Rocket Pharmaceuticals Inc

Rocket Pharmaceuticals Inc. together with its subsidiaries operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease a multi-organ lysosomal-associated disorder leading to early death due to heart failure. It has license agreements with Fred Hutchinson Cancer Research Center; Centro de Investigaciones Energéticas Medioambientales y Tecnológicas Centro de Investigacion Biomedica En Red and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO Inc. The company is headquartered in Cranbury New Jersey.