SOURCE-LINKED STUDY SUMMARY
In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia.
Abstract summarizedQuality not appraised
- What was studied
- An open-label phase 1 study tested liver-directed PCSK9 base editing and LDL cholesterol lowering.
- Who / model studied
- 35 adults with heterozygous familial hypercholesterolemia or premature coronary artery disease.
- Studied dose & schedule
- One intravenous infusion; six cohorts spanning 0.3–1.0 mg of total RNA per kilogram.
- What the study found
- Mean LDL reductions ranged from 9% to 62% across the lowest and highest doses. Fifteen participants had at least one year of follow-up. Infusion reactions and transient ALT elevations occurred; aspiration pneumonitis occurred in one participant.
- Limitations & context
- Small, uncontrolled early study of biomarkers. It does not establish cardiovascular-event prevention or lifetime safety; no dose-limiting toxicity is not proof of no risk.
Read summary source ↗ · Prepared 9/24/2026 · AI-assisted, source-based summary
Review record & corrections
Summary prepared: . Source scope: abstract. This is an AI-assisted source summary, not an independent clinician sign-off.
No public field-by-field revision history is available for this card yet. The date above is the summary date, not proof that every field was updated then.
Inspect the source · Correction policyResearch findings apply to the population and conditions studied. This educational summary is not a treatment recommendation or a formal assessment of study quality.
Original publication ↗ · DOI: 10.1056/nejmoa2601283