AAV8-Augury1 artificial miRNA

AAV8-Augury1 artificial miRNA is an experimental gene therapy candidate developed by Gordian Biotechnology for the treatment of obesity. The therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver an artificial microRNA (miRNA) designed to knock down the expression of Augury1, a novel target identified through Gordian's in vivo mosaic screening platform (Perturb-seq) in visceral adipose tissue. The mechanism of action involves the silencing of Augury1, which leads to the upregulation of esterification within the fatty acid/glyceride futile cycle. Preclinical studies in obese mouse models have demonstrated that delivery of this asset results in dose-dependent weight loss and fat mass reduction without loss of muscle mass or adverse effects on glucose tolerance and cholesterol levels.