Heart: the treatments of the future – here are the injection and gene therapy that lower high cholesterol and triglycerides
The research findings presented at the American Heart Association (AHA 2026) conference, which has just drawn to a close in Chicago
Key points
Cardiovascular medicine is gearing up for a significant shift in the fight against blood lipids – the number one enemy of the arteries and the main cause of heart attacks and strokes. This is revealed by some of the most interesting studies presented at the American Heart Association (AHA 2026) conference, which has just drawn to a close in Chicago. These are not drugs that doctors will be able to prescribe from tomorrow. Some are still in the early stages of trials, involve only a small number of patients and must pass key tests, particularly regarding long-term safety. But the direction is clear: in future, we will not simply combat high cholesterol and triglycerides with a daily tablet, but will seek to intervene decisively in the biological mechanisms that regulate their production and elimination. And, ideally, with treatments to be administered at intervals of several months or even just once in a lifetime.
An injection to lower both cholesterol and triglycerides by interfering with RNA
Among the most interesting developments to emerge from the American College of Cardiology congress is ARO-DIMER-PA, an experimental therapy based on RNA interference (siRNA) technology that reduces both LDL cholesterol (the ‘bad’ sort) and triglycerides in one go, in patients with mixed hyperlipidaemia. The drug silences the instructions that cause cells to produce certain proteins. In this case, there are two targets: PCSK9, which is involved in regulating LDL cholesterol, and APOC3, which contributes to maintaining high triglyceride levels.
The initial results, obtained in a ‘first-in-human’ study involving just 31 participants, show reductions in LDL cholesterol of up to 65 per cent and in triglycerides of up to 78 per cent. And the potential benefit is not limited to efficacy. siRNA therapies can maintain their effect over prolonged periods, paving the way for treatments administered at much longer intervals.
Genetic scissors to get rid of high triglycerides for good
An even more radical approach is taken by CTX310, a therapy based on CRISPR-Cas9, the gene-editing technology that won the Nobel Prize in Chemistry in 2020. Rather than temporarily blocking the production of a protein, this approach aims to modify the DNA of liver cells by inactivating the ANGPTL3 gene, which is involved in fat metabolism. Inhibiting ANGPTL3 removes a natural ‘brake’ on the enzymes that remove fats from the blood, thereby enabling a sustained reduction in lipid levels.
In a Phase 1b trial conducted in people with severe hypertriglyceridaemia, a single intravenous dose of CTX310 reduced triglyceride levels by 63 per cent at one month. The aim of this ‘definitive’ treatment is to achieve a result that lasts over time, without the need for periodic repeat treatment.
