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.
Male or female ≥18 years at the time of signing the informed consent
Capable and willing to provide signed informed consent
Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
Anti-AAVrh.74 capsid neutralizing antibody titer of >1:40
Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
Previous participation in a study of gene transfer or gene editing
Severe Right Ventricular (RV) dysfunction