A trial testing a new CRISPR-based treatment to lower cholesterol has officially kicked off in New Zealand. If it works as well as it did in animal trials, the one-and-done treatment could save countless lives — permanently lowering cholesterol and the risk of a heart attack.
Verve Therapeutics’ cholesterol treatment… is designed to permanently deactivate a gene in the liver that controls the production of PCSK9 — a protein that prevents the removal of excess cholesterol from the body.
In monkey trials, it reduced LDL cholesterol levels by 70% in just two weeks and kept them low for at least two years — suggesting that it could effectively cure high cholesterol in people.
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Related on the SLP
The delivery mechanism for Verve’s treatment to lower cholesterol is also notable.
Instead of taking the traditional approach to gene therapy, using a hollowed-out virus to deliver its CRISPR therapy, the company uses nanoparticles to deliver genetic instructions into cells, just like the ones used to package the mRNA vaccines for COVID-19.
The big picture: Therapies that work in animals often don’t translate to humans, and the permanence of Verve’s treatment means there is no undo button.
