ART002g1

ART002g1 is an investigational *in vivo* gene therapy that utilizes base editing technology to target the PCSK9 gene in the liver. Developed by researchers at Shanghai General Hospital and Shanghai Jiao Tong University School of Medicine, the therapy is designed to introduce a precise genetic modification that disrupts the expression of proprotein convertase subtilisin/kexin type 9 (PCSK9). By reducing PCSK9 levels, the drug aims to increase the availability of low-density lipoprotein (LDL) receptors on hepatocytes, thereby lowering circulating levels of low-density lipoprotein cholesterol (LDL-C). ART002g1 is currently being evaluated in a Phase 1 clinical trial for the treatment of patients with heterozygous familial hypercholesterolemia (HeFH) who require additional LDL-C reduction.