Drug intelligence / Profile preview

STP135G

Development stage
Discontinued
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, Intradermal, Intratumoral, Systemic
01

Overview

STP135G is an investigational RNA interference (RNAi) therapeutic developed by Sirnaomics for the treatment of hypercholesterolemia. It consists of a small interfering RNA (siRNA) designed to target and silence the expression of the *PCSK9* (proprotein convertase subtilisin kexin type 9) gene in hepatocytes. By reducing PCSK9 levels, the drug aims to increase the density of low-density lipoprotein receptors (LDLR) on the surface of liver cells, thereby enhancing the clearance of LDL cholesterol from the bloodstream. STP135G utilizes Sirnaomics' proprietary PDoV-GalNAc (Peptide Docking Vehicle-N-acetylgalactosamine) delivery platform, which facilitates targeted delivery to the liver via the asialoglycoprotein receptor (ASGPR). While initially developed as a potent PCSK9 inhibitor, recent pipeline updates suggest that its development may have been discontinued.

02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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