QURE - uniQure to enroll more patients in phase 1/2 trial for Huntington's disease

uniQure N.V. (NASDAQ:QURE) said no significant safety concerns were seen in the first four patients enrolled in higher-dose group of its phase 1/2 trial of AMT-130 to treat Huntington’s disease, a rare inherited disorder causing degeneration of nerve cells in the brain. As a result the independent Data Safety Monitoring Board recommended that final 12 patients in the second cohort were now cleared for enrollment. In the study to date, eight patients have been treated with AMT-130, and six patients have received imitation surgery. Full enrollment of the second, higher-dose cohort is expected to be completed in mid-2022. The company plans to share preliminary imaging and biomarker data from the initial four patients in the U.S. trial before the end of the year. The European, open-label Phase 1b/2 study of AMT-130 will enroll 15 patients with early manifest Huntington’s disease across two dose groups. Together with the U.S. study, the European study

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uniQure to enroll more patients in phase 1/2 trial for Huntington’s disease