VERVE-102 is a one-time injection that delivers molecular tools to liver cells to make a precise DNA change in the PCSK9 gene, which stops the production of a protein that raises LDL cholesterol.
Evidence from Studies
No evidence studies found yet.
What Would Prove This
Per GRADE and EBM methodology, here is what ideal scientific evidence would look like to definitively prove or disprove this claim, ordered from strongest to weakest.
Whether GalNAc-LNP delivery of adenine base editors to hepatocytes consistently achieves targeted PCSK9 editing and protein knockdown across multiple platforms.
A systematic review and meta-analysis of all preclinical and clinical studies using GalNAc-LNP to deliver adenine base editors targeting PCSK9, comparing editing efficiency, protein reduction, and off-target rates across delivery systems.
Whether VERVE-102 achieves higher PCSK9 editing efficiency than a control LNP without base editor in humans.
A double-blind RCT of 60 adults with HeFH randomized 1:1 to VERVE-102 or a control LNP containing non-targeting mRNA, with primary endpoint of PCSK9 editing efficiency by deep sequencing of liver biopsy samples at 4 weeks.
Whether the level of PCSK9 editing achieved by VERVE-102 correlates with the degree of LDL-C reduction in individuals over time.
A prospective cohort study of 100 patients receiving VERVE-102, measuring PCSK9 editing efficiency in serial liver biopsies and correlating it with serum PCSK9 and LDL-C levels over 12 months.
Whether the concentration of base editor mRNA in plasma after VERVE-102 infusion correlates with hepatocyte uptake and editing efficiency.
A cross-sectional analysis of 50 patients receiving VERVE-102, measuring plasma mRNA levels at 1, 6, and 24 hours post-infusion and correlating them with editing efficiency in paired liver biopsies.
Whether rare individuals show unexpected patterns of base editing (e.g., off-target edits, incomplete editing) after VERVE-102 administration.
Detailed reporting of 5–10 individuals who received VERVE-102 and underwent whole-genome sequencing to identify off-target edits or incomplete editing at the PCSK9 locus.