Drug intelligence / Profile preview

P901SNC

Development stage
Preclinical
Lead developer
National Cerebral and Cardiovascular Center Research Institute
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Intraperitoneal, Subcutaneous
01

Overview

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.

Other names
2′,4′-BNA NC-modified anti-PCSK9 AON
02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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