REGENXBIO Inc., a biotechnology company, focuses on the development, commercialization, and licensing of recombinant adeno-associated virus (AAV) gene therapy. The company's development programs include RGX-501, a product candidate for the treatment of homozygous familial hypercholesterolemia, which uses the AAV8 vector to deliver the human low-density lipoprotein receptor gene to liver cells; and RGX-111, a product candidate for the treatment of Mucopolysaccharidosis Type I, which uses the AAV9 vector to deliver the human a-l-iduronidase gene to the central nervous system. Its programs also include RGX-121, a product candidate for the treatment of Mucopolysaccharidosis Type II; RGX-314, a product candidate for the treatment of wet age-related macular degeneration; and RGX-321, a product candidate for the treatment of X-linked retinitis pigmentosa. 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.
Biotechnology Biology Emerging Technologies Molecular Biology Gene Therapy Genetics Medical Genetics Applied Genetics Wet Age Related Macular Degeneration Gene Delivery Retinitis Pigmentosa Adeno Associated Virus Homozygous Familial Hypercholesterolemia Iduronidase X Linked Retinitis Pigmentosa