Key result
Several new and emerging lipid-lowering agents targeting LDL cholesterol, triglycerides, and Lp(a) have been developed based on human genetic evidence.
This review highlights the development of novel lipid-lowering therapies targeting specific lipoproteins, driven by insights from human genetic evidence.
Genetic insights spur targeted lipid-lowering agents; extends mechanistic understanding but leaves clinical adoption open.
Several new or emerging drugs for dyslipidemia owe their existence, in part, to human genetic evidence, such as observations in families with rare genetic disorders or in Mendelian randomization studies. Much effort has been directed to agents that reduce LDL (low-density lipoprotein) cholesterol, triglyceride, and Lp[a] (lipoprotein[a]), with some sustained programs on agents to raise HDL (high-density lipoprotein) cholesterol. Lomitapide, mipomersen, AAV8.TBG.hLDLR, inclisiran, bempedoic acid, and gemcabene primarily target LDL cholesterol. Alipogene tiparvovec, pradigastat, and volanesorsen primarily target elevated triglycerides, whereas evinacumab and IONIS-ANGPTL3-L Rx target both LDL cholesterol and triglyceride. IONIS-APO(a)-L Rx targets Lp(a).
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Hegele et al. (2019) conducted a review in Dyslipidemia. Lipid-lowering agents was evaluated. Several new and emerging lipid-lowering agents targeting LDL cholesterol, triglycerides, and Lp(a) have been developed based on human genetic evidence.
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