Overview
- An interim analysis published this week showed a single intravenous dose of VERVE‑102 produced dose‑dependent PCSK9 protein drops and a mean LDL‑C fall of up to 62% at the highest 1.0 mg/kg dose in 35 participants.
- VERVE‑102 uses mRNA in targeted lipid nanoparticles to deliver an adenine base editor that alters a single base in the liver PCSK9 gene so the gene is effectively inactivated after one infusion.
- No treatment‑related serious adverse events or dose‑limiting toxicities were reported in the interim data, although investigators recorded mild‑to‑moderate infusion reactions and transient liver enzyme elevations and one case of aspiration pneumonitis in a patient with pre‑existing GERD.
- The FDA has granted VERVE‑102 Fast Track designation and Eli Lilly, which acquired Verve, plans a larger Phase 2 trial of roughly 200 people and long‑term monitoring of participants.
- Experts caution the findings are preliminary because the report covers only 35 people with limited follow‑up at the highest dose, so durability, off‑target editing and rare long‑term risks must be settled in larger, longer studies before clinical use or broad access can be considered.