A new gene-editing therapy has been tested on humans for the first time, resulting in a significant reduction in their low-density lipoprotein (LDL) cholesterol that could potentially last for decades. The study’s authors presented these early results at the American Heart Association’s Scientific Sessions 2023 in Philadelphia.
The study focused on nine patients—seven men and two women—with an average age of 54 years old. All patients had previously been diagnosed with heterozygous familial hypercholesterolemia and presented with extremely high LDL cholesterol levels despite already taking LDL cholesterol-lowering medications.
“These numbers are consistent with the fact that, despite available treatments, only about 3% of patients living with heterozygous familial hypercholesterolemia globally have reached target treatment goals,” senior author Andrew M. Bellinger, MD, PhD, chief scientific officer at Verve Therapeutics in Boston, said in a statement.
The investigational therapy in question, VERVE-101, is a CRISPR-based gene-editing drug designed to permanently turn off the PCSK9 gene in a patient’s liver. Previous animal studies had concluded that treatment could reduce PCSK9 levels by as much as 83% and reduce LDL cholesterol by as much as 69%. The animals in those studies were only given one dose of the gene-editing therapy, and their reductions in PCSK9 and LDL cholesterol have now been consistent for more than 2.5 years.
For this first-in-human study, the nine patients all received a single intravenous infusion of VERVE-101. The first three patients were given a dose of 0.1 mg/kg, but that dose was gradually increased as patients underwent treatment. The highest dose given to a patient was 0.6 mg/kg.
Overall, these patients all experienced significant reductions in LDL cholesterol. Higher doses were associated with making a bigger impact.
“We were thrilled to see that the previous testing we had done of VERVE-101 in animal models translated faithfully to these findings in humans,” Bellinger said in the same statement.
Researchers did note that two patients experienced a serious adverse cardiovascular event. These patients both presented with advanced coronary artery disease.
This study is still ongoing. In fact, it is still enrolling new patients. The current plan is to follow each patient for a total of 15 years.
Verve Therapeutics, the company behind this new medication, funded this analysis.
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Michael has more than 16 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.