In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia
Authors' conclusion
One dose of VERVE-102 led to dose-dependent, substantial, and sustained reductions in PCSK9 and LDL cholesterol levels.
Study context
- Population
- Adults with heterozygous familial hypercholesterolemia or premature CAD≥28 days of follow-up
- Participants
- 35 adults
- Comparison
- One IV infusion of VERVE-102 at one of six doses
- Outcome
- Safety and changes in blood PCSK9 protein and LDL-C levels
Methodological detailsopen-label, six dose cohorts
- Open-label
- Six dose cohorts
Main finding
As reported in the abstract
PCSK9
LDL-C
| Outcome | 0.3 mg per kilogram | 1.0 mg per kilogram |
|---|---|---|
| LDL-C absolute reduction | — not stated in the abstract | 78 mg per deciliter |
— not stated in the abstract
Safety
Dose-limiting toxic effects
- No dose-limiting toxic effects
Adverse events
- Aspiration pneumonitis in a participant with gastroesophageal reflux disease
- 1
- Mild-to-moderate infusion-related reactions
- Transient elevations in alanine aminotransferase levels
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. N Engl J Med. Published 25 May 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →