Overview

This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.

Eligibility

Key Inclusion Criteria:

1. Male or female ≥18 years at the time of signing the informed consent
2. Capable and willing to provide signed informed consent
3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
5. Anti-AAVrh.74 capsid neutralizing antibody assay ≤1:40
6. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment

Key Exclusion Criteria:

1. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
2. Previous participation in a study of gene transfer or gene editing
3. Severe Right ventricular (RV) dysfunction
4. Left ventricular ejection fraction by echocardiogram ≤50%
5. New York Heart Association (NYHA) Class IV heart failure.