AMT-161

AMT-161 is an investigational **AAV5-based gene therapy** from uniQure for **amyotrophic lateral sclerosis caused by C9orf72 mutations**. It uses the company's **miQURE microRNA gene-silencing platform** and is designed as a **one-time intrathecal treatment** to selectively reduce toxic repeat-expanded **C9orf72** transcripts, with the goal of lowering pathogenic RNA foci and dipeptide repeat protein formation while preserving normal C9orf72 expression. Publicly available company and pipeline materials indicate that the program has remained **preclinical/early research** rather than entering human trials as of the latest available updates.

Other names

AAV5-miC gene therapyAAV-5-miC gene therapyAAV 5-miC gene therapy