uniQure Reports Sustained Enzyme Increases in Phase I/IIa Fabry Disease Gene Therapy Trial

Reuters02-06

uniQure Reports Sustained Enzyme Increases in Phase I/IIa Fabry Disease Gene Therapy Trial

uniQure NV has announced updated preliminary data from its Phase I/IIa clinical trial of AMT-191, an investigational AAV gene therapy for Fabry disease. The data, presented at the 22nd Annual WORLDSymposium on February 3, 2026, showed sustained and dose-dependent increases in α-Gal A enzyme activity across all dose cohorts, with supraphysiological expression maintained for over a year in the longest treated patient. Six out of eleven patients met criteria to discontinue enzyme replacement therapy, and stable plasma lyso-Gb3 levels were observed post-dosing, regardless of enzyme replacement therapy status. The safety profile remains manageable, with no serious adverse events related to AMT-191 at lower doses, though dosing in the mid- and high-dose cohorts has been paused following asymptomatic liver enzyme elevations in two patients. An oral presentation of the results is scheduled for February 6, 2026, and the presentation will be available on uniQure's website following the event.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. uniQure NV published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9650145-en) on February 06, 2026, and is solely responsible for the information contained therein.

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The data, presented at the 22nd Annual WORLDSymposium on February 3, 2026, showed sustained and dose-dependent increases in α-Gal A enzyme activity across all dose cohorts, with supraphysiological expression maintained for over a year in the longest treated patient. Six out of eleven patients met criteria to discontinue enzyme replacement therapy, and stable plasma lyso-Gb3 levels were observed post-dosing, regardless of enzyme replacement therapy status. The safety profile remains manageable, with no serious adverse events related to AMT-191 at lower doses, though dosing in the mid- and high-dose cohorts has been paused following asymptomatic liver enzyme elevations in two patients. 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The data, presented at the 22nd Annual WORLDSymposium on February 3, 2026, showed sustained and dose-dependent increases in α-Gal A enzyme activity across all dose cohorts, with supraphysiological expression maintained for over a year in the longest treated patient. Six out of eleven patients met criteria to discontinue enzyme replacement therapy, and stable plasma lyso-Gb3 levels were observed post-dosing, regardless of enzyme replacement therapy status. The safety profile remains manageable, with no serious adverse events related to AMT-191 at lower doses, though dosing in the mid- and high-dose cohorts has been paused following asymptomatic liver enzyme elevations in two patients. 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ID: GNW9650145-en) on February 06, 2026, and is solely responsible for the information contained therein.</span></i>\n</p>\n</div></body></html>\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"LU2210150020.SGD","symbol_name":"Natixis Thematics Subscription Economy R/A SGD","start_time":0,"source_url":"https://api.refinitiv.com/data/news/v1/stories/urn:newsml:reuters.com:20260206:nNDLMPT6D:1","article_id":"2609598571","we_media_id":"1032215980","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2609598571","pubTimestamp":1770379516,"columns":[],"sourceInfo":null,"weMediaInfo":{"media_name":"Reuters","introduction":"Reuters.com brings you the latest news from around the world, covering breaking news in markets, business, politics, entertainment and technology","home_visible":1,"id":"1032215980","head_image":"https://community-static.tradeup.com/news/4567337cbdf294b657b1fa87c5488b48"},"summary":"uniQure NV has announced updated preliminary data from its Phase I/IIa clinical trial of AMT-191, an investigational AAV gene therapy for Fabry disease. 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The data, presented at the 22nd Annual WORLDSymposium on February 3, 2026, showed sustained and dose-dependent increases in α-Gal A enzyme activity across all dose cohorts, with supraphysiological expression maintained for over a year in the longest treated patient. Six out of eleven patients met criteria to discontinue enzyme replacement therapy, and stable plasma lyso-Gb3 levels were observed post-dosing, regardless of enzyme replacement therapy status. The safety profile remains manageable, with no serious adverse events related to AMT-191 at lower doses, though dosing in the mid- and high-dose cohorts has been paused following asymptomatic liver enzyme elevations in two patients. An oral presentation of the results is scheduled for February 6, 2026, and the presentation will be available on uniQure's website following the event.\n      \n\n\nDisclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. 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