The first human clinical trial of a novel gene-editing technique known as base editing has shown promising results in maintaining healthy levels of cholesterol. In the trial by Verve Therapeutics, physicians injected patients with a Clustered Regularly Interspace Short Palindromic Repeats (CRISPR)-based gene editing therapy called VERVE-101, which inactivated the liver gene PCSK9 that typically contributes to higher levels of low-density lipoprotein (LDL)—a key contributor to heart disease. The VERVE-101 clinical trial shows that this therapeutic approach can also be used to target genes associated with cardiovascular disease in addition to cancer genes, says GlobalData.
According to GlobalData, PCSK9 inhibitors, like VERVE-101, currently holds 20 per cent market share in the acute coronary syndrome market, but more interestingly, PCSK9 inhibitors have experienced an aggressive compound annual growth rate (CAGR) of 42 per cent between 2016 and 2023.
Joselia Carlos, Medical Device Analyst at GlobalData, comments, “The first PCSK9 inhibitor to receive FDA approval was Praluent by Sanofi in July 2015. Since then, other PCSK9 inhibitors have made it to market, such as Repatha by Amgen. However, what makes VERVE-101 groundbreaking is that it is the first time CRISPR technology has been successfully used for DNA base pair editing in humans.”
Results from the VERVE-101 clinical trial is evidence of how far precision and personalised medicine has come. According to GlobalData’s 2022 Thematic Research Report on Precision and Personalised Medicine, precision and personalised medicine is most often used in oncology.
Carlos continues, “Precision and personalised medicine is often used to detect genetic mutations, such as the BRCA1 and 2 mutations, that would likely lead to cancer. Similarly, this medical model can also detect if there are any genetic mutations in genes associated with other indications, which include cardiovascular disease. For example, an individual may have a mutation in the PCSK9 gene that would lead to higher levels of LDL.”
While VERVE-101 had promising results, the treatment came with some side effects. The most serious adverse event from this trial was that two out of the ten participants experienced heart attacks—including one who died from the heart attack five weeks later.
Carlos concludes, “VERVE-101 is the first of its kind, but it may be a hard sell considering there are viable and less risky alternatives such as Praluent by Sanofi and Repatha by Amgen. Further clinical trials are required to guarantee the safety of this novel drug.”