RP-A601

RP-A601 is an investigational gene therapy developed for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), a rare, inherited heart disease characterized by arrhythmias and risk of sudden cardiac death. The therapy consists of a recombinant adeno-associated virus serotype rh74 (AAVrh74) vector carrying a functional human PKP2 transgene, administered as a single intravenous infusion. RP-A601 aims to restore normal PKP2 protein expression in cardiac tissue, potentially offering a one-time, curative treatment that improves survival and quality of life for affected patients. Preclinical studies have shown decreased arrhythmias and increased survival in animal models. The drug has received FDA Fast Track and Orphan Drug designations and is currently being evaluated in Phase 1 clinical trials[1][4][5][6][8].

Other names

PKP2-ACM ProgramPKP-2-ACM ProgramPKP 2-ACM ProgramRocket Pharma PKP2 gene therapy