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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 its participation in several investor and scientific conferences, showcasing its advancements in gene therapy. Key events include Chardan's 6th Annual Genetic Medicines Conference from October 3-4, where a fireside chat with Dr. Ricardo Dolmetsch is scheduled. Additionally, at the European Society of Gene & Cell Therapy from October 11-14, uniQure will present preclinical data on various neurological diseases. Lastly, Dr. Dolmetsch will discuss Huntington's disease at the Latin American Huntington's Disease Conference from October 19-21.
uniQure N.V. (NASDAQ: QURE) announced participation in several investor and scientific conferences this September. Key events include Citi’s 17th Annual BioPharma Conference (Sept. 7-8), Wells Fargo 2022 Healthcare Conference (Sept. 7-9), H.C. Wainwright 24th Annual Global Investment Conference (Sept. 12-14), Cantor Fitzgerald Cell & Gene Therapy Conference (Sept. 15), and European Huntington’s Disease Network (Sept. 16-18). Management, including CEO Matt Kapusta and President of R&D Ricardo Dolmetsch, will engage in investor meetings, fireside chats, and presentations, sharing insights on gene therapies for severe medical needs.
Fulgent Genetics (NASDAQ: FLGT) has appointed Dr. Michael Nohaile and Dr. Leonard Post to its Board of Directors, effective August 1, 2022. Dr. Nohaile, previously at Generate Biomedicines and Amgen, has extensive experience in drug discovery and corporate strategy. Dr. Post, from Vivace Therapeutics, brings over 30 years of drug development experience, particularly in oncology and genetic diseases. Both appointments aim to enhance Fulgent's capabilities as it explores new therapeutic opportunities. The company emphasizes the value these experts will bring to its evolving operations.
uniQure's Phase I/II trial of AMT-130 for Huntington’s disease shows promising results with a 53.8% reduction in mutant HTT protein after 12 months. The treatment was well-tolerated, with no significant safety issues reported. Neurofilament Light Chain levels approached baseline, indicating potential neuronal protection. The company plans to present more data later this year and has completed enrollment for all 26 patients across its U.S. and European trials. CEO Matt Kapusta emphasized the progress in clinical investigations and the commitment to the Huntington's disease community.
uniQure N.V. (NASDAQ: QURE) announced the granting of equity awards to 22 employees to incentivize their employment. The board approved the issuance of 39,500 restricted stock units (RSUs) and options for 27,500 ordinary shares at an exercise price of $14.08 each on June 15, 2022. The options will vest over four years while the RSUs will vest over three years. This move follows Nasdaq Listing Rule 5635(c)(4) and demonstrates uniQure's commitment to attracting talent to advance their gene therapy initiatives.
uniQure N.V. (NASDAQ: QURE) announced the granting of equity awards to 32 newly hired employees as an incentive for their employment. As per Nasdaq Listing Rule 5635(c)(4), the grants, approved on April 29, 2022, include 84,900 restricted stock units and options to purchase 119,000 ordinary shares at an exercise price of $14.94 per share, reflecting the share's closing price on the grant date. The options will vest over four years, while the RSUs will vest over three years, contingent on the employees' continued service.
uniQure N.V. announces the FDA's acceptance for priority review of the Biologics License Application (BLA) for etranacogene dezaparvovec, a gene therapy aimed at treating hemophilia B. If approved, this therapy would be the first of its kind for patients suffering from this condition. The treatment has shown a 64% reduction in bleeding rates post-infusion in the pivotal HOPE-B trial. With CSL Behring as its commercialization partner, uniQure aims to provide a transformative solution for individuals with hemophilia B.
uniQure N.V. (NASDAQ: QURE) announced granting equity awards to 32 new employees to incentivize their employment. This decision, approved on April 29, 2022, follows Nasdaq Listing Rule 5635(c)(4). The grants consist of 84,900 restricted stock units (RSUs) and options to purchase 119,000 ordinary shares at an exercise price of $14.94 per share, which is the closing price on the Grant Date. The options will vest over four years; RSUs will vest over three years, subject to continued employment.
uniQure N.V. (NASDAQ: QURE) announced six data presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting from May 16-19, 2022, in Washington, D.C. Highlights include an oral presentation on alpha-synuclein lowering in Parkinson's models and several poster sessions covering advancements in AAV gene therapies. Dr. Ricardo Dolmetsch emphasized the innovative potential of the linQURE® cargo platform in silencing mutant C9orf72 in ALS preclinical models. The event underscores uniQure's commitment to advancing gene therapies for severe medical conditions.
uniQure N.V. (QURE) reported Q1 2022 financial results, with revenues of $1.8 million, up from $0.5 million in Q1 2021, driven by collaboration revenue from CSL Behring. The company received $55 million in milestone payments related to the regulatory progress of etranacogene dezaparvovec for hemophilia B, which was validated by the EMA for accelerated assessment. R&D expenses rose to $45 million, while SG&A expenses decreased to $11 million. The net loss was $46.7 million, or $1.00 per share. Cash and equivalents stood at $524.9 million, projected to fund operations into H1 2025.
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