Pub Date : 2025-10-13DOI: 10.1038/d41573-025-00164-1
Paul Ashigbie, Rajiv Shah, Thierry Diagana, Jonathan Spector
{"title":"Innovation in medicines for global health: a 20-year landscape analysis","authors":"Paul Ashigbie, \u0000 Rajiv Shah, \u0000 Thierry Diagana, \u0000 Jonathan Spector","doi":"10.1038/d41573-025-00164-1","DOIUrl":"10.1038/d41573-025-00164-1","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"24 11","pages":"818-819"},"PeriodicalIF":101.8,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/d41573-025-00164-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145283378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-10-10DOI: 10.1038/d41573-025-00168-x
Asher Mullard
Can Pfizer’s GDF15-blocking, appetite-boosting antibody ponsegromab address a cancer-induced wasting syndrome — and help patients live longer in the process? Can Pfizer’s GDF15-blocking, appetite-boosting antibody ponsegromab address a cancer-induced wasting syndrome — and help patients live longer in the process?
{"title":"Cancer cachexia contender enters first pivotal trial","authors":"Asher Mullard","doi":"10.1038/d41573-025-00168-x","DOIUrl":"10.1038/d41573-025-00168-x","url":null,"abstract":"Can Pfizer’s GDF15-blocking, appetite-boosting antibody ponsegromab address a cancer-induced wasting syndrome — and help patients live longer in the process? Can Pfizer’s GDF15-blocking, appetite-boosting antibody ponsegromab address a cancer-induced wasting syndrome — and help patients live longer in the process?","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"24 11","pages":"811-814"},"PeriodicalIF":101.8,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145261156","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}
Pub Date : 2025-10-08DOI: 10.1038/d41573-025-00167-y
Asher Mullard
{"title":"Roche buys MASH contender for up to US$3.5 billion","authors":"Asher Mullard","doi":"10.1038/d41573-025-00167-y","DOIUrl":"10.1038/d41573-025-00167-y","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"24 11","pages":"816-816"},"PeriodicalIF":101.8,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145246471","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}
Pub Date : 2025-09-30DOI: 10.1038/s41573-025-01291-5
Adrian Bot, Andrew Scharenberg, Kevin Friedman, Lin Guey, Robert Hofmeister, James I. Andorko, Michael Klichinsky, Frank Neumann, Jagesh V. Shah, Andrew J. Swayer, Kyle Trudeau, Drew Weissman, Matthias T. Stephan, Christian J. Buchholz, Carl H. June
Chimeric antigen receptor (CAR)-T cell therapy has transformed the outcomes of patients with haematological malignancies, yet its use is limited by labour-intensive manufacturing, constrained production capacity and variable clinical performance. In vivo CAR-T cell engineering, in which CAR-T cells are generated directly inside the patient’s body, seeks to overcome these challenges by eliminating the need for ex vivo cell processing and complex logistics, as well as improve clinical performance. Recent advances in virology, RNA medicines and nanotechnology have catalysed a radical overhaul of this approach, which uses targeted delivery systems such as lentiviral vectors and lipid nanoparticles to introduce CAR-encoding genetic material into endogenous T cells. Early clinical studies have shown efficient transduction, sustained CAR expression and initial signs of antitumour activity, establishing proof of concept. This Review explores the underlying technologies — including RNA delivered by lipid nanoparticles and engineered viral vectors — and discusses how they are being adapted to develop more broadly applicable, scalable, safe and effective CAR-T cell therapies. By removing the need for ex vivo manipulation and chemotherapeutic conditioning, this strategy could enable the wider application of CAR-T cell therapies not just to blood cancers but to autoimmune diseases for which ex vivo CAR-T cell therapies have shown strong promise, such as systemic lupus erythematosus. In vivo chimeric antigen receptor (CAR)-T cell engineering uses targeted delivery systems to generate CAR-T cells directly in patients, bypassing ex vivo manufacturing. This Review examines emerging viral and lipid nanoparticle platforms, early clinical proof of concept and potential applications beyond cancer.
{"title":"In vivo chimeric antigen receptor (CAR)-T cell therapy","authors":"Adrian Bot, Andrew Scharenberg, Kevin Friedman, Lin Guey, Robert Hofmeister, James I. Andorko, Michael Klichinsky, Frank Neumann, Jagesh V. Shah, Andrew J. Swayer, Kyle Trudeau, Drew Weissman, Matthias T. Stephan, Christian J. Buchholz, Carl H. June","doi":"10.1038/s41573-025-01291-5","DOIUrl":"10.1038/s41573-025-01291-5","url":null,"abstract":"Chimeric antigen receptor (CAR)-T cell therapy has transformed the outcomes of patients with haematological malignancies, yet its use is limited by labour-intensive manufacturing, constrained production capacity and variable clinical performance. In vivo CAR-T cell engineering, in which CAR-T cells are generated directly inside the patient’s body, seeks to overcome these challenges by eliminating the need for ex vivo cell processing and complex logistics, as well as improve clinical performance. Recent advances in virology, RNA medicines and nanotechnology have catalysed a radical overhaul of this approach, which uses targeted delivery systems such as lentiviral vectors and lipid nanoparticles to introduce CAR-encoding genetic material into endogenous T cells. Early clinical studies have shown efficient transduction, sustained CAR expression and initial signs of antitumour activity, establishing proof of concept. This Review explores the underlying technologies — including RNA delivered by lipid nanoparticles and engineered viral vectors — and discusses how they are being adapted to develop more broadly applicable, scalable, safe and effective CAR-T cell therapies. By removing the need for ex vivo manipulation and chemotherapeutic conditioning, this strategy could enable the wider application of CAR-T cell therapies not just to blood cancers but to autoimmune diseases for which ex vivo CAR-T cell therapies have shown strong promise, such as systemic lupus erythematosus. In vivo chimeric antigen receptor (CAR)-T cell engineering uses targeted delivery systems to generate CAR-T cells directly in patients, bypassing ex vivo manufacturing. This Review examines emerging viral and lipid nanoparticle platforms, early clinical proof of concept and potential applications beyond cancer.","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"25 2","pages":"116-137"},"PeriodicalIF":101.8,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145194819","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}
Pub Date : 2025-09-23DOI: 10.1038/d41573-025-00160-5
Sarah Crunkhorn
{"title":"Designing new antibiotics","authors":"Sarah Crunkhorn","doi":"10.1038/d41573-025-00160-5","DOIUrl":"10.1038/d41573-025-00160-5","url":null,"abstract":"","PeriodicalId":19068,"journal":{"name":"Nature Reviews. Drug Discovery","volume":"24 11","pages":"827-827"},"PeriodicalIF":101.8,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145131638","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}
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：