QURE - uniQure provides update on Phase I/II AMT-130 gene therapy trial for Huntington's disease

uniQure (NASDAQ:QURE) announces initial observations on the first four patients enrolled in the lower-dose cohort of its Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. Shares up 2.5% premarket at $29.47. Two of the four enrolled patients received AMT-130, and two patients experienced an imitation (sham) surgery. Neurofilament light chain, a key biomarker of injury in the brain, trended downward and returned to baseline levels in the treated patients, and structural MRI data are consistent with the safety profile of AMT-130. AMT-130 appears to be generally well tolerated both immediately after surgical administration and at one year of follow up in these four patients. Structural MRI did not reveal any clinically meaningful safety findings in either treated or control patients at one year of follow-up. The safety profile of AMT-130 in the low-dose cohort supports continued enrollment of patients in the higher-dose cohort of 6x1013 vg. Full safety and

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uniQure provides update on Phase I/II AMT-130 gene therapy trial for Huntington’s disease