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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.
CSL announced significant findings from the 24-month follow-up of the pivotal HOPE-B study for HEMGENIX, the first gene therapy approved for hemophilia B. The treatment resulted in sustained mean factor IX activity levels (36.7 IU/dL) and a 64% reduction in bleeding rates (ABR) among participants. Safety data indicated no serious treatment-related adverse effects. With 94% of patients discontinuing prophylaxis, HEMGENIX shows promise for long-term efficacy and safety in hemophilia B treatment, highlighting CSL's commitment to innovative therapies.
uniQure (NASDAQ: QURE) recently hosted a virtual investor event highlighting its gene therapy AMT-260, targeting refractory temporal lobe epilepsy. The event included insights from leading clinical experts and showcased promising preclinical data indicating AMT-260's efficacy and safety. The company plans to submit an investigational new drug application for AMT-260 and initiate clinical development in 2023. Additionally, uniQure reported advancements in its manufacturing platform, enhancing efficiency and reducing costs for AAV therapies.
uniQure N.V. announced the FDA's approval of HEMGENIX® (etranacogene dezaparvovec-drlb), a groundbreaking gene therapy for hemophilia B. This one-time treatment offers significant benefits, including a reduction in annual bleeding rates and discontinuation of prophylactic therapy for 94% of trial participants. Developed through a comprehensive clinical program, HEMGENIX enables patients to produce their own factor IX, improving their quality of life. uniQure will supply CSL for commercialization, having received $500 million in payments and potential future royalties valued at up to $1.5 billion.
The FDA has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first one-time gene therapy for adults with hemophilia B, allowing eligible patients to produce their own factor IX. This treatment reduces the frequency of annual bleeding episodes and eliminates the need for prophylactic therapies. In the pivotal HOPE-B trial, 94% of patients discontinued factor IX prophylaxis post-treatment, with a 54% reduction in the annualized bleeding rate. HEMGENIX represents a significant advancement in hemophilia B treatment, enhancing patient outcomes with a single infusion.
uniQure N.V. (NASDAQ: QURE) announced a Virtual Research & Development Event on November 29, 2022, from 8:30 a.m. to 10:30 a.m. EST, focusing on refractory temporal lobe epilepsy (rTLE) and its preclinical gene therapy candidate AMT-260. The event will feature Dr. Ellen Bubrick, a clinical expert from Brigham and Women’s Hospital, alongside presentations from uniQure’s R&D team and COO Pierre Caloz on advancements in the AAV manufacturing platform. The event will be webcast live and available for replay on uniQure’s website.
VectorY Therapeutics appoints Sander van Deventer as Chief Executive Officer, succeeding Alexander Vos. Van Deventer, a co-founder with 25 years of drug development experience, aims to advance the company's innovative vectorized antibody technology for neurodegenerative diseases, including ALS and Huntington's disease. Other leadership changes include Elena Ritsou as Chief Corporate Officer, Barbara Sanders promoted to Chief Technology Officer, and Frank Walsh joining as an Independent Board Member. These appointments are expected to strengthen VectorY’s strategic direction.
uniQure N.V. (NASDAQ: QURE) reported a net loss of $47.9 million for Q3 2022, with revenues of $1.4 million. The company resumed higher-dose patient enrollment for AMT-130 in Huntington's disease following a DSMB recommendation. The EMA awarded a Good Manufacturing Practice certificate for their Lexington facility, crucial for their gene therapy manufacturing. Cash and equivalents stood at $440.3 million, expected to fund operations until mid-2025. Upcoming events include a virtual investor presentation on November 29, 2022, focused on AMT-260 for refractory temporal lobe epilepsy.
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.
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