ART002

ART002 is a novel in vivo gene-editing therapy developed by Accuredit Therapeutics. It utilizes lipid nanoparticles (LNPs) to deliver Cas9 mRNA and a single-guide RNA (sgRNA) targeting the human PCSK9 gene. By precisely editing and silencing PCSK9, ART002 blocks its interaction with the LDL receptor (LDLR), reducing LDLR degradation and increasing the number of LDLRs on hepatocyte surfaces. This mechanism leads to significant reductions in plasma low-density lipoprotein cholesterol (LDL-C) levels after a single intravenous dose, offering long-term efficacy for patients with heterozygous familial hypercholesterolemia (HeFH). Clinical studies have shown that ART002 achieves up to 90% knockdown of PCSK9 protein and over 50% reduction in LDL-C, with an excellent safety profile and no serious adverse events reported[2][6]. The drug aims to transform chronic management of hypercholesterolemia by reducing reliance on patient adherence required by current therapies.