One-shot gene-editing therapy reduces LDL by up to 62% in early trial
TL;DR
In a small Phase I study, Verve Therapeutics’ VERVE-102, a one-time mRNA-based gene-editing therapy targeting PCSK9, reduced LDL by up to 62% in the highest-dose group among 35 participants, with no serious safety events aside from a transient mild rise in a liver enzyme; results suggest durable LDL-C lowering, but larger and longer trials are needed for confirmation, and the FDA has granted Fast Track designation.
- Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial Ars Technica
- A single dose of Lilly's PCSK9 base editor, VERVE-102, reduced PCSK9 by up to 88% and LDL-C by up to 62%, with durable effects supporting its potential as a one-time treatment for hypercholesterolemia investor.lilly.com
- One-and-Done Heart Disease Prevention? Scientists Show It May Be Possible. The New York Times
- A gene therapy lowered cholesterol in early trial results. It has a Philly backstory. Inquirer.com
- Lilly preps for ph. 2 launch after Verve gene editor cuts cholesterol on par with PCSK9 inhibitors Fierce Biotech
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