Next-Gen Therapeutics: Pioneering Drug Discovery with iPSCs, Genomics, AI, and Clinical Trials in a Dish

被引：2

作者：

Yildirim, Zehra ^{[1
,2
]}

Swanson, Kyle ^{[3
,4
]}

Wu, Xuekun ^{[1
,2
]}

Zou, James ^{[3
]}

Wu, Joseph ^{[1
,2
]}

机构：

[1] Stanford Univ, Sch Med, Stanford Cardiovasc Inst, Stanford, CA 94305 USA

[2] Stanford Univ, Sch Med, Div Cardiovasc Med, Stanford, CA 94305 USA

[3] Stanford Univ, Dept Comp Sci, Stanford, CA USA

[4] Greenstone Biosci, Palo Alto, CA USA

来源：

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY | 2025年 / 65卷

基金：

美国国家卫生研究院;

关键词：

drug discovery; cardiotoxicity; iPSC; clinical trials in a dish; PLURIPOTENT STEM-CELLS; HUMAN CARDIAC ORGANOIDS; SMOOTH-MUSCLE-CELLS; HEART-FAILURE; FUNCTIONAL MATURATION; REVEALS; DIFFERENTIATION; GENERATION; PLATFORM; ADULT;

D O I：

10.1146/annurev-pharmtox-022724-095035

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

In the high-stakes arena of drug discovery, the journey from bench to bedside is hindered by a daunting 92% failure rate, primarily due to unpredicted toxicities and inadequate therapeutic efficacy in clinical trials. The FDA Modernization Act 2.0 heralds a transformative approach, advocating for the integration of alternative methods to conventional animal testing, including cell-based assays that employ human induced pluripotent stem cell (iPSC)derived organoids, and organ-on-a-chip technologies, in conjunction with the innovative capacity of iPSC-derived clinical trial in a dish models designed for cardiovascular disease research. We also highlight how integrating iPSC technology with AI can accelerate the identification of viable therapeutic candidates, streamline drug screening, and pave the way toward more personalized medicine. Through this, we provide a comprehensive overview of the current landscape and future implications of iPSC and AI applications being navigated by the research community and pharmaceutical industry.

引用

页码：71 / 90

页数：20