Drug intelligence / Profile preview

Augury1 inhibitor

Development stage
Preclinical
Lead developer
Gordian Biotechnology
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

Augury1 inhibitor is an experimental adipose-targeted gene therapy developed by Gordian Biotechnology for the treatment of obesity and metabolic disorders. Discovered using an in vivo pooled screening platform in visceral adipose tissue, Augury1 is a novel genetic target whose knockdown in mature adipocytes activates lipolysis, upregulates lipid droplet structural proteins, and promotes fatty acid/glyceride futile cycling. Delivered as an AAV-based artificial miRNA, Augury1 inhibitor has demonstrated significant efficacy in diet-induced obese (DIO) mouse models, driving weight loss and improving glucose tolerance. When combined with the GLP-1 receptor agonist semaglutide, Augury1 inhibition acts additively to enhance weight loss and prevent weight regain following semaglutide cessation, while maintaining persistent insulin sensitization.

Other names
Augury1 knockdown therapyAugury-1 knockdown therapyAugury 1 knockdown therapyAugury1 miRNA therapyAugury-1 miRNA therapyAugury 1 miRNA therapy
02

Targets

ECRG4 (Augurin)

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