Does PCSK9 base editing (VERVE-102) work?

Read Off Label rates the evidence for PCSK9 base editing (VERVE-102) as Weak and the benefit magnitude as high, producing an overall grade of C- (4.0/10). Verve Therapeutics (Lilly).

Is PCSK9 base editing (VERVE-102) safe?

PCSK9 base editing (VERVE-102) has a med-high risk profile in published human data. Legal status: Investigational (Phase 1b/2; not approved). This is not medical advice — see the disclaimer.

Rankings / Metabolic Health

PCSK9 base editing (VERVE-102)

Metabolic Health · In-vivo base editing

Tier C-

Reviewed by Read Off Label · Last reviewed Jun 7, 2026 · How we grade

gene-editingbase-editingpcsk9cardiovascularfrontierinvestigationalemerging

4 / 10

Tier C-

Ev 3 Bn 8 Sf 3.5

Bottom line

Read Off Label grades PCSK9 base editing (VERVE-102) as C- (4.0/10) based on weak evidence, high benefit magnitude, and a med-high-risk safety profile.

Verve Therapeutics (Lilly).

Typical use: Single IV infusion (0. — Investigational.

What this is

Verve Therapeutics (Lilly). Heart-2 Phase 1b (NEJM 2026, n=35): a single infusion reduced PCSK9 up to 88% and LDL-C up to 62% (max -69%), durable to 18 months, no serious adverse events. First-in-human in-vivo base editing for hypercholesterolemia; Phase 2 planned late 2026. Frontier modality, durability/safety still early. Industry-sponsored.

Mechanism

One-time in-vivo CRISPR base edit of the PCSK9 gene in hepatocytes (lipid-nanoparticle delivered), permanently lowering PCSK9 and LDL-C, a single-dose alternative to chronic PCSK9 therapy.

Dose & route

Single IV infusion (0.3-1.0 mg/kg in Phase 1b)

Citations

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Common questions

Does PCSK9 base editing (VERVE-102) work?: Read Off Label rates the evidence for PCSK9 base editing (VERVE-102) as Weak and the benefit magnitude as high, producing an overall grade of C- (4.0/10). Verve Therapeutics (Lilly).
Is PCSK9 base editing (VERVE-102) safe?: PCSK9 base editing (VERVE-102) has a med-high risk profile in published human data. Legal status: Investigational (Phase 1b/2; not approved). This is not medical advice — see the disclaimer.
What is the typical dose for PCSK9 base editing (VERVE-102)?: Single IV infusion (0.3-1.0 mg/kg in Phase 1b)
How does PCSK9 base editing (VERVE-102) work?: One-time in-vivo CRISPR base edit of the PCSK9 gene in hepatocytes (lipid-nanoparticle delivered), permanently lowering PCSK9 and LDL-C, a single-dose alternative to chronic PCSK9 therapy.

This is an independent synthesis of published research by a non-clinician. Scores are opinions supported by citations, not prescriptions. See the full disclaimer and methodology for how this score was produced and what it does and doesn't mean.