Rocket Pharmaceuticals Inc. is a late-stage biotechnology company focused on developing gene therapies for rare and devastating pediatric diseases. The company employs a multi-platform approach utilizing both adeno-associated viral (AAV) and lentiviral vector (LV) technologies to address genetic disorders with significant unmet medical needs. Its pipeline includes investigational therapies targeting conditions such as Danon disease, Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and arrhythmogenic cardiomyopathy. The company has established partnerships with leading academic institutions, children's hospitals, and research centers worldwide to advance its clinical programs. Rocket maintains strategic collaborations with organizations including Children's Hospital of Philadelphia, Fred Hutchinson Cancer Research Center, and multiple universities to support the development and validation of its gene therapy candidates. Headquartered in Cranbury, New Jersey, and founded in 2015, Rocket Pharmaceuticals operates with a mission to seek gene therapy cures that address the root causes of genetic diseases, aiming to transform treatment paradigms for rare pediatric conditions.