CURRENT STATUS
Cardiology is shifting from chronic risk-factor management toward durable, mechanism-level cures. In-vivo base editing of PCSK9 (Verve's VERVE-102) has shown dose-dependent LDL lowering in first-in-human dosing: a potential one-and-done cholesterol treatment. The first Lp(a)-lowering agents (pelacarsen, olpasiran) are in Phase 3 outcomes trials, finally addressing a genetically driven risk factor with no prior therapy. AAV gene therapies for genetic cardiomyopathy (MYBPC3, PKP2) are dosing patients, AI-ECG already flags asymptomatic left-ventricular dysfunction from a standard tracing, and direct cardiac reprogramming is converting scar fibroblasts into cardiomyocytes in large-animal models. Guideline-directed medical therapy plus devices remain the standard-of-care bridge while these mature.
KEY BREAKTHROUGHS
Verve Therapeutics VERVE-102: in vivo base editing to permanently lower cholesterol in one dose
AI-ECG detecting atrial fibrillation, heart failure, and valve disease from standard 12-lead ECGs
CRISPR knockout of PCSK9 in non-human primates showing durable 60% LDL reduction
Cardiac reprogramming: converting scar fibroblasts back into beating cardiomyocytes
SI-COMPRESSED PIPELINE
SI TOOLS ACCELERATING CURES
KEY ORGANIZATIONS
KEY CLINICAL TRIALS
HEART-2: VERVE-102 In Vivo Base Editing of PCSK9 (Phase 1b)
ViewVerve Therapeutics (Eli Lilly)
A single intravenous infusion of a base editor that permanently switches off the PCSK9 gene in the liver: a potential one-and-done treatment for stubbornly high cholesterol. Reported dose-dependent LDL reductions up to 62%, with mean PCSK9 knockdown of 51–88%. A Phase 2 trial is planned.
TIMELINE ESTIMATE
One-shot cholesterol cure: 3–5 years. Heart-failure / cardiomyopathy gene therapy: 4–6 years. Cardiac regeneration: 8–12 years.