Experimental Gene Silencing Therapy Found To Reduce Risk Of Genetic Heart Disease

Jun, 2022 - By SMI

Results of phase 1 trials of this experimental siRNA therapy found that levels of lipoprotein(a) in blood that lead to cardiovascular disease are reduced by up to 98%

Lipoprotein(a) or Lp(a) is a known risk factor for atherothrombotic cardiovascular disease. Various studies in past demonstrated a causal link between blood concentrations of lipoprotein(a) and the risk of cardiovascular disease such as stroke, peripheral arterial disease, and even cardiovascular death. Now researchers at Cleveland Clinic developed an experimental gene silencing therapy that reduces blood levels of lipoprotein by 98% further reducing risk of genetic heart disease.

The researchers in this new study developed the first treatment that can almost completely decrease levels of lipoprotein(a). In the Phase 1 trial of this therapy, the scientists administered subjects with an injection of SLN360, which is a tiny interfering RNA molecule or siRNA molecule that silences the gene responsible for production of lipoprotein. The subjects involved in the trial had Lp(a) levels that were twice the amount considered normal. The team gave a placebo to 8 of the participants out of 32 and others were given doses of SLN360 of either 30mg, 100mg, 300mg, or 600mg. Further, the scientists kept the subjects under observation for 24 hours after administering the doses and then the subjects were examined for a period of five months.

The results of this trial showed that subjects who received placebo showed small changes in Lp(a) levels in that period. However, the subjects who received SLN360 doses showed significant reduction. The subjected who received 100mg were seen to have Lp(a) level reduction by 86%, subjects with 300mg showed 96% drop, and those with 600mg showed 98% drop in Lp(a) levels. The highest amount of doses also reduced bad LDL cholesterol levels by 25% without causing any side effects. The team is hoping that this experimental therapy or other similar RNA therapies could be used for reducing genetic risk of heart disease in patients.