Uniqure Biopharma B.v.

This early-stage trial is testing a one-time gene therapy called AMT-130, which is injected directly into the brain. The goal is to see if lowering the harmful huntingtin protein is safe and can slow the progression of Huntington's disease in 43 people with early symptoms. The st…

This early-stage study is testing a one-time gene therapy called AMT-162 for adults with ALS caused by a specific faulty SOD1 gene. The therapy is injected into the spinal fluid and aims to 'silence' the harmful gene to slow the disease. Researchers will first check if the treatm…