HeFH affects about one in every 200 to 500 people worldwide.
patients usually carry a mutation in the LDLR gene, which causes a decrease in LDLR expression and elevates LDL-C levels in the blood.
over time, high LDL-C accumulates in the arteries of the heart, causing blood flow to decrease or block, eventually leading to a heart attack or stroke.
previous studies have shown that PCSK9 gene infestion can increase LDL-patient expression, leading to lower LDL-C levels and reducing the risk of coronary heart disease.
VERVE-101 causes the PCSK9 gene to inferces by altering the DNA genetic sequence of PCSK9 by a single A-to-G.
latest data have shown that monobase editing of the PCSK9 gene in the livers of non-human primates can consistently reduce levels of LDL-C in the blood and PCSK9 proteins in the blood.
study, a single intravenous infusion reduced blood LDL-C by 59% in week 2 and was maintained within 6 months of treatment.
LDL-C decreased by an average of 61% over that time period.
the average blood PCSK9 protein level decreased by 89% over the same six-month period.