BMN 365

BMN 365 is an investigational adeno-associated virus (AAV) gene therapy developed by BioMarin Pharmaceutical. It was designed to deliver a functional copy of the plakophilin-2 (PKP2) gene for the treatment of arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C), specifically targeting patients with PKP2 mutations. The therapy aimed to address the underlying genetic deficiency by enabling cardiac cells to produce functional PKP2 protein, potentially restoring normal heart rhythm and function in affected individuals. Preclinical studies demonstrated proof of concept for this approach; however, development was discontinued before clinical trials commenced[1][4][5][7].