~ Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington's disease ~
LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 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 first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismuta...
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,...
LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 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 first patient has been dosed in a Phase I/IIa clinical trial of AMT-191 for the treatment of Fabry disease, a rare, inherited genetic disease. The Phase I/IIa study is a m...
uniQure N.V. released positive 24-month trial data for AMT-130 gene therapy for Huntington's Disease, leading to a significant stock price increase. The company has aggressively restructured to extend its cash runway, focusing on AMT-130 development with confidence in its success. Despite risks, including potential negative trial data, its strong balance sheet and upcoming catalysts make it pos...
~ Announced RMAT designation for AMT-130 in Huntington's disease and positive interim Phase I/II data demonstrating the slowing of disease progression and reductions in a key biomarker of neurodegeneration; Meeting with FDA expected in the second half of 2024 to discuss potential for expedited clinical development ~
uniQure stock (NASDAQ: QURE), a gene therapy company, has seen a stellar 150% rise in a week, faring significantly better than its peer – Editas Medicine stock –, up 12%. This massive rise in QURE stock can be attributed to the positive outcome from its phase I-II clinical trials of AMT-130 for Huntington's disease.
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