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P901SNC is an antisense oligonucleotide (AON) designed to target and inhibit the expression of proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA. It utilizes a 2′,4′-bridged nucleic acid (BNA) with a nitrogen-containing bridge (2′,4′-BNA NC) modification, which enhances nuclease stability and RNA binding affinity compared to conventional BNA or MOE modifications. By suppressing hepatic PCSK9, P901SNC prevents the PCSK9-mediated degradation of low-density lipoprotein receptors (LDLR), thereby increasing hepatic LDL uptake and significantly lowering serum LDL cholesterol levels. Preclinical studies in mice have demonstrated its potency in reducing cholesterol without the hepatotoxicity sometimes associated with earlier BNA generations.
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