Latest News and Press Releases

~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and...

~ No serious adverse events and 92% reduction in seizure frequency observed in the first trial participant through first five months of follow up ~ ~ Data to be presented today at Epilepsy...

~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no...

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics...

~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington’s disease; Initiated preparations for a potential...

~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~ ~ Company expects to initiate enrollment of second dose cohort in the...

~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~ ~ Company Expects to Initiate Enrollment of Second Dose...

LEXINGTON, Mass. and AMSTERDAM, Jan. 08, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...

LEXINGTON, Mass. and AMSTERDAM, Jan. 07, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...