The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety and proof of concept (evidence from pilot project) in 15 patients with early manifest Huntington’s disease. The gene therapy product candidate AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene.
The vector is inactive and just a vehicle to deliver the drug. This a one-time injection directly into the brain. The trial is called, open-label, so the participants know what they are receiving. There are 2 doses, one low, one high.