uniQure
Clinical trials sponsored by uniQure, explained in plain language.
A gene therapy company developing treatments for severe genetic diseases.
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Upcoming readouts
Trials from this sponsor expected to report results within the next 12 months.
- New gene therapy targets rare, aggressive ALS Results expected Sep 2026 (estimated)
- Gene therapy breakthrough targets severe epilepsy in first human trial Results expected Nov 2026 (estimated)
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Brain gene therapy aims to silence Huntington's disease
Disease control OngoingThis 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…
Phase 1/2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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New gene therapy targets rare, aggressive ALS
Disease control OngoingThis 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…
Phase 1/2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC