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gatuzosiran
- Development stage
- Unknown
- Lead developer
- Arrowhead Pharmaceuticals
- 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
Gatuzosiran (GSK4532990, formerly ARO-HSD) is a GalNAc-conjugated small interfering RNA (siRNA) therapeutic designed to target and reduce the expression of hydroxysteroid 17-beta-dehydrogenase-13 (HSD17B13) in hepatocytes. HSD17B13 is a liver-specific lipid droplet-associated protein, and loss-of-function mutations in its gene are strongly associated with a reduced risk of chronic liver diseases, including metabolic dysfunction-associated steatohepatitis (MASH) and alcohol-related liver disease (ALD). Gatuzosiran was originally developed by Arrowhead Pharmaceuticals using its TRiM (Targeted RNAi Molecule) platform and was licensed to GSK in 2021 for global development (excluding Greater China, where rights were retained by Arrowhead and later licensed/developed via its joint venture Visirna Therapeutics). While GSK discontinued the development of gatuzosiran for MASH in early 2026 to focus on other pipeline priorities, it continues clinical development for ALD. Visirna Therapeutics continues to develop the asset (as VSA-006) for MASH in Greater China.
02
Targets
HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)
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