Rocket Pharmaceuticals

Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing gene therapies for rare and severe pediatric diseases. The company is advancing several clinical-stage programs using lentiviral vectors to address conditions such as Fanconi anemia, which leads to bone marrow failure, and leukocyte adhesion deficiency-I, a disorder impacting immune function. Additionally, Rocket is exploring therapies for pyruvate kinase deficiency, a rare blood disorder, and infantile malignant osteopetrosis, characterized by excessive bone density. The company is also developing an adeno-associated virus-based gene therapy targeting Danon disease, a multi-organ condition associated with early heart failure. Rocket Pharmaceuticals has formed various collaborative agreements with prominent research institutions, including Fred Hutchinson Cancer Research Center and Stanford University School of Medicine, to further its research and development efforts. Headquartered in New York, the company is focused on creating innovative treatment options for underserved medical needs.