UniQure Biopharma B.V. HD GeneTRX2 study endorsed* by EHDN

Study aims and goal: A Phase Ib/II Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington’s Disease.
No. of study participants and sites: This phase Ib/II b study aims to recruit 15 participants from approximately 5 study sites in Germany, Poland and the UK.
For further information see the clinical trial registration.