BioCentury
ARTICLE | Clinical News

One-year follow up for uniQure's liver gene therapy

October 28, 2014 3:01 AM UTC

uniQure N.V. (NASDAQ:QURE) reported top-line one-year follow-up data from a completed Phase I study in eight acute intermittent porphyria (AIP) patients receiving varying doses of the company's AMT-021, a recombinant adeno-associated viral (AAV) vector containing the porphobilinogen deaminase gene.

Patients received a single IV injection of up to 2x10^13 genetic components/kg AMT-021. Liver biopsies obtained at one year showed the presence of porphobilinogen deaminase DNA in liver cells. No safety concerns were reported. Similar results were reported at six months. ...