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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) announced participation in several upcoming virtual conferences in September 2020. Key events include:
- Citi’s 15th Annual BioPharma Virtual Conference (Sept. 9-10) with a live fireside chat on Sept. 9.
- Baird’s Virtual Global Healthcare Conference (Sept. 9-10) featuring one-on-one investor meetings.
- Wells Fargo Global Healthcare Conference (Sept. 9-10) with a fireside chat on Sept. 10.
- H.C. Wainwright 22nd Annual Global Investment Conference (Sept. 14-15) and Cantor Fitzgerald Virtual Global Healthcare Conference (Sept. 15-17).
- Barclays Boston Biotech Bus Trip (Sept. 23-25).
These events aim to provide insights into uniQure's gene therapy advancements.
uniQure N.V. (NASDAQ: QURE) appointed Ricardo Dolmetsch, Ph.D. as the new President of Research and Development, effective September 14, 2020, succeeding Sander van Deventer, M.D., Ph.D.. Dolmetsch, previously Global Head of Neuroscience at Novartis, brings extensive experience in gene therapy, notably in developing treatments for neurodegenerative diseases. CEO Matt Kapusta praised Dolmetsch's expertise and vision. Under Dolmetsch's leadership, uniQure aims to advance therapies for Huntington’s disease and expand its pipeline. The company plans an extraordinary shareholder meeting in Q4 2020 to re-elect Dr. Robert Gut as a non-executive director.
uniQure N.V. (NASDAQ: QURE) reported significant developments in its Q2 2020 results, including a global licensing agreement with CSL Behring for its hemophilia B gene therapy, potentially worth over $2 billion. The company completed initial dosing in a Phase I/II trial for AMT-130, targeting Huntington’s disease. Financially, uniQure held $314.3 million in cash, expected to fund operations into 2024, despite reporting a net loss of $42.6 million. The revenue declined to $1.5 million, a decrease from $2.5 million year-over-year.
CSL Behring has announced its acquisition of exclusive global rights to AMT-061, a novel gene therapy for hemophilia B from uniQure (QURE). The program, currently in Phase 3 trials, could offer long-term benefits to patients. An upfront payment of US$450 million will be made to uniQure, along with milestone payments and royalties. The therapy aims to restore Factor IX levels in patients, potentially eliminating the need for ongoing treatments. This acquisition supports CSL Behring's commitment to innovative therapies in hemophilia and expands its gene therapy portfolio.
uniQure has entered a licensing agreement with CSL Behring granting exclusive global rights to develop and commercialize etranacogene dezaparvovec, a gene therapy for hemophilia B. uniQure will receive over $2 billion in total, including $450 million upfront and $1.6 billion in milestone payments, along with royalties in the low-twenties percentage range of net sales. This collaboration aims to leverage CSL Behring's global reach to enhance patient access and enable uniQure to accelerate its pipeline, including advancing treatments for Huntington’s disease and Fabry disease.
uniQure (NASDAQ: QURE) announced the treatment of the first two patients in its Phase I/II clinical trial for AMT-130, aimed at Huntington’s disease. This double-blind study, involving 26 patients, will assess safety, tolerability, and efficacy signals. One patient received AMT-130 while the other underwent sham surgery. The trial features an 18-month core study followed by long-term follow-ups. The innovative delivery method involves MRI-guided surgery. This milestone reflects uniQure's commitment to developing effective therapies for Huntington’s disease, a condition with no currently approved treatments.
uniQure N.V. has appointed Leonard E. Post, Ph.D., to its Board of Directors, enhancing its research leadership. Dr. Post, with over 35 years in biotechnology, will also chair the Research & Development Committee. His past roles include significant positions at BioMarin and Onyx Pharmaceuticals, focusing on gene therapy. This strategic move aims to bolster uniQure's pipeline, particularly in preparing for marketing authorization of etranacogene dezaparvovec for hemophilia B. This leadership change reflects a commitment to advancing innovative therapies for severe medical conditions.
uniQure N.V. (NASDAQ: QURE) announced a virtual format for its 2020 Annual General Meeting on June 17, 2020, due to COVID-19 concerns. Shareholders of record as of May 20, 2020, can participate via a virtual meeting platform but cannot vote in real-time or attend in person. The meeting’s details can be accessed through the uniQure website. The company is focused on advancing gene therapy treatments for severe genetic diseases, including hemophilia and Huntington’s disease.
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