lepodisiran

Lepodisiran is an investigational small interfering RNA (siRNA) therapy developed to reduce elevated levels of lipoprotein(a) [Lp(a)], a genetically determined risk factor for atherosclerotic cardiovascular disease and aortic stenosis. Lepodisiran works by inhibiting the production of apolipoprotein(a), the key protein component of Lp(a), in the liver. It is designed as a Dicer substrate siRNA with a tetraloop structure and is conjugated to N-acetyl-galactosamine (GalNAc) to facilitate targeted delivery into hepatocytes via liver-specific receptors. Clinical trials have shown that single subcutaneous doses can lower Lp(a) levels by over 90% for up to nearly one year, with durable effects and no major safety concerns reported so far[1][2][3][4][5][6][7]. Developed by Eli Lilly.

Other names

LY3819469 sodiumLY-3819469 sodiumLY 3819469 sodiumlepodisiran