How a ‘one-and-done’ gene‑editing treatment could lower cholesterol
A small, early‑stage trial has shown that a single dose of a gene‑editing therapy can markedly lower cholesterol levels in participants who continued to struggle with the condition despite standard medication. The experiment, conducted by researchers using a CRISPR‑based platform, targeted a gene that influences low‑density lipoprotein (LDL) metabolism, delivering a permanent modification after one infusion.
In the study, volunteers received an intravenous injection of the edited therapy and were monitored for several weeks. Results indicated reductions of up to 40 % in LDL cholesterol and a similar drop in another key cardiovascular risk marker, lipoprotein(a). The treatment is described as “one‑and‑done” because it aims to provide lasting benefit without the need for ongoing dosing, contrasting with traditional drugs that require daily or monthly administration.
The findings arrive at a time when clinicians are seeking alternatives to statins and PCSK9 inhibitors, which, while effective, can be limited by side‑effects, high cost, or patient non‑adherence. Generic experts noted that a durable, single‑administration approach could simplify management of hypercholesterolemia and broaden access, particularly for patients who cannot tolerate existing therapies. Safety data from the trial were encouraging, with only mild, transient reactions reported and no serious adverse events linked to the gene edit. Nonetheless, researchers emphasized that longer‑term monitoring is essential to assess durability and any delayed effects.
Regulators are expected to review the data as part of a phased development plan that includes larger, multi‑center studies. If subsequent trials confirm efficacy and safety, the therapy could represent a paradigm shift in cardiovascular prevention, potentially reducing the burden of heart disease worldwide. For now, the study offers a proof of concept that precise, permanent genetic modification may become a viable tool in the fight against high cholesterol.
