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uniQure Initiates Phase I in Acute Intermittent Porphyria
Date:12/11/2012

tients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.

About acute intermittent porphyria

Acute Intermittent porphyria (AIP) is a rare genetic disease which is caused by mutations in the porphobilinogen deaminase (PBGD) gene; one of the enzymes of the heme biosynthesis pathway.  Mutations in this gene cause insufficient activity of the protein resulting in partially disruption of heme synthesis.  This in turn leads to accumulation of toxic intermediates (ALA and PBG) giving rise to a wide variety of problems including acute, severe abdominal pains, psychiatric and neurological disorders, and muscular weakness. Acute porphyric attacks can be life-threatening and the long-term consequences include irreversible nerve damage, liver cancer and kidney failure. Currently, the only curative therapy is liver transplantation and thus, new curative options are urgently needed.  Severe AIP patients are suffering poor quality of life with palliative treatments for the different symptoms including glucose or heme infusions for metabolic replacement and inhibition of toxic metabolic production.

About AIPGENE

AIPGENE is a European Commission Framework Programme 7-funded consortium (Grant Agreement number 261506) which was put together with the aim to develop the orphan gene therapy drug AAV5-AAT-PBGD (AMT-021) for the treatment of Acute Intermittent porphyria (AIP). The consortium's objective is to contribute to alleviating the negative impact of this disease on the quality of life of the patients and their families. Overall coordinator of the project is the Centre for Applied Medical Research (CIMA) at the University of Navarra, Pamplona, Spain. Apart from uniQure, other members of the consortium are the Clinical University of Navarra, Pamplona, Spain; Karolinska University Hospital, Stockholm, Sweden; German Cancer Research Center (NCT-DKFZ), Heidelberg, Germany; DIGNA Biotech, Pamplona, Spa
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SOURCE uniQure B.V.
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