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GSK'990
- Development stage
- Unknown
- Lead developer
- GSK
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Injectable (likely Subcutaneous Or Intravenous; Specific Route Not Explicitly Stated But Typical For siRNA Therapies)
01
Overview
GSK'990 is an investigational small interfering RNA (siRNA) therapeutic developed by GSK to selectively target and inhibit 17-beta-hydroxysteroid dehydrogenase 13 (HSD17B13), a liver-expressed enzyme implicated in the pathogenesis of steatotic liver diseases. By reducing HSD17B13 expression, GSK'990 aims to slow or halt disease progression in alcohol-related liver disease (ALD) and metabolic dysfunction-associated steatohepatitis (MASH). In early clinical studies, GSK'990 demonstrated robust knockdown of its target and encouraging reductions in markers of liver injury. The drug is currently being evaluated in phase II trials for both ALD and MASH, including patients with advanced fibrosis[4][6]. It represents a precision medicine approach informed by genetic insights into SLD pathophysiology.
02
Targets
HSD17B13 (17β-Hydroxysteroid Dehydrogenase Type 13)
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