ARO-HSD is a hepatocyte targeted siRNA therapeutic designed to silence HSD17β13 expression g D17β13 express ARO-HSD Favorable safety profile ARO-HSD was well tolerated with no treatment-related serious adverse events or drug discontinuations Proof of concept In this proof-of-concept study, ARO-HSD was well tolerated and able to significantly reduce HSD17β13 expression in a group of 18 patients, resulting in an improvement in markers of liver injury 18 Patients with suspected or confirmed NASH 16 Healthy volunteers ALT reduction -42.3% Mean change from baseline at day 71 at 200 mg HSD17β13 loss-of-function mutations have been shown to protect against alcoholic and non-alcoholic liver disease including NASH 16 Hepatic HSD17β13 mRNA reduction-93.4%Reduction from baseline at day 71 at 200 mg Hepatic HSD17β13 protein reduction -83.0%Reduction from baseline at day 71 at 200 mg Highlights HSD17B13 loss-of-function mutations are protective against alcohol-related and non-alcoholic liver disease, including NASH.ARO-HSD is a hepatocyte-targeted siRNA therapeutic designed to silence HSD17B13 expression.ARO-HSD demonstrated a favorable safety profile in healthy volunteers and patients with suspected NASH.Mean change from baseline at Day 71 in hepatic HSD17b13 mRNA was -93.4% (ARO-HSD 200 mg).
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