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Lipoprotein(a) [Lp(a)], an apoB100-containing lipoprotein with an attached apolipoprotein(a) molecule, has been associated with adverse cardiovascular events in observational and mendelian randomization studies. No studies yet show, however, that lowering Lp(a) levels reduces cardiovascular events, and no medications are currently FDA-approved for Lp(a) lowering. In a manufacturer-funded, phase 1, dose-escalation study, researchers examined the Lp(a)-lowering potential of lepodisiran, a short interfering RNA (siRNA) molecule inhibiting hepatic synthesis of apolipoprotein(a), thereby inhibiting Lp(a) assembly (NCT04914546).
The trial randomized 48 adults (mean age, 47 years; 35% women; 15% Black) without cardiovascular disease and with serum …