Suzhou-based gene editing specialist AccurEdit Therapeutics announced that its in vivo gene editing product, ART002, has achieved drug saturation and safely reduced low-density lipoprotein cholesterol (LDL-C) with a single dose in a multi-center, open, single-arm, exploratory investigator-initiated trial (ITT) for heterozygous familial hypercholesterolemia. This marks the first time an in vivo gene editing product has achieved such results in humans.
Trial Results
The trial demonstrated that ART002 effectively reduced LDL-C levels with just one dose. The product delivers sgRNA and Cas9 mRNA targeting the PCSK9 gene via lipid nanoparticles (LNPs), enabling specific knockout of the PCSK9 gene in liver cells. This significantly reduces PCSK9 protein expression, blocks its binding to LDLR, and increases the number of LDLR on the surface of liver cells, thereby reducing plasma LDL-C levels.
Product Profile
ART002 is designed for precise editing of the LDL-C regulatory gene PCSK9 through a single dose, aiming to persistently reduce LDL-C levels and lower the risk of cardiovascular disease. The drug’s ability to achieve sustained LDL-C reduction with a single administration represents a significant advancement in gene editing therapy.-Fineline Info & Tech
