U.S., Jan. 21 -- ClinicalTrials.gov registry received information related to the study (NCT07353398) titled 'Early Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy' on Jan. 12.
Brief Summary: ART002g1 is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharm...