One injection, 62% lower LDL - permanently. Gene editing for cholesterol is here.
. is correct. Besides GLP-1 antagonist like Ozempic or, more recently, Retatrutide, this is probably the next breakthrough that is almost impossible to put into words.
Verve Therapeutics (Eli Lilly) just published Phase 1 results for VERVE-102 in the NEJM.
It's a single-infusion base-editing therapy that inactivates PCSK9 in the liver. At the highest dose, PCSK9 dropped 88% and LDL cholesterol fell 62%. Reductions held for at least a year.
35 patients with familial hypercholesterolemia or premature coronary artery disease. No dose-limiting toxicities. Main side effects: mild infusion reactions and transient liver enzyme elevations.
Still Phase 1, still small, no cardiovascular outcome data yet. But the proof of concept for permanent, one-shot LDL reduction via gene editing is real.
This is an absolute game changer and im not exaggerting.
Elevated LDL cholesterol is responsible for an estimated 4.4 million deaths every year worldwide and remains the single biggest modifiable driver of cardiovascular disease, the leading cause of death globally.
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Crémieux
@cremieuxrecueil
Eli Lilly has done it.
They've gone and made what seems to be a powerful, permanent gene therapy for LDL cholesterol.
That means they'll be able to effectively prevent most heart disease with a single infusion!
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