Uniqure Biopharma B.v.
Clinical trials sponsored by Uniqure Biopharma B.v., explained in plain language.
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New gene therapy targets rare, aggressive ALS
Disease control OngoingThis study 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 it might slow…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 26, 2026 08:29 UTC
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Gene therapy aims to slow Huntington's in first human trial
Disease control OngoingThis study tests a new gene therapy called AMT-130 for people with early-stage Huntington's disease. The therapy is injected directly into the brain to lower the production of a harmful protein that causes the disease. The study includes 43 adults aged 25 to 65 and is focused on …
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 22, 2026 13:52 UTC