Clinical Trials:


hand print and women smiling PKP2-ACM

RP-A601 is being developed for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), 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.

The Phase 1 pivotal trial for RP-A601 is currently recruiting patients. The trial is evaluating the safety and preliminary efficacy of RP-A601 in at least six adult PKP2-ACM patients with implantable cardioverter defibrillators (ICDs) and overall high risk for severe 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 more details about the trial, visit

For more information about PKP2-ACM, visit our Patients and Community Page

To read our Expanded Access statement, click here.

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