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uniQure Announces Successful Transfection Of Liver Cells With AAV5 And Strong Safety Data From Acute Intermittent Porphyria Clinical Trial


5/27/2014 9:00:58 AM

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AMSTERDAM, The Netherlands, May 27, 2014 (GLOBE NEWSWIRE) -- 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. The results, presented Saturday, May 24, at the 17th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) by Dr. Gloria González-Aseguinolaza from the AIPGENE Consortium also provided evidence that the AAV5 vector from uniQure's bacculovirus production platform is safe by inducing liver cell transfection in AIP patients without liver enzyme perturbations at the tested dose levels.

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