A single gene edit could replace daily cholesterol pills forever. Early trials show one infusion cutting LDL by 39-55% for over a year, with no daily medication needed. The catch: a $200,000-$300,000 price tag and years until FDA approval.
How a Single Infusion Rewrites Liver Cells
Verve’s therapy targets a gene called PCSK9, which instructs liver cells to produce a protein that reduces the number of LDL receptors on cell surfaces. Fewer receptors mean less cholesterol gets cleared from the bloodstream. Some people carry natural PCSK9 mutations that silence this gene. They enjoy remarkably low LDL levels and heart disease risk throughout their lives.
The treatment uses base editing, a refined form of CRISPR that changes a single DNA letter without cutting both strands of the double helix. This precision matters. Traditional CRISPR cuts can trigger unintended insertions or deletions. Base editing acts more like a molecular pencil correcting one character in a manuscript.
Delivery relies on lipid nanoparticles, the same fatty-envelope technology proven safe in mRNA COVID-19 vaccines. These shuttle the editing machinery directly into hepatocytes via a one-time intravenous infusion lasting under an hour. Once inside, the edit is permanent. Every time an edited liver cell divides, the daughter cell inherits the corrected DNA, maintaining the therapeutic effect indefinitely.
What the Clinical Evidence Shows So Far
Early-phase human trials have produced encouraging results, though still preliminary. A PCSK9-targeted editing infusion cut LDL cholesterol by a significant margin in patients with heterozygous familial hypercholesterolemia, a genetic condition affecting roughly 1 in 250 people. Phase 1 data indicated LDL reductions in the range of 39-55% at six months, comparable to combining high-dose statins with injectable PCSK9 inhibitors. The difference? A single treatment.
Perhaps more notable: the reductions persisted without fading over 12 months of follow-up. No participants required additional cholesterol medications during that period. Safety monitoring found mild infusion-related reactions but no serious adverse events. Genomic sequencing of blood samples detected no unintended off-target edits.
These are small, early-stage cohorts. The field still needs large Phase 3 trials with thousands of participants to confirm both long-term safety and cardiovascular outcome benefits. A realistic timeline points to FDA approval by 2029-2030 for familial hypercholesterolemia patients, with broader use coming later.
