uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
uniQure N.V. - Ordinary Shares (QURE)
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Source: GlobeNewswire
LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024, uniQure announced the dosing of the first patient in its U.S., multi-center, open-label Phase I/IIa trial of AMT-191. “This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of delivering meaningful benefit given the suboptimal effectiveness of current chronic treatments,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “This designation supports our Phase I/IIa clinical trial and we look forward to rapidly generating clinical proof-of-concept data and pr
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- uniQure (NASDAQ: QURE) was upgraded by analysts at StockNews.com to a "sell" rating.MarketBeat
- uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry DiseaseGlobeNewswire
- uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALSGlobeNewswire
- uniQure (NASDAQ: QURE) had its price target raised by analysts at HC Wainwright from $25.00 to $70.00. They now have a "buy" rating on the stock.MarketBeat
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- 1/8/25 - Form 424B5
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