A single administration of the experimental gene therapy CTX310 reduced levels of “bad” cholesterol by approximately half, with the effect lasting for at least a year. The results of the technology’s first trial in humans were published in the New England Journal of Medicine, UNN writes.
Details
The therapy uses CRISPR-Cas9 gene-editing technology. Following a single intravenous administration, it reaches liver cells and switches off the ANGPTL3 gene, which is involved in regulating blood fat levels.
The study involved 15 patients with lipid metabolism disorders who had responded insufficiently to drug treatment. After 12 months, patients who received the highest dose of CTX310 had, on average, LDL cholesterol levels that remained 52.5% lower and triglyceride levels that were 47.8% lower.
One administration — an effect lasting at least a year
According to Cleveland Clinic cardiologist and one of the study’s authors, Luke Laffin, the duration of the effect proved impressive. During the year of observation, the researchers also recorded no serious adverse events associated with CTX310.
If the effectiveness and safety of this approach are confirmed by larger studies, gene editing could potentially become a fundamentally new way to combat high cholesterol — instead of taking medication continuously, the gene that affects lipid levels is altered after a single administration of the therapy.
At the same time, CTX310 remains an experimental treatment. This is an early-phase clinical trial involving only 15 people, so researchers still need to confirm its long-term safety and effectiveness in a much larger number of patients. The trial participants are planned to be monitored for another 15 years.
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