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uniQure Announces Clinical Update on First Patients in Phase I / II Clinical Trial of AMT - 130 Gene Therapy for the Treatment of Huntington's Disease tools.eurolandir.com/tools/Pressreleases/GetPress Release / - Treatment was well tolerated with no significant safety issues related to AMT - 130 in first two treated patients through one year of follow - up ~ - Neurofilament Light Chain ( NfL ) rose as expected immediately following surgery and returned to baseline in treated patients ~ - A total of 19 patient procedures have been performed in the U.S. Phase I / II clinical trial , with higher - dose cohort enrollment expected to be completed by mid - 2022 ~ ~ - Screening initiated in European open - label Phase I / II study ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON , Mass . and AMSTERDAM , The Netherlands , Dec. 16 , 2021 ( GLOBE NEWSWIRE ) uniQure N.V. ( NASDAQ : QURE ) , a leading gene therapy company advancing transformative therapies for patients with severe medical needs , today announced initial observations on the first four patients enrolled in the lower - dose cohort of its ongoing Phase I / II clinical trial of AMT - 130 for the treatment of Huntington's disease . Two of the four enrolled patients received AMT - 130 , and two patients experienced an imitation ( sham ) surgery in this randomized , blinded clinical trial being conducted in the United States . " We are pleased with these data that support the tolerability of AMT - 130 , a potential one time gene - therapy approach for Huntington's disease , " stated Ricardo Dolmetsch , Ph.D. , president of research and development at uniQure . " 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. Based on recommendations of the DSMB and steering committee , we will disclose efficacy data including volumetric MRI changes once all patients in the first two cohorts are unblinded . We look forward to evaluating safety , biomarker and functional data on a larger number of patients and over a longer period during the ongoing study . " " AMT - 130 appears to be generally well tolerated both immediately after surgical administration and at one year of follow up in these four patients , " stated Jody Corey Bloom , M.D. , PhD , professor of neurosciences at University of California , San Diego and chairperson of the Data Safety Monitoring Board ( DSMB ) for AMT - 130 . " We are encouraged by the early data in two treated patients and will continue to provide comprehensive safety oversight of program as additional patients are enrolled and the 1/4