Gene-editing drug slashes bad cholesterol 62 percent in trial

May 28, 2026

Iniulat ng AI

An experimental therapy called VERVE-102 lowered LDL cholesterol by up to 62 percent after a single dose in an early safety study. The results come from a Phase I trial involving 35 patients with high cholesterol or early cardiovascular disease. Data were published this week in the New England Journal of Medicine.

Researchers reported that the treatment appeared safe, with no serious side effects even at the highest dose. Participants experienced only a temporary mild rise in liver enzymes. The drug uses mRNA packaged in nanoparticles to edit the PCSK9 gene in liver cells, permanently reducing production of a protein that raises LDL levels.

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Scientific illustration of HELZ2 protein in the liver regulating cholesterol release.

UT Southwestern researchers identify HELZ2 protein that controls the liver’s release of cholesterol-carrying particles

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Scientists at UT Southwestern Medical Center report they have identified a protein, HELZ2, that acts as a key regulator of how many cholesterol-carrying particles the liver releases into the bloodstream by affecting the gene APOB. The study was published in the American Heart Association journal Circulation and could inform future research into heart disease and fatty liver disease.

DNA hairpin molecules lowered cholesterol by 47% in mice by silencing PCSK9, researchers report

Researchers from the University of Barcelona and the University of Oregon report that short DNA molecules known as polypurine reverse Hoogsteen hairpins (PPRHs) suppressed the PCSK9 gene and reduced blood cholesterol in a mouse model. In transgenic mice carrying the human PCSK9 gene, a single injection of one candidate (HpE12) cut plasma PCSK9 by 50% and total cholesterol by 47% three days later, according to findings published in Biochemical Pharmacology.

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Nanoparticles reduced amyloid-β and improved cognition in an Alzheimer’s mouse model, researchers report

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July 22, 2026 14:05

Gene-editing drug slashes bad cholesterol 62 percent in trial

Mga Kaugnay na Artikulo

UT Southwestern researchers identify HELZ2 protein that controls the liver’s release of cholesterol-carrying particles

DNA hairpin molecules lowered cholesterol by 47% in mice by silencing PCSK9, researchers report

ApoB test outperforms LDL for guiding cholesterol treatment

Virus injection halts pancreatic tumor growth in trial

Experimental drug reduced DNA damage and inflammation in an Alzheimer’s mouse model, King’s College London says

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mRNA injection reduces chromosome errors in older eggs

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