Single-dose gene therapy halves LDL cholesterol in early Chinese trial
An experimental one-shot gene-editing jab reduced LDL-C levels by about 50% in six months for six Chinese patients with familial hypercholesterolemia, with no serious safety issues reported.
A small Chinese study tested a single-dose gene-editing injection designed to silence the PCSK9 gene in liver cells of patients with familial hypercholesterolemia. Six adults aged 34 to 62 received the therapy, and those given the highest dose experienced a 52% reduction in low-density lipoprotein cholesterol and a 74% drop in PCSK9 enzyme activity after six months. Reported side effects were limited to brief fever and muscle soreness, with no serious complications observed.
The investigational treatment uses a reengineered enzyme from the bacterium Hafnia paralvei to edit human DNA. Participants will be followed for up to 15 years to assess durability and safety. The findings echo results from a recent Cleveland Clinic trial that used a different gene target, ANGPTL3, achieving similar lipid-lowering effects.
