News & Videos - UNIQURE N.V.

~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial temporal lobe epilepsy (mTLE) and Fabry disease, in addition to SOD1-ALS, are...

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline at 18 months ~ ~ AMT-130 continues to be generally well-tolerated across both doses ~ ~ Data support continuing clinical development of...

LEXINGTON, Mass. and AMSTERDAM, Dec. 08, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to newly hired employees. The company granted equity awards to 28 employees as a material inducement to commencing their employment. In the aggregate, those employees received 142,300 restricted share units and options to purchase 52,500...

LEXINGTON, Mass. and AMSTERDAM, Nov. 29, 2023 (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 cleared the Investigational New Drug (IND) application for AMT-191, the Company’s gene therapy candidate for Fabry disease . AMT-191 comprises an AAV5 vector that delivers an α-galactosidase A (GLA) transgene designed to target...

~ Announced FDA clearance of Investigational New Drug (IND) application for AMT-260 in refractory mesial temporal lobe epilepsy ~ ~ On track to provide clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease later in the fourth quarter of 2023 ~ ~ uniQure and CSL win 2023 Prix Galien USA award for HEMGENIX ® ~ ~ Announced strategic reorganization to focus on advancing multiple clinical-stage programs and expect to deliver $180 million of cost savings over...

LEXINGTON, Mass. and AMSTERDAM, Oct. 24, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium. “Our multiple presentations at ESGCT demonstrate uniQure’s continued leadership...

~ Reduction of 28% of workforce not related to HEMGENIX ® manufacturing obligations; Total cost savings of $180 million to extend cash runway into second quarter of 2027 ~ ~ Discontinuing investments in more than half of research and technology projects, centralizing operations, and streamlining organization ~ ~ Prioritizing continued development of AMT-130 in Huntington’s disease and near-term initiation of clinical trials for AMT-260 in refractory mesial temporal lobe epilepsy, AMT-162 in...

LEXINGTON, Mass. and AMSTERDAM, Oct. 02, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to newly hired employees. The Company granted equity awards to 42 employees as a material inducement to commencing their employment. In the aggregate, those employees received 98,900 restricted share units and options to purchase 90,500 ordinary...

LEXINGTON, Mass. and AMSTERDAM, Sept. 05, 2023 (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 cleared the Investigational New Drug (IND) application for AMT-260, the Company’s gene therapy candidate for refractory mesial temporal lobe epilepsy ( MTLE ). AMT-260 comprises an AAV9 vector that locally delivers two...

~ Announced encouraging interim analysis data from ongoing Phase I/II clinical trial of AMT-130 in Huntington’s disease showing preservation of function compared to baseline and clinical benefits relative to natural history; both dose cohorts of AMT-130 continue to be generally well-tolerated ~ ~ Preparations underway to initiate two Phase I/II clinical trials in refractory temporal lobe epilepsy and SOD1-ALS later this year; IND for Fabry disease expected to be submitted in the second half of...

LEXINGTON, Mass. and AMSTERDAM, June 26, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the appointment of Walid Abi-Saab, M.D., as Chief Medical Officer, effective immediately. He will report to Matt Kapusta, Chief Executive Officer of uniQure, and will be based in the Company’s Basel, Switzerland office. Dr. Abi-Saab will be responsible for leading all clinical...

The Schall Law Firm , a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of uniQure N.V. (“uniQure” or “the Company”) (NASDAQ: QURE ) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. If you are a shareholder who suffered a loss, click here to participate . We also encourage you to...

The Schall Law Firm , a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of uniQure N.V. (“uniQure” or “the Company”) (NASDAQ: QURE ) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. If you are a shareholder who suffered a loss, click here to participate . We also encourage you to...

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Thinking about buying stock in bluebird bio, Uniqure, Terawulf, Metaworks Platforms, or Nikola Corp? PR Newswire NEW YORK, June 21, 2023 NEW YORK , June 21, 2023 /PRNewswire/ -- InvestorsObserver issues critical PriceWatch Alerts for BLUE, QURE, WULF, MWRK, and NKLA. To see how InvestorsObserver's proprietary scoring system rates these stocks, view the InvestorsObserver's PriceWatch Alert by selecting the corresponding link. BLUE:...

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~ ~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease ~ ~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in patients treated with the low-dose of AMT-130 and declining towards baseline at 12 months in patients treated with the high-dose of AMT-130 ~ ~ Suppression of CSF mHTT in low-dose...

LEXINGTON, Mass. and AMSTERDAM, June 20, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced the achievement of a $100 million milestone associated with the first commercial sale of HEMGENIX ® in the United States by its partner, global biotechnology leader CSL (ASX: CSL). HEMGENIX ® (etranacogene dezaparvovec) is a one-time administered gene therapy for the treatment of adults...

LEXINGTON, Mass. and AMSTERDAM, June 16, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the grant of inducement equity awards to Jeannette Potts, the Company’s newly hired Chief Legal and Compliance Officer, and other newly hired employees. The inducement awards to Ms. Potts consist of a non-qualified option to purchase 81,300 ordinary shares of the Company and...

~ uniQure to receive $375 million upfront cash payment ~ ~ Under the existing agreement with CSL Behring, uniQure retains the rights to future milestones totaling up to $1.5 billion and maintains an interest in HEMGENIX ® royalties ~ ~ Capped royalty financing provides uniQure non-equity-dilutive capital to advance its broad gene therapy pipeline, including AMT-130 for Huntington’s disease ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 15, 2023 (GLOBE NEWSWIRE) -- uniQure N.V....

~ Clinical update from U.S. Phase I/II trial in Huntington’s disease expected in second quarter 2023 ~ ~ Continued progress towards initiating two new Phase I/II clinical studies for AMT-260 in refractory temporal lobe epilepsy and AMT-162 in SOD1-ALS ~ LEXINGTON, Mass and AMSTERDAM, the Netherlands, May 09, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial...