VERVE-102 Heart-2 Cohort 3 Data (HeFH)
Cohort 3 dose-escalation data for Verve’s in-vivo base-editing therapy targeting PCSK9 in heterozygous familial hypercholesterolemia (HeFH). Watch: PCSK9 protein knockdown %, LDL-C reduction, hepatic and immunogenicity safety events.
What’s at stake
VERVE-102 is Verve Therapeutics' in vivo base-editing therapy designed to provide a one-time, permanent reduction in LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH) — a dominant genetic disorder affecting 1 in 250 people that causes severely elevated LDL from birth. A single IV infusion delivers an adenine base editor via lipid nanoparticles to liver cells, converting a single DNA letter in the PCSK9 gene to a permanent loss-of-function variant that prevents the liver from degrading LDL receptors. The goal is lasting LDL-C reduction from one infusion, as an alternative to lifelong statin therapy and bi-weekly PCSK9 inhibitor injections.
No primer in glossary yet.
- PCSK9targetProprotein Convertase Subtilisin/Kexin type 9
Liver enzyme that degrades LDL receptors. Inhibiting it (mAbs, siRNA, base-editing) lowers LDL-C dramatically.
- LDL-CendpointLow-density lipoprotein cholesterol
"Bad" cholesterol. FcRn inhibitors are known to elevate LDL by 20-40%, which matters for chronic dosing in patients with cardiovascular risk.
How Ph2 readouts in Cardio-Renal have landed.
Reference, not prediction. We surface the historical record so you can read it yourself. We never extrapolate to the upcoming event.
Competitive landscape
| Drug | Company | Modality | Mechanism | Phase | Next catalyst |
|---|---|---|---|---|---|
| AMVUTTRAvutrisiran | ALNY | siRNA | TTR-targeting RNAi | PDUFA · Dec 26 | |
| plozasiran | ARWR | siRNA | APOC3-targeting RNAi | CONFERENCE · May 26 | |
| olezarsen | IONS | oligonucleotide | ApoCIII antisense oligonucleotide | PDUFA · Dec 26 | |
| XPHOZAHtenapanor | ARDX | small molecule | NHE3 inhibitor | CMC · Jul 26 |
Disclosure trail
- Apr 15, 2026·15d agopinned · highest confidenceHIGH confPRQTRtop claimQ4'26
“VERVE-102 Heart-2 Cohort 3 dose-escalation data in patients with heterozygous familial hypercholesterolemia is expected in the fourth quarter of 2026.”
conf 85%via llm