Target intelligence / Profile preview

17-beta-hydroxysteroid dehydrogenase type 13 (HSD17B13)

Target
HSD17B13
Molecular classification
Enzyme, Hydroxysteroid dehydrogenase
01

Overview

HSD17B13 is a liver-specific enzyme crucial for hepatic lipid droplet metabolism. Its upregulation promotes fat accumulation in NAFLD/NASH. Loss-of-function genetic variants protect against advanced forms of fatty liver disease including cirrhosis and cancer. Structural studies have enabled rational drug design targeting this enzyme—several inhibitors are now advancing through clinical development as potential therapies for metabolic liver diseases.

Other names
NIIL497SCDR9SDR16C3
02

Mechanism of action

Inhibition of HSD17B13 enzymatic activity

03

Biological functions

Lipid metabolismRetinol dehydrogenase activityLipid droplet metabolism
04

Disease associations

Non-alcoholic fatty liver disease (NAFLD)Metabolic dysfunction-associated steatohepatitis (MASH)Liver fibrosisCirrhosisHepatocellular carcinoma (HCC)
05

Safety considerations

Potential for off-target effects due to structural similarities with other hydroxysteroid dehydrogenasesLong-term effects of HSD17B13 inhibition on overall lipid metabolism are not fully understood
06

Interacting drugs

BI-3231
07

Biomarkers

HSD17B13 expression levels in liver tissuers72613567:TA variant

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