Revolutionizing Cholesterol Management: The Potential of CRISPR Base Editing in Lowering LDL Cholesterol

Did you know that as of 2024 reports people suffering with elevated levels of Low density lipoprotein (LDL) cholesterol has gained attention and global health concern.

According to World heart federation the mortality rate due to high LDL cholesterol has contributed approximately 4.4 million deaths annually accounting for 7.8% of all deaths.

Lowering LDL cholesterol levels is crucial in reducing the risk of major cardiovascular events. Studies have shown that a 1 mmol/L reduction in LDL cholesterol can lead to a significant decrease in the risk of heart attacks and strokes. 

Many companies across the world is trying to solve this concern either by developing the diagnostic techniques focusing on early interventions or by developing therapeutic solutions to save the life !!

One such company is Verve Therapeutics, A US based Bio-Tech company has developed a single treatment using " CRISPR Base edit - Technology " to lower the LDL cholesterol.

Diving In :

Scientists are now working on a revolutionary gene-editing therapy that could permanently reduce LDL cholesterol levels, potentially changing how we treat heart disease forever.

You have probably heard CRISPR-Cas9, which cuts the DNA at a specific place like scissors. This is effective but can cause unintended effects. Base editing, however, doesn’t cut the DNA. It changes just one letter in the genetic code!

VERVE-102 is the name of the new gene-editing medicine being developed by Verve Therapeutics.

It’s designed for people who have very high cholesterol, especially due to inherited conditions like familial hypercholesterolemia. In this case, it targets a gene called PCSK9 in the liver, which plays a key role in controlling cholesterol levels. Unlike traditional way of lowering LDL, which were drugs that should have used regularly, this renovate treatment is for lifetime.

But is it Really working ?

In early human trials (called Phase 1b), the treatment showed exciting results:

·    People who got the highest dose saw their LDL cholesterol drop by about 53% on average.

·    The maximum drop was 69%.

·    This was all from a single infusion just one treatment.

·     No serious side effects were reported at that dose so far.

These results suggest that the treatment might work really well, and with just one dose instead of taking daily pills or injections.

Traditional cholesterol treatments	VERVE-102 gene-editing treatment
Usually involve daily pills (like statins) or frequent injections	Just one single infusion
Need to be taken for life	Designed to last a lifetime
Some people forget or stop taking them if they feel normal	No need to remember pills or appointments it's done once
Don’t always work well for everyone especially in genetic cases	Could be more effective, especially for people with inherited high cholesterol

High LDL cholesterol is one of the biggest causes of heart attacks and strokes worldwide.

If this treatment works as expected:

• Millions could avoid future heart problems.

• Healthcare costs could drop dramatically.

• It could open the door to gene editing for other common diseases too.

It’s part of a larger movement toward precision medicine, where treatments are designed to match your DNA instead of a one-size-fits-all pill.

References:

1. World Heart Federation. (2024). Cholesterol Roadmap. World Heart Federation. Retrieved from https://world-heart-federation.org/cvd-roadmaps/whf-global-roadmaps/cholesterol

2. Verve Therapeutics. (2025). Verve Therapeutics Announces Positive Initial Data from the Heart-2 Phase 1b Clinical Trial of VERVE-102, an In Vivo Base Editing Medicine Targeting PCSK9. GlobeNewswire. Retrieved from https://www.globenewswire.com/news-release/2025/04/14/3060774/0/en/Verve-Therapeutics-Announces-Positive-Initial-Data-from-the-Heart-2-Phase-1b-Clinical-Trial-of-VERVE-102-an-In-Vivo-Base-Editing-Medicine-Targeting-PCSK9.html