Genetics and gene editing trials

3 studies from 2006 to 2026. Results: 3 safety, side effects or genetics. Every summary comes from the trial's PubMed abstract.

All 3 studies

• Heart-22026 · VERVE-102 · 35 people

  A single intravenous dose of the base-editing drug VERVE-102 produced dose-dependent, durable reductions in PCSK9 and LDL cholesterol, with an acceptable early safety profile, in a small phase 1 trial.

  Early safety/biomarker dataGenetics and gene editingInvestigational

• Ference 20162016 · PCSK9/HMGCR variants · 112,772 people

  Genetic mimicry shows that lowering LDL cholesterol via PCSK9 variants protects against cardiovascular events about as much as HMGCR (statin-target) variants, but both are linked to a smaller, additive increase in diabetes risk.

  Supports LDL causalityGenetics and gene editingNot applicable

• Cohen 20062006 · PCSK9 variants · 12,887 people

  People who naturally carry LDL-lowering PCSK9 gene variants from birth have a dramatically lower lifetime risk of coronary heart disease, supporting that lowering LDL-C, even modestly, over a lifetime substantially cuts cardiovascular risk.

  Supports LDL causalityGenetics and gene editingNot applicable

Other medicines

• Statins
• Ezetimibe and bile acid binders
• Bempedoic acid
• PCSK9 inhibitors
• Lp(a) medicines
• CETP inhibitors
• Fibrates, omega-3 and niacin
• APOC3, ANGPTL3 and lomitapide
• Meta-analyses

Search and filter all 106 studies in the trial database. Education only, not medical advice.