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 FDA agrees that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval ~ Conference call today at 8:30 a.m. ET... Read More

LEXINGTON, Mass. and AMSTERDAM, Nov. 21, 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 GenTLE Phase I/IIa clinical trial of AMT-260 for the treatment of refractory mesial temporal lobe epilepsy ( MTLE ). “The dosing of the first patient in our Phase... Read More

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 Presented positive interim data from Phase I/II trial of AMT-130 demonstrating slowing of Huntington’s disease progression and reductions in key biomarker of neurodegeneration Initiated patient dosing in new Phase I/II studies of AMT-162... Read More

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 dismutase 1 (SOD1), a rare, inherited... Read More

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... Read More

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 multi-center, open-label trial... Read More

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 Initiated patient screening for three additional Phase I/II studies in mesial temporal lobe epilepsy, SOD1 ALS,... Read More

uniQure maintains preferential access to industry-leading manufacturing capabilities to support its pipeline of gene therapy candidates Immediate reduction in cash burn, projected to save $40 million annually LEXINGTON, Mass. and AMSTERDAM, July 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... Read More

Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to... Read More

Strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. BOSTON and INDIANAPOLIS , July 1, 2024 /PRNewswire/ -- Genezen, a best-in-class gene therapy CDMO, today announced an agreement to acquire uniQure's (Nasdaq: QURE) commercial gene therapy operations in Lexington, MA , bringing a strategic manufacturing facility and... Read More

Proposed divestiture streamlines uniQure’s focus and represents an important milestone in its ongoing effort to significantly reduce operating expenses Genezen to manufacture global commercial supply of HEMGENIX ® and support uniQure’s pipeline programs as a preferred customer uniQure to reduce cash burn by $40 million per year and receive total consideration of $25 million uniQure’s comprehensive review of operations and... Read More

Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 AMT-130 is the first therapeutic candidate to receive RMAT Designation for Huntington’s disease Receiving RMAT designation enables increased collaboration with the FDA on regulatory approval planning, in addition to the opportunity for expedited product development LEXINGTON, Mass. and AMSTERDAM, June 03, 2024 (GLOBE... Read More

On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 Comprehensive review of operations and options to reduce expenses underway and expected to be... Read More

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... Read More

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... Read More

HEMGENIX ® is the first and only gene therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia B and to show sustained efficacy and safety at three years post-treatment Long-term data continues to show a one-time infusion of HEMGENIX offers elevated and sustained factor IX activity levels for years and significantly reduces the rate of annual bleeds versus factor IX prophylaxis with a... Read More

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... Read More

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... Read More

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... Read More

HEMGENIX ® , the first and only FDA-approved gene therapy for adults with hemophilia B, wins for Best Product for Rare/Orphan Diseases KING OF PRUSSIA, Pa. and LEXINGTON, Mass. , Oct. 27, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) and uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the companies... Read More

CSL's HEMGENIX ® is a one-time, single dose treatment for adults with hemophilia B who require routine prophylaxis OTTAWA, ON , Oct. 26, 2023 /CNW/ -- Global biotechnology leader CSL (ASX: CSL) today announced that Health Canada has authorized HEMGENIX ® (etranacogene dezaparvovec), the first and only gene therapy for the treatment of hemophilia B. HEMGENIX is indicated for the treatment of adults with hemophilia B who... Read More

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... Read More

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... Read More

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... Read More

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... Read More

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... Read More

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... Read More

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... Read More

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... Read More

HEMGENIX ® , the first and only FDA approved gene therapy for hemophilia B, has been proven to elevate and sustain factor IX levels for years, significantly reduce the rate of annual bleeds versus standard of care, and reduce or eliminate the need for prophylactic treatment in 94% of patients (51 out of 54) after a one-time infusion KING OF PRUSSIA, Pa. , June 20, 2023 /PRNewswire/ -- Global biotechnology leader CSL Behring... Read More

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... Read More

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... Read More

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... Read More

LEXINGTON, Mass. and AMSTERDAM, May 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 that 12 data presentations, including two oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting being held May 16-20 in Los Angeles, California. “Our... Read More

LEXINGTON, Mass. and AMSTERDAM, May 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 that the Company granted equity awards to 38 employees as a material inducement to commencing their employment. The equity awards were approved by the Company’s board of directors on April 24, 2023 (the “Grant... Read More

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 13, 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 Company granted equity awards to 31 employees as a material inducement to commencing their employment. The equity awards were approved by the Company’s board of directors on February... Read More

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 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 its participation in the following upcoming investor and scientific conferences: Cowen Annual Healthcare Conference, March 6 – 8, 2023 Boston, MA Members of uniQure’s management team, including... Read More

Achieved U.S. and European Commission marketing approvals of first and only gene therapy for adults with hemophilia B Advancing development of AMT-130 for patients with Huntington’s disease – clinical update from U.S. Phase I/II trial expected in second quarter 2023 Hosted virtual investor event featuring AMT-260 in refractory temporal lobe epilepsy - expected to enter clinical development in second half of 2023 Expanded... Read More

Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds, reduce or eliminate the need for prophylactic therapy and generate elevated and sustained factor IX levels for years after a one-time infusion 18-month data support the ongoing benefit of HEMGENIX ® HEMGENIX ® is approved for adults with hemophilia B in the United States,... Read More

Data follow the historic approval of the first gene therapy for hemophilia B, which reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion 18-month data support the efficacy and safety of HEMGENIX ® and the ongoing benefit of this treatment HEMGENIX ® is approved for adults with hemophilia B in the... Read More

Historic approval represents the first gene therapy in Europe to treat hemophilia B and provides a new treatment option for patients that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels Approval marks uniQure’s second internally-developed and manufactured gene therapy to achieve licensure in the European Union LEXINGTON, Mass.... Read More

License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs APB-102 and uniQure’s c9orf72-ALS program have the potential to address most inherited forms of ALS uniQure plans to initiate a Phase I/II trial of APB-102 in the second half of 2023 LEXINGTON, Mass. and AMSTERDAM and CAMBRIDGE, Mass., Jan. 31, 2023 (GLOBE NEWSWIRE) --... Read More

LEXINGTON, Mass. and AMSTERDAM, Dec. 20, 2022 (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 Company granted equity awards to employees as a material inducement to commencing their employment. The equity grants were approved by the Company’s board of directors on December 7, 2022, October 31,... Read More

LEXINGTON, Mass. and AMSTERDAM, Dec. 16, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received a positive opinion recommending conditional marketing authorization (CMA) for etranacogene dezaparvovec from the Committee for Medicinal... Read More

Long-term data from the pivotal HOPE-B study show a one-time infusion of HEMGENIX generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding 24-month results reinforced safety of treatment, with no serious treatment-related adverse effects related to HEMGENIX infusion HEMGENIX, recently approved by the U.S. Food and Drug Administration, is the first and only gene therapy for the treatment... Read More

This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion With the approval of HEMGENIX, CSL now offers an even more comprehensive portfolio of treatments for people living with hemophilia B, ushering in a new era of treatment options KING OF... Read More