A clinical trial has shown that an experimental gene therapy based on CRISPR technology can sustainably lower levels of bad cholesterol after a single dose, with the effect lasting for at least a year.
The treatment, called CTX310, targets a gene in liver cells responsible for producing the ANGPTL3 protein, which plays a role in regulating blood lipid levels. The treatment aims to reduce the production of this protein, mimicking a natural genetic variation in some individuals that is associated with lower lipid levels and a reduced risk of cardiovascular disease.
In the trial, 15 people with severe cholesterol or triglyceride disorders received a single intravenous dose of CTX310, and the researchers then followed up with them for 12 months.
One participant died 179 days after starting treatment, but researchers concluded that the death was not related to the treatment. The other 14 participants did not experience any new serious side effects during the follow-up period.
The most notable results were seen in the four participants who received the highest dose, as their ANGPTL3 protein levels decreased by an average of 78.6%, while their LDL cholesterol decreased by 52.5% and their triglycerides by 47.8%.
These findings are significant because high levels of LDL cholesterol can lead to plaque buildup in the arteries, increasing the risk of heart attacks and strokes. Traditional treatments, such as statins, need to be taken regularly to maintain their effectiveness.
CTX310, on the other hand, relies on a gene modification that is supposed to have a long-lasting effect, which may make it a treatment option in the future that does not require repeated doses.
However, the results are still preliminary, as the trial was limited to only 15 participants, making it necessary to conduct larger and longer-term studies to confirm the effectiveness and safety of the treatment.
Phase 1A was the first human trial of CTX310, while Phase 1B is currently underway to evaluate the treatment in specific groups of patients with lipid disorders, using a dose equivalent to the highest dose that showed effectiveness in the previous phase.
The study's findings were published in the New England Journal of Medicine.
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