Uniqure Biopharma B.v.
Clinical trials sponsored by Uniqure Biopharma B.v., explained in plain language.
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One-Shot gene therapy trial offers new hope for men with rare genetic disease
Disease control Recruiting nowThis is an early-stage study testing a single-dose gene therapy called AMT-191 in adult men with classic Fabry disease. The main goals are to find a safe dose and see how the body processes the treatment. The therapy aims to help the liver produce a missing enzyme, potentially of…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
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Brain gene therapy trial offers hope for untreatable epilepsy
Disease control Recruiting nowThis first-in-human study is testing whether a new gene therapy called AMT-260 can safely reduce seizures in adults with drug-resistant temporal lobe epilepsy. All 12 participants will receive a single dose delivered directly to the brain using advanced MRI guidance, without remo…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC