VERVE-102
What it is & what research shows
Reference overviewVERVE-102 is an investigational gene-editing medicine designed to inactivate PCSK9 in liver cells after one infusion. The objective is sustained LDL cholesterol reduction. Heart-2 studied adults with inherited high cholesterol or premature coronary disease. Its early open-label findings support cholesterol lowering, with a published report available below. [1.1][1.2]
A small uncontrolled study cannot establish lifetime safety or prevention of heart attacks. Infusion reactions and transient liver-enzyme elevations were observed. Editing is intended to be durable; it is not equivalent to stopping a tablet or allowing an antibody dose to wear off. A source-linked study result with a verified dose and schedule is not yet available in this overview. This is a gap in this page, not a claim that no research exists. [2.1][2.2]
Sources & reading scope
- [1.1] Heart-2: PCSK9 base editing with VERVE-102Published human study · Open-label early human study; LDL outcomes and adverse events.
- [1.2] Heart-2 trial recordTrial registration · Study design and any posted results; registration does not establish treatment benefit.
- [2.1] Heart-2: PCSK9 base editing with VERVE-102Published human study · Open-label early human study; LDL outcomes and adverse events.
- [2.2] Heart-2 trial recordTrial registration · Study design and any posted results; registration does not establish treatment benefit.
Evidence summarized on PED Evidence
1 completed summaries
1 human-study reports
0 randomized-trial candidates
0 safety-topic reports
Studies & trial records
Original papers, trial registrations and sponsor disclosures are labeled separately. Multiple links may describe the same study.
More about VERVE-102: identity, safety & reference details
Also known as VERVE102
Start with the research
An outcome-specific synthesis has not been prepared for this compound. This is a coverage gap, not evidence that it has no effect.
Open studies and trial records- Uses & research
Heart-2 studied adults with inherited high cholesterol or premature coronary disease. Its early open-label findings support cholesterol lowering, with a published report available below. [1][2]
- Known half-life
No source-verified value in this profile yet. A treatment schedule or duration of an effect is not a drug half-life.
Half-life describes how quickly the measured drug concentration falls by half. It does not set a dosing interval.- Evidence & important limits
A small uncontrolled study cannot establish lifetime safety or prevention of heart attacks. Infusion reactions and transient liver-enzyme elevations were observed. Editing is intended to be durable; it is not equivalent to stopping a tablet or allowing an antibody dose to wear off. [1][2]
Safety & evidence boundaries
A small uncontrolled study cannot establish lifetime safety or prevention of heart attacks. Infusion reactions and transient liver-enzyme elevations were observed. Editing is intended to be durable; it is not equivalent to stopping a tablet or allowing an antibody dose to wear off.
Explore safety questions by system
These are literature filters, not claims that this compound causes each effect. A keyword match is not a confirmed adverse reaction.
Identity, names & formulations
Canonical compound: VERVE-102 · verve102
Searchable names: VERVE102
Names share a parent research page. Different esters, salts, routes and brands are not assumed to have the same half-life, indication or exposure.
Combination evidence stays attached to the studied combination. It is not automatically assigned to each ingredient.
Understand the evidence types
Read the model and design first, then the population, outcome and limitations. These categories describe evidence, not a best-compound ranking.
- Randomized human trials
- Compare assigned interventions. Randomization does not by itself establish low bias or relevance to every population.
- Controlled human studies
- Use a comparison group; allocation and confounding still matter.
- Prospective human research
- Follows participants forward. It may be observational or interventional, so this is not a separate quality grade.
- Observational human research
- Describes associations. Confounding can explain differences.
- Case reports and series
- Useful for unusual events and safety signals, not reliable rates or effect sizes.
- Animal research
- Can test biology and function in a living model. Human exposure, benefit and safety remain separate questions.
- Cell and tissue research
- Tests responses in a preparation, including human-derived cells. It is not a human participant trial.
- Mechanistic and computational evidence
- Helps explain or predict a pathway; predictions need experimental and clinical testing.
- Anecdotal reports
- Self-reported experiences can generate questions. Product identity, selection bias and other interventions may be uncontrolled.
Systematic reviews synthesize studies and depend on their quality. Preprint status and abstract versus full-paper reading are separate labels. Citation popularity measures attention, not reliability. A study summary is not a formal quality appraisal.
Sources for this profile
- 1. Heart-2: PCSK9 base editing with VERVE-102 Published human study · Open-label early human study; LDL outcomes and adverse events.
- 2. Heart-2 trial record Trial registration · Study design and any posted results; registration does not establish treatment benefit.
Reference facts checked 2026-09-24. Source scope is listed above; this is not a formal quality appraisal. See each label for full prescribing information.
Study model describes the source. Confidence depends on methods, replication, population and outcome.
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VERVE-102 study summaries
Human-first reading order. Intervention candidates are distinguished from background mentions in the library.
- In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. · Human · 2026-05-25