LDL-C: 62% reduction within arm · Time-averaged from day 28 through each participant’s last follow-up
1.0 mg/kg, single dose
Adults with heterozygous familial hypercholesterolemia or premature coronary artery disease
Open-label, uncontrolled dose escalation; mean of participant time-averaged responses from day 28 onward.
Safety context
Infusion reactions and transient ALT elevations occurred; no dose-limiting toxicity. One aspiration-pneumonitis serious event was judged unrelated.
Limitations: Seven participants received this dose. This is early biomarker evidence, without a randomized comparator or demonstrated cardiovascular benefit.
Status in this atlas: Current approval status is not summarized in this therapy record. A trial phase or a brand name does not establish approval for this use.
Designed to edit the PCSK9 gene in liver cells, reducing PCSK9 production.
Uses, doses and availability depend on the formulation, indication and country. Study doses below describe research; they are not prescribing instructions.
The molecule
A delivery system containing messenger RNA encoding an adenine base editor and a guide RNA directed at PCSK9, packaged in a GalNAc lipid nanoparticle.
Structure reference
VERVE-102
A delivery system containing messenger RNA encoding an adenine base editor and a guide RNA directed at PCSK9, packaged in a GalNAc lipid nanoparticle.
Shown: Multicomponent RNA and lipid-nanoparticle formulation.
This is a multicomponent gene-editing formulation, not one small molecule. The source describes components and delivery architecture; no exact whole-formulation atomic structure has been verified. No generic DNA helix is presented as the drug.
Selected sourced milestones, not a complete development history. Dates distinguish study starts, publications, announcements and approvals; the first listed date is not necessarily when development began.
Announcement
First Heart-2 participant dosed
Verve announced that the first participant had received VERVE-102 in Heart-2, an early clinical trial in adults with HeFH or premature coronary artery disease. This date records the dosing announcement rather than an inferred exact study-start date.
Verve reported early results in 14 participants, including dose-dependent PCSK9 and LDL-C lowering after one infusion. These were sponsor-reported interim findings from small dose cohorts.
The phase 1 report included 35 treated participants across six dose cohorts and described sustained PCSK9 and LDL-C reductions, with at least one year of follow-up in 15 participants. This is the plotted trial; its small, open-label design cannot establish cardiovascular-event benefit or long-term safety.
First public registration of phase 2 HEART-3 (NCT07839936): approximately 175 participants, not yet recruiting in the checked record. LDL-C at day 90 is primary and ApoB is secondary. The October 2026 start and August 2028 primary completion are registry estimates, not confirmed dosing or results dates. No cardiovascular-event benefit is established.