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ARO-AMG1 is an investigational RNA interference (RNAi) therapeutic under development by Arrowhead Pharmaceuticals and Amgen. It is a subcutaneously administered small interfering RNA (siRNA) therapeutic designed to target and silence the messenger RNA (mRNA) of the lipoprotein(a) (Lp(a)) gene, thereby reducing the production of Lp(a) protein in the liver. Elevated Lp(a) is a well-known genetic risk factor for cardiovascular disease, and therapies to lower Lp(a) are a major unmet clinical need. ARO-AMG1 utilizes Arrowhead's proprietary RNAi delivery technologies and has been the subject of a Phase 1 clinical study focused on evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and subjects with elevated Lp(a). The development of ARO-AMG1 is part of a partnership between Arrowhead Pharmaceuticals and Amgen.
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