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ReGenX Biosciences, LLC (RGNX)

750 17th St. NW, Suite 1100
Washington D.C., DC, USA

Category: Drug Delivery

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Maryland Biotech Industry Map

ReGenX Biosciences, LLC (RGNX) is a biopharmaceutical company developing novel therapeutics and research tools based on adeno-associated viral vectors, or AAV vectors. Its mission is to be an innovator in the development of novel therapeutics using AAV vectors to treat diseases with unmet medical needs; to create a platform of novel products and services using AAV vectors to enable pioneering basic pharmaceutical drug discovery and research and to become the leader in the production of high quality AAV vectors for all applications. The company was formed in 2009 as a partnership between Dr. Wilson, the University of Pennsylvania, and GlaxoSmithKline, to develop and commercialize therapeutics and life science research products utilizing Dr. Wilson's proprietary, novel AAV vectors for delivery of genetic material and related technologies.

ReGenX's technology and capabilities can enhance existing products or create entirely new products in a broad range of areas including: Cell therapy (including stem cells), Gene therapy, Gene modulation (e.g., RNAi and microRNA), Genetic vaccines and Protein therapy (e.g., monoclonal antibodies, cytokines). Its AAV vector technology enables the efficient, rapid, safe and specific delivery of genetic material into cells. Once delivered into a cell, the genetic material can use the existing cellular machinery to produce specific proteins encoded by the genetic material. This process may be used to treat disease by initiating normal protein production or gene regulation in cells that may have defects in their endogenous function. Delivery of genetic material may also enable cells to produce more of a certain protein or different proteins than they would normally produce, thereby treating a disease state.

ReGenX is engaged in reagent services, consulting services and research licenses. Its AAV vector applications include: Molecular Therapy, Target Validation, Creating Disease Models, GWAS etc. The company's AAV7 is targeted against Ocular diseases including: Age-Related Macular Degeneration and Leber Congenital Amaurosis; AAV8 is targeted against Metabolic disorders including: Familial Hypercholesteremia, Hurler Syndrome (MPS I), Maroteaux-Lamy Syndrome (MPS VI), Gaucher's disease and Retinitis Pigmentosa - an Ocular disease and AAV9 is targeted against Neurodegenerative disorders including: Amyotrophic Lateral Sclerosis, Parkinson's disease and Alzheimer's disease.