RCKT

Rocket Pharmaceuticals, Inc. focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells. The company also has a clinical stage in vitro virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure.