[Glowing DNA Helix. Photo Credit to StockCake]

A recent study from Verve Therapeutics has revealed a new therapy called VERVE-102 that can reduce cholesterol levels with just a single injection.

Cholesterol, a waxy, fat-like substance found in the bloodstream, is essential for building healthy cells and hormones.

However, excessive levels of Low-density lipoprotein (LDL), commonly known as “bad cholesterol”, can lead to serious health issues.

When LDL cholesterol travels through the arteries, it may build up and form plaque, which narrows arteries, reduces blood flow, and increases the risk of heart attacks, strokes, and other cardiovascular diseases. 

For many years, high levels of LDL cholesterol have been treated with statins, a class of drugs that reduce cholesterol production in the liver.

Statins are effective for most patients and are typically taken once daily.

Despite these benefits, long-term use is necessary, and some patients may experience side effects such as muscle pain, liver damage, or digestive problems.

More recently, an alternative treatment called PCSK9 inhibitors, which are injectable medications that boost the liver’s ability to clear LDL, has become available.

In spite of these drugs offer strong cholesterol-lowering effects, they still require regular injections, are costly, and can be difficult for patients to maintain over time.

Now, a groundbreaking new therapy, VERVE-102, is showing promise as a long-lasting, possibly permanent alternative.

The experimental treatment utilizes CRISPR base editing, a gene-editing technology that modifies DNA without cutting it.

This therapy targets a gene in the liver called PCSK9, which normally limits the number of LDL receptors, the cellular components that remove LDL cholesterol from the bloodstream.

By deactivating this gene with a single, precise edit using the CRISPR base editing technique, VERVE-102 enhances the liver’s ability to remove LDL from the blood.

In early clinical trials, patients who received a single intravenous infusion of VERVE-102 experienced an average 53% reduction in LDL cholesterol levels within 28 days.

Patients who received the highest dose saw their cholesterol levels drop by up to 69%.

Most importantly, no serious side effects were observed, suggesting that the treatment is both effective and well-tolerated.

These results demonstrate the convenient and powerful potential of this treatment. 

This therapy could transform the future of cholesterol treatment.

For instance, unlike statins or PCSK9 inhibitors, which require daily pills or repeated injections, VERVE-102 may offer a one-time solution for patients with severe or inherited high cholesterol.

Although the treatment is still in the early stages of development, initial results are encouraging.

The U.S. Food and Drug Administration (FDA) has granted the therapy Fast Track designation, allowing faster regulatory review.

Verve Therapeutics, now part of pharmaceutical giant Eli Lilly, has already begun testing higher doses in other countries including the UK, Canada, and Australia, and aims to launch larger trials by 2025.

If proven safe in broader trials, VERVE-102 could represent a major leap forward in cardiovascular medicine, potentially helping millions reduce their risk of heart disease with a single, permanent treatment.