Experimental high cholesterol treatment edits DNA in the body to lower LDL levels

A treatment in clinical trials lowers 'bad' cholesterol by targeting a specific gene in the body. (Image credit: Rasi Bhadramani via Alamy)

An experimental gene therapy to lower high cholesterol is showing encouraging results in clinical trials and is moving closer to approval.

VERVE-102 is being tested in patients with familial hypercholesterolemia (FH), an inherited disorder characterized by high levels of low-density lipoprotein (LDL) – the “bad” cholesterol – in the blood. The drug is also being tested in patients with premature coronary artery disease (CAD), in which the arteries narrow and cannot supply enough oxygen-rich blood to the heart muscle. The age at which CAD is considered “premature” varies, but it typically develops before age 65 in women and before age 55 in men.

Both categories of patients “require significant and sustained reductions” in LDL levels in the blood, Verve Therapeutics, the drug’s developer, said in an April statement. In an ongoing clinical trial, the company tested the drug in 14 patients with familial hypercholesterolemia and/or premature coronary artery disease (CAD) and found that a single dose of the drug reduced LDL levels by an average of 53%.

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