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
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.
- 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.
- NfL increased as expected immediately following the AMT-130 surgical procedure and returned to baseline in the two treated patients.
- Nineteen patients have been enrolled in the clinical trial to date, including 9 of 16 in the higher-dose cohort.
- The European, open-label Phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntingtons disease across two dose cohorts.