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uniQure N.V. (NASDAQ: QURE) is a leading gene therapy company focused on developing transformative therapies for patients suffering from severe genetic and other devastating diseases. Based on its proprietary technology platform, uniQure aims to deliver single-treatment solutions with potentially curative results. The company is dedicated to addressing unmet medical needs in the areas of hemophilia, Huntington's disease, and cardiovascular diseases.
uniQure's core business revolves around advancing a robust pipeline of gene therapies, including its collaboration with Bristol Myers Squibb to develop treatments for cardiovascular diseases. The company’s most notable product, HEMGENIX®, is the first and only gene therapy approved for the treatment of adults with hemophilia B. Recent data from the HOPE-B study presented at the American Society of Hematology Annual Meeting confirmed the long-term efficacy and safety of HEMGENIX®, highlighting its ability to offer elevated and sustained factor IX activity levels for years post-treatment.
The company's financial highlights and strong partnerships underscore its commitment to innovation and leadership in the gene therapy field. uniQure continues to make significant strides with current projects and patient enrollment for new trials expected to begin in the first half of 2024.
With its validated modular technology platform and a dedicated focus on severe genetic diseases, uniQure’s work holds significant promise for transforming patient care and outcomes. The company's latest developments and financial performance are followed closely by investors and the biotechnology community, reflecting its pivotal role in advancing gene therapy solutions.
uniQure N.V. (NASDAQ: QURE) has announced the completion of the first patient procedures in the Phase I/II clinical trial for AMT-130, targeting Huntington's disease. This trial's second cohort will involve 16 patients at a higher dose, following a review by the Data Safety Monitoring Board. So far, seven patients have been treated with AMT-130, with five receiving imitation surgeries. The company plans to enroll more patients and expects to share initial imaging and biomarker data before year-end.
uniQure N.V. (NASDAQ: QURE) will host a virtual Research & Development Day on June 22, 2021, from 8:30 to 11:30 a.m. ET, focusing on its expanding gene therapy pipeline for CNS and liver-directed disorders. The company will also participate in multiple upcoming virtual conferences including the Jefferies Virtual Healthcare Conference (June 1-4), European Hematology Association Virtual 2021 (June 7-13), and Goldman Sachs 42nd Annual Global Healthcare Conference (June 8-11). Notably, results from the pivotal Phase 3 HOPE-B trial will be presented during the EHA conference.
uniQure, a leader in gene therapy, announced its upcoming participation in several virtual investor and scientific conferences, including the Jefferies Virtual Healthcare Conference and the European Hematology Association Virtual 2021. Key highlights include a Research & Development Day on June 22, 2021, focusing on advancements in gene therapy for CNS and rare liver-directed disorders. Additionally, the company will present pivotal data from the HOPE-B trial for etranacogene dezaparvovec targeting hemophilia B.
uniQure has announced the independent Data Safety Monitoring Board (DSMB) has recommended proceeding with the second, higher-dose cohort of its Phase I/II clinical trial for AMT-130, aimed at treating Huntington’s disease. This decision follows a positive review of safety data from the first cohort of ten patients. Moving forward, uniQure plans to enroll patients in the higher-dose cohort and initiate clinical development in Europe later this year. The trial aims to establish the safety and efficacy of AMT-130 in patients with early-stage Huntington's disease.
uniQure presented pivotal Phase III HOPE-B trial data for etranacogene dezaparvovec at the ASGCT Annual Meeting. The study, involving 54 hemophilia B patients, showed that pre-existing neutralizing antibodies (NAbs) to AAV5 did not significantly correlate with FIX activity. Participants with NAbs achieved similar treatment results compared to those without. Notably, 57.4% of subjects had no pre-existing NAbs, and all but one participant achieved stable FIX levels, suggesting etranacogene dezaparvovec could be a viable therapy for a broader patient base.
uniQure N.V. (NASDAQ: QURE) reported its Q1 2021 results, highlighting its advancements in gene therapy for hemophilia B and Huntington's disease. The company closed a $450 million licensing deal with CSL Behring and completed patient enrollment for a Phase I/II trial of AMT-130 for Huntington's. Cash reserves increased to $260.8 million, with funding expected until 2024. However, the net loss rose to $41.6 million, with R&D expenses reaching $32.7 million. Upcoming catalysts include 52-week follow-up data from the HOPE-B trial and a planned R&D Day.
CSL Behring announced the closing of its global commercialization and license agreement with uniQure for etranacogene dezaparvovec (AMT-061), a novel gene therapy for hemophilia B. This agreement follows the completion of antitrust reviews and includes a $450 million upfront payment to uniQure, along with potential milestone payments and royalties. Etranacogene dezaparvovec aims to deliver near-normal levels of Factor IX for patients and could transform treatment for hemophilia B patients. Clinical trials show promise, with no serious treatment-related adverse events reported thus far.
uniQure N.V. has announced that the waiting period for its licensing agreement with CSL Behring for etranacogene dezaparvovec has ended, making the deal effective from May 6, 2021. uniQure will receive a $450 million upfront payment due by May 13, 2021, with potential future payments up to $1.6 billion based on milestones. The agreement includes tiered royalties of up to low-twenties percentage on sales. uniQure remains responsible for the pivotal HOPE-B study and initial manufacturing, while CSL Behring will handle commercialization.
uniQure N.V. (NASDAQ: QURE) announced its participation in three upcoming virtual investor and scientific conferences. The Truist Securities Life Sciences Summit is scheduled for May 4-5, 2021, featuring one-on-one meetings. The American Society of Gene and Cell Therapy (ASGCT) virtual event will take place from May 11-14, 2021, showcasing five data presentations, including two oral presentations on May 12 and 13. Finally, at the RBC Capital Markets Global Healthcare Conference on May 18-19, 2021, there will be additional virtual meetings and a fireside chat with CEO Matt Kapusta.
uniQure N.V. (NASDAQ: QURE) announced five data presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting from May 11-14. The presentations will focus on gene therapy advancements targeting hemophilia and Huntington’s disease, highlighting the AAV5 vector's efficacy amid pre-existing neutralizing antibodies. Notable sessions include two oral presentations on the Phase 3 HOPE-B trial and miQURE technology's proof-of-concept for liver targeting. These sessions aim to showcase uniQure's innovation in gene therapy for severe medical conditions.
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