Cardiotropic AAV gene therapy for heart failure: a phase 1 trial.

ABSTRACT: Innovative approaches for the treatment of heart failure are needed beyond conventional medical therapy to reverse ventricular dysfunction and modify the course of the disease. AB-1002, a chimeric cardiotropic adeno-associated viral vector that delivers constitutively active protein phosphatase 1 inhibitor 1 to cardiomyocytes, improves cardiac function in preclinical models of heart failure. Here we carried out a phase 1 study to evaluate the safety and feasibility of a single antegrade coronary artery infusion of AB-1002 in patients with nonischemic cardiomyopathy, New York Heart Association class III heart failure, and a left ventricular ejection fraction of 15-35%. Patients received 3.25 × 10¹³ viral genomes (cohort 1, n = 6) or 1.08 × 10¹⁴ viral genomes (cohort 2,

SUBMITTER: Henry TD 

PROVIDER: S-EPMC12618250 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature