Heart failure & cardiomyopathies
Cardiotropic AAV gene therapy for heart failure: a phase 1 trial
Authors' conclusion
The preliminary assessments of efficacy outcomes showed improvements in the New York Heart Association class and left ventricular ejection fraction in both cohorts and improvements in peak oxygen consumption and 6-min walk test performance in cohort 1.
Study context
- Population
- Nonischemic cardiomyopathy, NYHA class III HF, LVEF 15–35%9 men · 2 women
- Participants
- 6 vs 5
- Comparison
- Cohort 1 vs Cohort 2
- Cohort 1n = 6
- Cohort 2n = 5
- Outcome
- Safety and feasibility of a single antegrade coronary artery infusion of AB-1002
Methodological detailsdose-escalation cohorts
- Dose-escalation cohorts
Main finding
As reported in the abstract
Safety
Death, considered not related to AB-1002
1
Further findings
Preliminary efficacy
- Improvements in NYHA class and LVEF in both cohorts
- Improvements in peak oxygen consumption and 6-min walk test in cohort 1
- Preliminary assessments
Safety
Adverse events
- Adverse events (AEs) or serious AEs attributed to study treatment
- 0
- Death (not treatment-related)
- 1
- Mild, asymptomatic, self-limiting liver enzyme elevations, predominantly in cohort 2
Original publication
Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nat Med. Published 21 October 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →