Chinese Gene Therapy Lowers Cholesterol to Infant Levels

Researchers at Xi’an Jiaotong University First Affiliated Hospital, led by Yuan Zuyi, successfully treated patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disease, using a novel gene therapy. HoFH prevents the liver from clearing “bad” cholesterol, leading to dangerously high levels and early cardiovascular events. Existing treatments like medication and liver transplants have limited effectiveness and risks.

Yuan Zuyi’s team developed NGGT006, a gene therapy that delivers a functional low-density lipoprotein receptor gene to liver cells via a viral vector. This aims to restore the liver’s ability to metabolize cholesterol. Clinical trials involving three patients showed that a high dose of NGGT006 reduced LDL cholesterol levels by over 96% in one patient, bringing them down to levels comparable to those of an infant.

Preclinical and clinical data demonstrate the therapy's safety and efficacy, with sustained lipid control observed for at least 52 weeks post-treatment. The team plans to conduct larger clinical trials to confirm these results and further refine the treatment.