Drug intelligence / Profile preview

STP146G

Development stage
Unknown
Lead developer
Sirnaomics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

STP146G is an investigational RNA interference (RNAi) therapeutic developed by Sirnaomics for the treatment of hypercholesterolemia. It utilizes a small interfering RNA (siRNA) molecule conjugated to a N-acetylgalactosamine (GalNAc) moiety, which enables highly specific delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR). Once inside the liver cells, STP146G triggers the RNA-induced silencing complex (RISC) to degrade the messenger RNA (mRNA) encoding Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9). By inhibiting the production of PCSK9, the drug prevents the degradation of low-density lipoprotein receptors (LDLR), thereby increasing their density on the cell surface and facilitating the clearance of LDL cholesterol from the blood. This approach offers the potential for potent and durable lipid-lowering effects with infrequent dosing.

02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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