Rocket Pharmaceuticals IncFRA Rocket Stock Report

Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. The company is headquartered in New York City, New York and currently employs 240 full-time employees. The company went IPO on 2015-02-18. The firm's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe paediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. The firm's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a pediatric heart failure condition. The company also has a preclinical AAV-based gene therapy program in BAG3-associated dilated cardiomyopathy.