REGENXBIO Inc., incorporated on July 16, 2008, is a clinical-stage biotechnology company. The Company's gene therapy product candidates are designed 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. The Company develops products candidates across the therapeutic areas of metabolic, neurodegenerative and retinal diseases. Its product candidate, RGX-314, is meant for the treatment of wet age-related macular degeneration (wet AMD). Its product candidate, RGX-501, is meant for the treatment of homozygous familial hypercholesterolemia (HoFH). It is also developing product candidates, such as RGX-111 and RGX-121, to address the neurological symptoms of two severe genetic lysosomal storage diseases, Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II), respectively.
The Company's gene therapy product candidates deliver genes to cells using adeno-associated virus (AAV) vectors, which are non-replicating viral delivery vehicles that are not known to cause disease. Its product candidates utilize viral vectors from its gene delivery platform, which it calls its NAV Technology Platform. Its NAV Technology Platform consists of exclusive rights to AAV7, AAV8, AAV9, AAVrh10 and over 100 other AAV vectors (NAV Vectors). As of December 31, 2016, nine NAV Technology Licensees were developed over 20 product candidates using its NAV Vectors. MPS I is caused by defects in the alpha-l-iduronidase (IDUA) gene and MPS II is caused by defects in the iduronate-2-sulfatase (IDS) gene, both of which encode for enzymes that are responsible for breakdown of cellular waste products. The Company has received investigational new drug application (IND) for RGX-314 for the treatment of wet AMD. It has initiated the enrollment in the Phase I/II clinical trial for RGX-501.
RGX-501 uses the AAV8 vector to deliver the human low-density lipoprotein receptor (LDLR) gene to liver cells. RGX-111 uses the AAV9 vector to deliver the human alpha-l-iduronidase (IDUA) gene to the central nervous system (CNS). It has received orphan drug product designation and rare pediatric disease designation from the Food and Drug Administration (FDA) for RGX-111. RGX-121 uses the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the CNS. It has received orphan drug product designation and rare pediatric disease designation from the FDA for RGX-121. RGX-314 is being developed as a one-time subretinal treatment for wet AMD that includes the NAV AAV8 vector encoding a gene for a monoclonal antibody fragment. The expressed protein is designed to neutralize anti-vascular endothelial growth factor (VEGF) activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation. Its product development pipeline includes treatment candidates for liver-targeted expression of genes.
The Company competes with Abeona Therapeutics Inc., Applied Genetic Technologies Corporation, BioMarin Pharmaceutical Inc., bluebird bio, Inc., Genzyme Corporation, Sangamo BioSciences, Inc., Spark Therapeutics, Inc., uniQure N.V. Roche, Regeneron, Aegerion, Sanofi, Shire and Amgen.
9600 Blackwell Rd Ste 210
The company trades on NASDAQ exchange. The company headquarter is located in Maryland, MD. The company was established in the 2008.| Ticker | RGNX |
| Long Name | REGENXBIO Inc. |
| Short Name | REGENXBIO |