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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