Drug intelligence / Profile preview

STP152G

Development stage
Preclinical
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

STP152G is a GalNAc-conjugated small interfering RNA (siRNA) therapeutic developed by Sirnaomics for the treatment of transthyretin (ATTR) amyloidosis. It utilizes the company's proprietary GalAhead platform, specifically the mxRNA (miniaturized RNAi triggers) technology, which consists of single ~30 nucleotide oligonucleotides designed to downregulate individual genes. STP152G is designed to silence the expression of the transthyretin (TTR) gene in the liver, thereby reducing the production of both mutant and wild-type TTR protein to prevent amyloid fibril formation and deposition. The drug is administered via subcutaneous injection and is currently in preclinical development.

02

Targets

ASGPR (Asialoglycoprotein Receptor 1)TTR (Transthyretin)

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