Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial

Therapeutic potential
The participants were all people who had either developed cardiovascular disease early in life (at or below age 55 for men or 65 for women) or were people who had an inherited condition that caused high cholesterol.
The drug was developed by Verve Therapeutics, which was bought by Eli Lilly last year for $1.3 billion. While Verve has been testing the drug on people at some of the highest risks from high cholesterol, its co-founder and now Lilly Senior Vice President Sekar Kathiresan hopes to see its use expand, providing an option for patients with high cholesterol that doesn’t require lifetime management with medications, like statins.
Riyaz Patel, the co-author on the trial and a cardiologist at Barts Health NHS Trust and professor of cardiology at University College London, agreed. In a statement released by Lilly, Patel said the early data was “encouraging,” indicating that VERVE-102 could “offer a novel approach to achieving substantial and durable LDL-C reduction with a one-time treatment.”
“Many patients with elevated LDL-C struggle to achieve sustained control despite ongoing efforts with the medicines available today, putting them at significant risk for cardiovascular events. With coronary artery disease still one of the leading causes of death worldwide, the need for new approaches is real,” Patel said.
While the researchers and Lilly are optimistic, this is, again, a very small, very preliminary look at the drug in clinical action. It will require further trials, with more people and longer follow-ups, to convincingly demonstrate safety and efficacy. But, if all goes well, Lilly notes that the Food and Drug Administration has already granted VERVE-102 a Fast Track designation for review.




