QURE - uniQure gains over 15% after trial data for Huntington's disease therapy

uniQure N.V. (NASDAQ:QURE) added more than 15% after the Dutch biotech announced data from a low-dose cohort of its ongoing U.S.-based Phase I/II clinical trial for gene therapy AMT-130 in the treatment of Huntington’s disease. Out of the 10 patients enrolled in the randomized, blinded study, four patients received an imitation surgical procedure while six received the experimental therapy. Highlights of the findings include an increase of disease biomarker Neurofilament Light Chain (NfL) in the cerebrospinal fluid after the AMT-130 surgical procedure, and the NfL level reached the baseline at 12 months. Mean CSF NfL peaked in six patients one month after the surgical procedure, and thereafter declined to stand 8% higher than the baseline while the mean biomarker level remained constant or slightly dropped in the four control patients. Additionally, the findings at 12 months indicate ~54% mean reduction of mutant HTT (mHTT) in CSF of four evaluable patients who received

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uniQure gains over 15% after trial data for Huntington’s disease therapy