Rocket Azione

Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company. The Company 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. It 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.