VERVE-102: how it works, status and trials
Phase 2Gene editingLowering LDL cholesterol
VERVE-102 is an investigational one-time infusion that edits a single letter of DNA to switch off the PCSK9 gene in the liver. It is in phase 2 trials and is not approved.
In technical terms: A single-letter DNA edit permanently switches off the PCSK9 gene.
The highlighted badge marks where VERVE-102 acts. Open the full map or compare every therapy.
At a glance
- Acts on
- PCSK9 gene · liver
- How it is given
- Base-editor mRNA + guide RNA in lipid nanoparticle · single IV infusion
- What the main trial found
- PCSK9 −88%, LDL-C −62% at 1.0 mg/kg (phase 1b)
- Status (curated October 25, 2026)
- Phase 2
Where it fits in the body
The LDL-receptor cycle
LDL particles are cleared from blood when they bind LDL receptors on liver cells. After the particle is taken in, a receptor normally returns to the surface and can be reused many times. PCSK9 escorts the receptor to the lysosome, where it is destroyed. Every PCSK9-pathway drug (5–10) works by keeping more receptors on the surface.
RNA and gene-editing therapies
These drugs act before a protein is made. siRNA and antisense drugs are attached to GalNAc, which hepatocytes take up, and they destroy one specific mRNA. A dose lasts weeks to months. Gene editors (10, 13) make a permanent single-dose change to the DNA and are in early trials.
Trials and registry status
From ClinicalTrials.gov, PubMed and openFDA, refreshed monthly. Last run October 27, 2026.
VERVE-102 from Eli Lilly (Verve)
Stage: Phase 2. 2 active of 2 registered trials. 1 new papers in the last 31 days.
- HEART-3 (NCT07839936): not yet recruiting, primary completion 2028-08
Evidence
- Phase 1b, NEJM 2026 (PubMed 42187087)
Common questions
How does VERVE-102 work?
VERVE-102 is an investigational one-time infusion that edits a single letter of DNA to switch off the PCSK9 gene in the liver. It is in phase 2 trials and is not approved. In technical terms: A single-letter DNA edit permanently switches off the PCSK9 gene. It acts on PCSK9 gene · liver.
How much does VERVE-102 lower cholesterol or other lipids?
PCSK9 −88%, LDL-C −62% at 1.0 mg/kg (phase 1b). Individual results differ, and a clinician can say what to expect for one person.
Is VERVE-102 approved, and what is the status?
Phase 2 (curated October 25, 2026). Registry stage as of October 27, 2026: VERVE-102, Phase 2.
How is VERVE-102 taken?
Base-editor mRNA + guide RNA in lipid nanoparticle · single IV infusion.
Read more
Related therapies
- Statins: Approved (generic)
- Bempedoic acid (Nexletol, Nexlizet): Approved 2020
- Ezetimibe (Zetia): Approved (generic)
- Bile acid sequestrants (colesevelam): Approved (generic)
Educational, not medical advice. Mechanism and status text curated October 25, 2026; registry data refreshed October 27, 2026. Registries can lag company announcements. Effects are placebo-adjusted results from the main published trial, not head-to-head comparisons. Investigational agents are available only in clinical trials. Preval Health does not practice medicine; licensed clinicians in a partner network provide care. If you have chest pain or signs of a stroke, call 911.