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UniQure Reports Positive Data From Fabry Disease Gene Therapy Trial
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UniQure Reports Positive Data From Fabry Disease Gene Therapy Trial
Mar 11, 2026 3:14 AM

08:06 AM EST, 02/06/2026 (MT Newswires) -- UniQure N.V. ( QURE ) said Friday that updated preliminary trial results for its experimental gene therapy AMT-191 showed increased enzyme levels in all 11 Fabry disease patients treated.

The phase I/IIa study tested three dose levels and higher doses led to stronger and longer-lasting enzyme increases, with effects lasting up to more than a year in some patients, the company said.

Six patients were able to stop regular enzyme replacement therapy after meeting study criteria, the company added.

The therapy showed a generally manageable safety profile, the company said, adding that some patients experienced temporary liver enzyme elevations, which led it to pause further dosing at higher levels.

The company said the results support the potential of AMT-191 as a one-time treatment for Fabry disease.

UniQure ( QURE ) shares were 1.2% higher in premarket trading.

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