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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.
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