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uniQure Announces Successful Transfection of Liver Cells with AAV5 and Strong Safety Data from Acute Intermittent Porphyria Clinical Trial

May 27, 2014

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uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced successful transfection of liver cells with the porphobilinogen deaminase gene (PBGD) from an ongoing Acute Intermittent Porphyria (AIP) dose-escalation Phase I trial conducted in collaboration with the AIPGENE Consortium, a pan-European collaboration.