The Role of Genetics in Advancing Cardiometabolic Drug Development

Purpose of Review The objective of this review is to explore the role of genetics in cardiometabolic drug development. The declining costs of sequencing and the availability of large-scale genomic data have deepened our understanding of cardiometabolic diseases, revolutionizing drug discovery and de...

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Bibliographic Details
Published in:Current atherosclerosis reports 2024-05, Vol.26 (5), p.153-162
Main Authors: Abou-Karam, Roukoz, Cheng, Fangzhou, Gady, Shoshana, Fahed, Akl C.
Format: Article
Language:English
Subjects:
Angiology
Animals
Cardiology
Cardiovascular Diseases - drug therapy
Cardiovascular Diseases - genetics
Drug Development
Drug Discovery - methods
Humans
Medicine
Medicine & Public Health
Review
Topical Collection on Cardiometabolic Disease and Treatment
Citations: Items that this one cites
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Summary:Purpose of Review The objective of this review is to explore the role of genetics in cardiometabolic drug development. The declining costs of sequencing and the availability of large-scale genomic data have deepened our understanding of cardiometabolic diseases, revolutionizing drug discovery and development methodologies. We highlight four key areas in which genetics is empowering drug development for cardiometabolic disease: (1) identifying drug candidates, (2) anticipating drug target failures, (3) silencing and editing genes, and (4) enriching clinical trials. Recent Findings Identifying novel drug targets through genetic discovery studies and the use of genetic variants as indicators of potential drug efficacy and safety have become critical components of cardiometabolic drug discovery. We highlight the successes of genetically-informed therapeutic strategies, such as PCSK9 and ANGPTL3 inhibitors in lipid lowering and the emerging role of polygenic risk scores in improving the efficiency of clinical trials. Additionally, we explore the potential of gene silencing and editing technologies, such as antisense oligonucleotides and small interfering RNA, showcasing their promise in addressing diseases refractory to conventional treatments. Summary In this review, we highlight four use cases that demonstrate the vital role of genetics in cardiometabolic drug development: (1) identifying drug candidates, (2) anticipating drug target failures, (3) silencing and editing genes, and (4) enriching clinical trials. Through these advances, genetics has paved the way to increased efficiency of drug development as well as the discovery of more personalized and effective treatments for cardiometabolic disease.
ISSN:1523-3804
1534-6242
1534-6242
DOI:10.1007/s11883-024-01195-6