RCKT

Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders. The Company's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a 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 Company's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a pediatric heart failure condition. It also has a preclinical AAV-based gene therapy program in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM).