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American Gene Technologies Granted FDA Orphan Drug Designation For Phenylketonuria | American Gene Technology

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American Gene Technologies (AGT), a leading gene and cell therapy company in the discovery and development of lentiviral vector based therapeutics, announces that the U.S. Food and Drug Administration (FDA) received orphan-drug designation #DRU-2018-6572 for the treatment of phenylketonuria (PKU) using its proprietary, lentiviral vector based technology. PKU is a debilitating inherited disease affecting 1 in 13,500 children born in the U.S. Mandatory screening identifies affected children who can be supported with a strict synthetic diet that does not cure disease. If successful, AGT’s lentiviral vector approach will provide a permanent cure, improving quality of life for more than 16,000 people living with PKU in the U.S. alone.

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REGENXBIO Announces Exclusive Worldwide License with Ultragenyx for the Treatment of CDKL5 Deficiency Disorder Using NAV Vectors

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REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that Ultragenyx Pharmaceutical Inc. has exercised an option for an exclusive worldwide license to REGENXBIO’s NAV Vectors, including NAV AAV9, for the treatment of CDKL5 Deficiency Disorder (CDD).

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