Clinical Trials:
Plakophilin-2 Related Arrhythmogenic Cardiomyopathy (PKP2-ACM)

RP-A601 is being developed for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), also known as ARVC and ARVD, a devastating, inherited heart disease caused by mutations in the PKP2 gene and associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
RP-A601 is an investigational gene therapy consisting of a recombinant adeno-associated serotype rh74 (AAVrh74) capsid containing a functional version of the human PKP2 transgene which is administered as a single intravenous (IV) infusion.
The single-arm, open-label, multi-center Phase 1 study (ClinicalTrials.gov ID: NCT05885412) is evaluating the safety and preliminary efficacy of RP-A601 in adult PKP2-ACM patients with implantable cardioverter defibrillators (ICDs) and overall high risk for arrhythmias. The study assesses the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death.
For details about participating in a Natural History Study for PKP2-ACM, click here.
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