Rocket Pharmaceuticals is a clinical-stage biotechnology company advancing a pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a pediatric heart failure condition. Rocket's clinical programs using lentiviral vector (LV)-based gene therapy are for the treatment of Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD).