Uniqure Biopharma B.v.
Clinical trials sponsored by Uniqure Biopharma B.v., explained in plain language.
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Gene therapy breakthrough targets severe epilepsy in first human trial
Disease control Recruiting nowThis early-stage study tests a new gene therapy called AMT-260 in 12 adults with a severe form of epilepsy that doesn't respond to standard medications. The treatment is delivered directly into the brain using MRI guidance. The main goals are to check safety and see if it can red…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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Gene therapy trial aims to fix enzyme defect in fabry disease
Disease control Recruiting nowThis early-stage trial is testing a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The therapy uses a harmless virus to deliver a working copy of the GLA gene to the liver, so the body can produce the missing enzyme. The main goals are to chec…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC