~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~

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 needs, today announced the pricing of its underwritten public offering of 4,411,764 of its ordinary shares at a public offering price of $17.00 per share. The aggregate gross proceeds to uniQure from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by uniQure, are expected to be approximately $75 million. All securities to be sold in the offering are being sold by uniQure. In addition, uniQure has granted to the underwriters a 30-day option to purchase up to 661,764 additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is expected to close on or about January 10, 2025, subject to the satisfaction of customary closing conditions.

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 needs, today announced that it has commenced an underwritten public offering of its ordinary shares and pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will be offered by uniQure. In addition, uniQure intends to grant the underwriters a 30-day option to purchase up to 15% additional ordinary shares at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

The company is testing a gene therapy for Huntington's disease and could soon seek an accelerated approval.

uniQure stock surges after FDA agrees on an accelerated approval pathway for its Huntington's disease treatment, AMT-130.

~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 21, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), 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 GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE).

~ 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. (NASDAQQURE), 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 dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS.

LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), 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.

LEXINGTON, Mass. and AMSTERDAM, Aug. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQQURE), 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 multi-center, open-label trial being conducted in the United States with two dose-escalating cohorts assessing the safety, tolerability and early signs of efficacy of AMT-191 in individuals with Fabry disease.

CS Diagnostics Group (OTC: FZRO) signed a landmark agreement to sell and transfer tangible and intangible assets, including patents, for the CS Protect-Hydrogel to CS Diagnostics Corp. The hydrogel-based tissue spacer, designed for radiation therapy, increases the distance between healthy tissue and cancer cells, protecting healthy tissues from high radiation doses.

QURE stock results show that uniQure beat analyst estimates for earnings per share and beat on revenue for the second quarter of 2024.

~ 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 maintains preferential access to industry-leading manufacturing capabilities to support its pipeline of gene therapy candidates ~

UniQure N.V. (NASDAQQURE) shares are skyrocketing Wednesday following the release of promising interim data from its Phase I/II clinical trials of AMT-130, a gene therapy targeting Huntington's disease.