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Public evidence
Homo sapiensPCSK9

Do the public VERVE-102 results establish editing efficiency, LDL lowering, or prevention of cardiovascular events?

Change

One administration of investigational VERVE-102 targeting liver PCSK9.

Measured

Circulating PCSK9, LDL cholesterol and safety observations.

Observed outcome

In the VERVE-102 1.0 mg/kg total-RNA cohort, mean circulating PCSK9 fell 88% from baseline.

Source 1
System
Homo sapiens · Adults with heterozygous familial hypercholesterolemia or premature coronary artery disease
Setting
Open-label phase 1, single-ascending-dose study; 35 participants across six cohorts, each with at least 28 days of follow-up.
Comparison
Change from participant baseline; no randomized control arm described.

What was observed

Checked findings go into your research brief.

5
ObservedSource 1

In the VERVE-102 1.0 mg/kg total-RNA cohort, mean circulating PCSK9 fell 88% from baseline.

Abstract: Results, dose-dependent mean reductions

Measurement details
Reported value
88 % mean reduction from baseline
Group
Highest dose cohort (1.0 mg/kg total RNA)
ObservedSource 1

In the VERVE-102 1.0 mg/kg total-RNA cohort, mean LDL cholesterol fell 62% from baseline, an absolute reduction of 78 mg/dL.

Abstract: Results, corresponding LDL cholesterol reductions

Measurement details
Reported value
62 % mean reduction from baseline
Group
Highest dose cohort (1.0 mg/kg total RNA)
Absolute change
-78 mg/dL
ObservedSource 1

In the 35-participant VERVE-102 phase 1 study, no dose-limiting toxic effects were reported; infusion reactions and transient ALT elevations occurred.

Abstract: Results, safety observations

ObservedSource 1

In the VERVE-102 phase 1 study, 15 of 35 treated participants had at least one year of follow-up.

Abstract: Results, follow-up duration

Measurement details
Reported value
15 participants with at least 1 year follow-up
Group
All treated participants
Denominator
35
ObservedSource 2

Lilly reported VERVE-102 biomarker durability up to 18 months; this is the maximum reported follow-up, not follow-up for every participant.

Trial summary and durability statement

What this supports

Interpretation
  • Reported PCSK9 and LDL changes support biological activity in the studied participants.
What this does not establish
  • Blood protein reduction is not a measured percentage of edited hepatocytes.
  • These results do not establish prevention of cardiovascular events.
Gene location & edit specificity

No exact edit interval established by this evidence review.

Same gene, different setting

Compare the contexts before carrying a finding across them.

PCSK9: human genetics supports the targetHomo sapiens · ARIC study participants; published population groups

Sources

Reviewed Sep 26, 2026
1In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia2026 · Peer-reviewed clinical study
Reviewed
Abstract only
Sections
Abstract: Methods · Abstract: Results · Abstract: Conclusions
DOI
10.1056/NEJMoa2601283
A total of 35 participants across the six dose cohorts received VERVE-102
Abstract: Results
Read the source
2Lilly: VERVE-102 Heart-2 interim results, May 25, 20262026 · Sponsor announcement
Reviewed
Selected announcement sections
Sections
Trial summary · Reported dose-group LDL-C changes · Durability statement
Read the source