First Trial of ‘Base Editing’ in Humans Lowers Cholesterol–But Raises Safety Concerns

November 13, 2023

Double helix

(Nature) – The first trial in humans of the precise gene-editing technique known as base editing has shown promising results for keeping cholesterol levels in check. The approach injects into people a treatment called VERVE-101, which permanently deactivates a gene in the liver called PCSK9. That gene controls the level of low-density lipoprotein (LDL), or ‘bad’ cholesterol — a key contributor to heart disease…

…But the findings have also drawn criticism. Two serious adverse events in the trial, including a death, have raised safety concerns, and Verve’s share price plummeted by nearly 40% following the results’ release despite their promise. (Read More)