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 Cranbury, New Jersey and currently employs 268 full-time employees. The company went IPO on 2015-02-18. The firm is advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Its lentiviral (LV) vector-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. The company also has two clinical stages and one pre-clinical stage in vivo adeno-associated virus (AAV) programs, including programs for Danon disease, Plakophilin-2 Arrhythmogenic Cardiomyopathy and BAG3 Dilated Cardiomyopathy.