dysregulated expression in the CNS relative to other tissues, many of which have been genetically or functionally linked to neurological disorders and may therefore represent tractable therapeutic targets. As key regulators of metabolite and ion fluxes, SLCs shape developmental programmes and cellular states associated with epilepsy, neurodegeneration and autism spectrum disorders. Recent advances in assigning biochemical and cellular functions to previously uncharacterized SLCs, together with emerging chemical strategies to increase or decrease transporter abundance, are expanding the therapeutic landscape and positioning SLC biology at the forefront of next-generation CNS drug discovery.</p>","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148875443","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}
Margot Bleys, Kelly Farley, Bonnie Walton, Minsoo Khang, John Wu
{"title":"The landscape for in vivo CAR T cell therapies.","authors":"Margot Bleys, Kelly Farley, Bonnie Walton, Minsoo Khang, John Wu","doi":"10.1038/d41573-026-00140-3","DOIUrl":"https://doi.org/10.1038/d41573-026-00140-3","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":" ","pages":""},"PeriodicalIF":91.2,"publicationDate":"2026-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148851314","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}