Rocket Pharmaceuticals

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 (CIEMAT), Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; CIEMAT and UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. is headquartered in Cranbury, New Jersey.

Rocket Pharmaceuticals’ mission is to improve the lives of those affected by rare diseases by advancing gene therapy treatments through research, development, and commercialization.

Our vision is to become the leading gene therapy research and development company in the world, advancing treatments for rare diseases and helping to cure them for patients around the world.

Rocket Pharmaceuticals has won prestigious awards for its innovation and achievements in gene therapy development, including the 2020 Breakthrough Award from the International Society of Gene & Cell Therapy.