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May 27, 2014
05:53 EDTQUREuniQure announces successful transfection of liver cells with AAV5
uniQure announced successful transfection of liver cells with the porphobilinogen deaminase gene, or PBGD, from an ongoing Acute Intermittent Porphyria, or AIP, dose-escalation Phase I trial conducted in collaboration with the AIPGENE Consortium, a pan-European collaboration. The results 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. AMT-021 aims to provide long-term normalization of the PBGD protein in order to prevent acute AIP attacks and the associated complications. The ongoing Phase I trial, conducted by Digna Biotech as part of the AIPGENE Consortium, has enrolled eight patients with severe AIP. The study's primary objective is to assess the safety of systemic administration and to determine the maximum tolerated dose of AMT-021. Secondary objectives include exploring urinary levels of toxic metabolites to determine their adequacy as biomarkers of treatment effect. uniQure received orphan drug designation for AMT-021 in 2009 from the European Medicines Agency.
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