RP-A602

RP-A602 is an investigational gene therapy being developed by Rocket Pharmaceuticals for the treatment of arrhythmogenic cardiomyopathy caused by pathogenic variants in the plakophilin-2 (PKP2) gene. It uses an adeno-associated virus (AAV) vector to deliver a functional copy of PKP2 to cardiomyocytes, aiming to restore desmosomal integrity, improve cardiac electrical and mechanical function, and reduce the risk of ventricular arrhythmias and heart failure progression in affected patients. As a one-time, targeted genetic medicine, RP-A602 is designed for systemic administration with cardiac tropism and is in early-stage clinical development, including in patients with PKP2-associated arrhythmogenic cardiomyopathy.