uniQure N.V. announced the positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington?s disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment. A total of 14 blinded administration procedures have been completed as of August 2021. 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, high-dose cohort is expected to be completed in mid-2022. The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington?s disease is exploring the safety, tolerability, and efficacy signals in a planned 26 total patients with early manifest Huntington?s disease split into a 10 patient, low-dose cohort followed by a 16 patient, high-dose cohort; patients will be randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. A total of 16 patients in the clinical trial will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The European, open-label Phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntington?s disease across two dose cohorts. Together with the U.S. study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development or into a confirmatory study should an accelerated registration pathway be feasible. AMT-130 is the company?s first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE? platform.