{"id":306977,"date":"2023-11-16T11:42:10","date_gmt":"2023-11-16T16:42:10","guid":{"rendered":"https:\/\/platohealth.ai\/first-human-clinical-trial-using-crispr-to-lower-cholesterol-levels\/"},"modified":"2023-11-17T05:54:07","modified_gmt":"2023-11-17T10:54:07","slug":"first-human-clinical-trial-using-crispr-to-lower-cholesterol-levels","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/first-human-clinical-trial-using-crispr-to-lower-cholesterol-levels\/","title":{"rendered":"First Human Clinical Trial Using CRISPR to Lower Cholesterol Levels","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"

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This therapeutic approach involves injecting patients with VERVE-101, which inactivates the liver gene PCSK9 that typically contributes to higher levels of low-density lipoprotein. Credit: PhotobyTawat\/ Shutterstock.<\/figcaption><\/figure>\n<\/div>\n

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Verve Therapeutics released promising results of the first human clinical trial of a novel gene editing technique known as base editing that can help maintain healthy levels of cholesterol. <\/p>\n

This therapeutic approach involves injecting patients with VERVE-101, a Clustered Regularly Interspace Short Palindromic Repeats (CRISPR)-based gene editing therapy, which inactivates the liver gene PCSK9<\/em> that typically contributes to higher levels of low-density lipoprotein (LDL)\u2014a key contributor to heart disease.<\/p>\n

The first PCSK9<\/em> inhibitor to receive FDA approval was the Praluent injection by Sanofi<\/a> in July 2015. Since then, other PCSK9<\/em> inhibitors have made it to market, such as the injection Repatha by Amgen<\/a>. <\/p>\n

According to GlobalData, PCSK9<\/em> inhibitors currently hold a 20% market share in the acute coronary syndrome market and have experienced an aggressive compound annual growth rate of 42% between 2016 and 2023.<\/p>\n

The PCSK9 <\/em>inhibitor injections\u2019 high price, access barriers and need for repeated injections are still limiting the widespread usage of these drugs. However, there is ongoing R&D to develop an oral PCSK9<\/em> inhibitor that would provide a more convenient and accessible option for patients. For example, Merck<\/a> has already initiated clinical trials on its oral cyclic peptide PCSK9 <\/em>inhibitor\u2014MK-0616.<\/p>\n

Even though VERVE-101 is an injectable, what makes this drug different from the rest is that it is the first time CRISPR technology has been successfully used for DNA base pair editing in humans. <\/p>\n

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