Drug intelligence / Profile preview

SLN365

Development stage
Unknown
Lead developer
Silence Therapeutics
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

SLN365 is an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to inhibit the expression of the *HSD17B13* (Hydroxysteroid 17-Beta Dehydrogenase 13) gene in the liver. Developed by Silence Therapeutics in collaboration with AstraZeneca, it utilizes Silence's proprietary mRNAi GOLD™ (GalNAc Oligonucleotide Liver Delivery) platform. The target, HSD17B13, is a liver-specific enzyme; genetic variants that result in its loss of function have been shown to provide protection against the progression of chronic liver diseases, including metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), and cirrhosis. By silencing this gene, SLN365 aims to mimic this protective genetic effect to treat patients with liver fibrosis and inflammation.

02

Targets

HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)

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