Lepodisiran: Extended-Duration RNA Targets Lipoprotein

JAMA Network

About The Study: In this phase 1 study of 48 participants with elevated lipoprotein(a) levels, lepodisiran was well tolerated and produced dose-dependent, long-duration reductions in serum lipoprotein(a) concentrations. The findings support further study of lepodisiran, which is a short interfering RNA directed at hepatic synthesis of apolipoprotein(a), an essential component necessary for assembly of lipoprotein(a) particles.

Authors: Steven E. Nissen, M.D., of the Cleveland Clinic Center for Clinical Research in Cleveland, is the corresponding author.

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(doi:10.1001/jama.2023.21835)

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