Posted by Michael Wonder on 20 Jul 2025
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-A601 gene therapy for PKP2 arhythmogenic cardiomyopathy
17 July 2025 - -- Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to RP-A601, the Company’s investigational adenovirus associated virus based gene therapy for the treatment of PKP2-arrhythmogenic cardiomyopathy, a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death.
RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A601 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.
