The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety, proof of concept, and dosing in 15 total patients with with early manifest Huntington’s disease split into a five person manifest Huntington’s disease split into a five person, low-dose open-label(patients know what they are getting) cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130. The therapeutic goal is to inhibit the production of the mHTT. Using AAV vectors to deliver micro-RNAs directly to the brain. A one time injection.
Estimated Study Completion Date: May 2026