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 form of ALS
Disease control OngoingThis study tests a single-dose gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The therapy is injected into the spinal fluid to deliver a working gene. The main goal is to check safety and tolerability, while also looking for early signs o…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 17, 2026 06:19 UTC
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One-Time brain injection aims to slow Huntington's disease
Disease control OngoingThis study tests a new gene therapy called AMT-130 for people with early-stage Huntington's disease. It involves a one-time injection into the brain to lower the production of the harmful huntingtin protein. The trial includes 43 participants and is focused on safety and whether …
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 13, 2026 15:59 UTC