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uniQure Announces Positive Topline Results from Phase I/II Trial in Sanfilippo B Syndrome Patients Using AAV5-Based Gene Therapy


uniQure N.V. (Nasdaq: QURE) today announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur (Biotherapies for Neurodegenerative Diseases Unit, Institut Pasteur/INSERM) in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales (collectively "the consortium") in four Sanfilippo B syndrome (MPSIIIB) patients treated with a novel gene therapy, AMT-110. In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid (CSF) from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months. These results validate the effective transmission of the NAGLU gene with the AAV5 vector. The trial demonstrated that incremental cognitive development was maintained in all four patients, aged 20 to 53 months at study onset. The therapy consists of uniQure's proprietary AAV5

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