AAV8-hFGF1∆HBS

AAV8-hFGF1∆HBS is an adeno-associated virus (AAV) serotype 8-based gene therapy designed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). It utilizes the thyroxine-binding globulin (TBG) promoter to drive the hepatocyte-specific expression of hFGF1∆HBS, a modified version of human fibroblast growth factor 1 (FGF1) featuring three substitutions in its heparin-binding sites. This modification is intended to reduce the mitogenic activity associated with wild-type FGF1 while preserving its metabolic benefits. In preclinical models, the therapy has been shown to reduce hepatic steatosis, inflammation, and fibrosis by normalizing fatty acid synthesis and suppressing fatty acid uptake through the downregulation of stearoyl-CoA desaturase-1 (SCD1) and cluster of differentiation 36 (CD36).

Other names

hFGF1∆HBS gene therapyAAV8-mediated hFGF1∆HBShFGF1ΔHBS