Uniqure Biopharma B.v.

This early-stage trial tests a one-time gene therapy called AMT-130, injected directly into the brain, to lower the production of the harmful huntingtin protein. It involves 43 adults with early Huntington's disease. The study is primarily checking safety, but also looks for sign…

This early-stage trial tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that i…