RCKT vs RGNX

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 pediatric diseases. It has four 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; pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia; and infantile malignant osteopetrosis, a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. 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. The company also has a research and collaboration agreement with Lund University; and strategic collaboration agreement with Stanford University School of Medicine. Rocket Pharmaceuticals, Inc. is headquartered in Cranbury, New Jersey.

REGENXBIO Inc.

REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase I/IIa clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II; RGX-111, which is in Phase I clinical trial for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease; and RGX-501, which is in Phase I/II clinical trials to treat homozygous familial hypercholesterolemia. REGENXBIO Inc. also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop vectorized antibodies for the treatment of neurodegenerative diseases. The company was formerly known as ReGenX Biosciences, LLC and changed its name to REGENXBIO Inc. in September 2014. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.