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uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

Excerpt from the Press Release:

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 2021 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the independent Data Safety Monitoring Board (DSMB) overseeing the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease has met and reviewed safety data for the fully enrolled first cohort of ten patients. This data set included nine-month safety data from the first two enrolled patients, six-month safety data from the next two enrolled patients, and 30-day safety data on remaining six patients in the trial. The DSMB recommended continued dosing in the study, and uniQure will now begin to enroll patients in the higher-dose cohort of the trial. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, six patients have been treated with AMT-130, and four patients received the imitation surgery.

“We are very pleased with the progress of the study and with the recommendations of the DSMB. We look forward to proceeding with dose-escalation in the second cohort of patients,” said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “We remain highly focused on progressing this therapy given the significant unmet need in the Huntington’s disease community and are committed to initiating clinical development of AMT-130 in Europe in the second half of this year and to sharing initial imaging and biomarker data from the U.S. clinical trial before the end of the year.”

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