ROCKET PHARMACEUTICALS, INC.

Based in Cranbury, New Jersey, Rocket Pharmaceuticals is a late-stage biotechnology company developing in vivo gene therapies targeting rare genetic and cardiovascular disorders. The company's pipeline consists of adeno-associated viral (AAV) programs and lentiviral (LV) programs, each designed to address distinct disease mechanisms through gene therapy approaches. Its AAV candidates target Danon disease, a multi-organ lysosomal disorder causing progressive heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inherited cardiac condition characterized by ventricular dysfunction and arrhythmia risk; and BAG3 Dilated Cardiomyopathy. The company's lentiviral programs address Leukocyte Adhesion Deficiency-I, Fanconi Anemia, and Pyruvate Kinase Deficiency, all rare hematologic or immune disorders.

The company operates primarily as a clinical-stage entity focused on advancing its therapeutic candidates through regulatory development. Rocket Pharmaceuticals holds multiple license agreements with academic medical centers and research institutions, including UCL Business PLC, the University of California, Temple University, and REGENXBIO, Inc., which provide access to underlying technology platforms and intellectual property. Founded in 1999 and incorporated in Delaware, the company has established partnerships that support its research and development activities across its gene therapy portfolio.