QURE - uniQure completes patient enrollment for phase I/II trial of AMT-130 for Huntington's disease
uniQure (NASDAQ:QURE) completed patient enrollment in the first two cohorts of Phase I/II trial of its therapy, AMT-130, to treat early stage Huntington’s disease. The early-to-mid stage study includes two dose cohorts of 26 patients randomized to either treatment with the company's therapy or to an imitation surgical procedure. The first dose cohort comprised of 10 patients, of which six patients received treatment with AMT-130 and four patients received imitation surgery, while the second dose cohort included 16 patients, of which ten patients received AMT-130 and six patients received imitation surgery. The company expects to provide clinical update in Q2 from the interim analysis in the first cohort. AMT-130 comprises a recombinant AAV5 vector carrying a DNA cassette encoding a microRNA that lowers Huntingtin protein in patients. Press Release
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uniQure completes patient enrollment for phase I/II trial of AMT-130 for Huntington’s disease