A new experimental gene-editing treatment may one day offer a long-lasting solution for people with dangerously high cholesterol, potentially replacing the need for daily medication, according to early clinical trial results published in the New England Journal of Medicine.The therapy uses CRISPR-Cas9 technology to disable a gene called ANGPTL3, which plays a key role in regulating levels of low-density lipoprotein (LDL), often referred to as “bad” cholesterol, along with triglycerides, another blood fat linked to heart disease. In a small pilot study involving 15 patients with severe, treatment-resistant cholesterol disorders, those who received the highest dose of the therapy saw LDL levels fall by nearly 50% and triglycerides drop by about 55%.Researchers say the approach could eventually provide a “one-and-done” treatment for people with inherited or hard-to-manage cholesterol conditions, many of whom currently rely on multiple medications over their lifetime.
A treatment inspired by a natural mutation
The idea behind the therapy stems from a rare genetic mutation found in about 1 in 250 people. Those born with a non-functioning ANGPTL3 gene naturally maintain very low cholesterol and triglyceride levels and have a significantly reduced risk of cardiovascular disease, without apparent negative health effects. Scientists hoped to replicate this benefit through targeted gene editing.Unlike traditional gene therapies, which may act broadly across the body, this treatment is designed to modify only liver cells, where cholesterol production and removal are regulated. Researchers say this targeted approach may reduce long-term risks.
Encouraging but early results
The trial was primarily conducted to test safety rather than effectiveness. Most side effects reported were minor, including temporary infusion reactions. One participant died six months after treatment, but investigators said the individual had advanced cardiovascular disease and received a dose too small to have a therapeutic effect, and regulators have determined the death was not caused by the therapy.Larger Phase 2 and Phase 3 trials are being prepared to evaluate how well the treatment works in broader groups of patients and to monitor potential long-term effects. Participants will be followed for up to 15 years as required by regulatory guidelines for gene-editing therapies.
A possible shift in long-term heart disease treatment
High LDL cholesterol remains one of the strongest risk factors for heart disease, which is the leading cause of death worldwide. While several effective medications exist, including statins and newer injectable drugs, many patients struggle with side effects, adherence, or persistent high cholesterol levels.Experts caution that although the early findings are promising, it is too soon to know whether the cholesterol reductions will last for life or whether unforeseen safety concerns may emerge. For now, doctors stress that people currently prescribed cholesterol-lowering medication should continue taking it.If future studies confirm long-term safety and effectiveness, the therapy could reshape how cardiovascular disease is prevented, offering some patients a simpler, more durable option for controlling cholesterol over the course of their lives.Note: The information provided in this article is for educational purposes only and is not intended as medical advice. Always consult with a qualified healthcare professional before starting any new medication or treatment and before changing your diet or supplement regimen.
