Rocket Pharmaceuticals Inc 정보 회사

Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company focused on the development of gene therapies for rare and devastating diseases. The Company has two clinical stage and one pre-clinical stage in vivo adeno-associated viral (AAV) programs in the United States, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood.