Welcome to our dedicated page for Uniqure news (Ticker: QURE), a resource for investors and traders seeking the latest updates and insights on Uniqure stock.
uniQure (QURE) is a leading biotechnology company advancing innovative gene therapies for severe genetic diseases, including hemophilia and Huntington's disease. This dedicated news hub provides investors and stakeholders with timely updates on clinical developments, regulatory milestones, and strategic partnerships.
Access the most comprehensive collection of official press releases, clinical trial progress reports, and financial disclosures directly impacting QURE's trajectory. Our curated feed ensures you never miss critical updates about therapeutic advancements or collaborative ventures with industry leaders like Bristol Myers Squibb.
This resource serves investors seeking material events analysis, researchers tracking gene therapy innovations, and healthcare professionals monitoring treatment breakthroughs. All content is sourced from verified channels to maintain accuracy and compliance with financial reporting standards.
Bookmark this page for streamlined access to uniQure's latest announcements. Check back regularly for real-time updates on one of biotech's most dynamic gene therapy innovators.
uniQure announced positive interim data from Phase I/II trials of AMT-130 for Huntington's disease, showing a statistically significant, dose-dependent slowing in disease progression. At 24 months, high-dose patients demonstrated an 80% slowing of disease progression compared to a propensity score-weighted external control. Additionally, a significant reduction in CSF neurofilament light protein (NfL) levels was observed. The trials also indicated that AMT-130 remains well-tolerated with no new serious adverse events. uniQure aims to meet with the FDA in the second half of 2024 to discuss expedited clinical development pathways for AMT-130.
uniQure announced the sale of its manufacturing facility in Lexington, Massachusetts, to Genezen for $25 million, consisting of $12.5 million in Series C Preferred Stock and a $12.5 million convertible note. This divestiture will allow uniQure to reduce its annual cash burn by $40 million and is part of a broader strategy to cut operating expenses. Genezen will now manufacture HEMGENIX® and support uniQure's pipeline programs, with the majority of current employees transitioning to Genezen. The deal is expected to close in early Q3 2024, and uniQure will use the proceeds to repay $50 million in debt, further saving on interest expenses.
Genezen announced its acquisition of uniQure's commercial gene therapy operations in Lexington, MA. The acquisition includes a state-of-the-art, commercially-licensed viral vector manufacturing facility, enhancing Genezen's ability to provide late-phase and commercial gene therapy development services. The Lexington site will become Genezen's global AAV center of excellence, complementing its existing operations in Indianapolis. Genezen will also enter into strategic supply agreements for uniQure's clinical portfolio and CSL's HEMGENIX® product. The transaction is supported by additional growth equity from Ampersand Capital Partners.
uniQure has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy, AMT-130, for Huntington’s disease. This designation is based on 24-month interim data from Phase I/II clinical trials released in December 2023 and represents a significant milestone.
AMT-130 is the first therapy for Huntington’s disease to receive RMAT designation, facilitating closer collaboration with the FDA and potentially expediting its development and approval process. This decision underscores AMT-130's potential to address the unmet medical needs of Huntington’s disease patients.
uniQure plans to present updated interim data from ongoing Phase I/II studies in mid-2024, including follow-up results on 29 treated patients, 21 of whom will have data spanning at least two years.
uniQure N.V. reported its first quarter 2024 financial results and recent progress, focusing on advancing AMT-130 for Huntington's disease and initiating new Phase I/II trials for Fabry disease, ALS, and MTLE. The company is on track to interact with the FDA in Q2 2024 and reduce expenses to increase shareholder value. Financially, uniQure's revenues increased to $8.5 million, with a cash position of $555.7 million as of March 31, 2024.
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