Drug intelligence / Profile preview

VSA006

Development stage
Unknown
Lead developer
Visirna 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

VSA006 is an investigational small interfering RNA (siRNA) therapeutic developed by Visirna Biotechnology for the treatment of non-alcoholic steatohepatitis (NASH) and non-alcoholic fatty liver disease (NAFLD). The drug targets the mRNA of hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) in the liver, leading to a reduction in HSD17B13 protein levels. This mechanism is designed to mimic the protective effect of naturally occurring loss-of-function genetic variants of HSD17B13, which are associated with a reduced risk of chronic liver disease progression and cirrhosis. VSA006 is administered via injection and is currently being evaluated in Phase 2 clinical trials to assess its efficacy, safety, and pharmacokinetic profile in patients with NASH.

02

Targets

HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)

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