uniQure
Clinical trials sponsored by uniQure, explained in plain language.
A gene therapy company developing treatments for severe genetic diseases.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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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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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 1/2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Aug 26, 2026 00:00 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 1/2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC