~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~ ~ Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to baseline at 24 months in patients treated with AMT-130; Mean CSF NfL levels for both ...

Strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. BOSTON and INDIANAPOLIS, July 1, 2024 /PRNewswire/ -- Genezen, a best-in-class gene therapy CDMO, today announced an agreement to acquire uniQure's (Nasdaq: QURE) commercial gene therapy operations in Lexington, MA, bringing a strategic manufacturing facility and a world-class team of employees to the company. Under the terms of the agreement, Gene ...

~ Proposed divestiture streamlines uniQure’s focus and represents an important milestone in its ongoing effort to significantly reduce operating expenses ~ ~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure’s pipeline programs as a preferred customer ~ ~ uniQure to reduce cash burn by $40 million per year and receive total consideration of $25 million ~ ~ uniQure’s comprehensive review of operations and options to reduce expenses is ongoing and expected to be complete in the ...

文章核心观点 - 公司出售其位于马萨诸塞州的全球制造设施给Genezen公司 [1][2][3] - 该交易将使公司每年减少约4000万美元的现金消耗 [4][5] - 公司正在进一步审查业务以识别其他成本削减机会,并预计将在2024年第三季度完成 [4] 根据相关目录分别进行总结 交易细节 - Genezen以2500万美元的总对价收购公司的商业规模基因疗法制造设施 [2] - 其中包括1250万美元的新发行C系列优先股和1250万美元的可转换票据 [2] - 公司首席执行官将加入Genezen董事会 [2] - Genezen将负责为CSL制造HEMGENIX®的全球商业供应,并为公司的试验性基因疗法提供开发和制造服务 [3] - 公司和CSL将获得优先客户地位,大部分员工将被聘用到Genezen以确保顺利过渡 [3] 交易背景和目标 - 该交易实现了公司的关键战略目标,即大幅降低成本的同时保持开发和潜在商业化基因疗法产品候选物的能力 [4] - 公司正在继续密切审查业务,以识别其他成本削减机会,并预计将在2024年第三季度完成 [4] - 更精简的公司将增强其战略灵活性,推进其为患者提供转型性药物的使命 [4] 交易影响 - 交易完成后,公司预计将偿还5000万美元的未偿债务,从而产生额外的利息费用节省 [5] - 该交易将使公司每年减少约4000万美元的现金消耗 [4]

~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~ ~ AMT-130 is the first therapeutic candidate to receive RMAT Designation for Huntington's disease ~ ~ Receiving RMAT designation enables increased collaboration with the FDA on regulatory approval planning, in addition to the opportunity for expedited product development ~ LEXINGTON, Mass. and AMSTERDAM, June 03, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advanci ...

Exhibit 99.1 uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress ~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~ ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands Not ...

Exhibit 99.1 uniQure Announces 2023 Financial Results and Highlights Recent Company Progress ~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial tempor ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Jurisdiction of incorpor ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands ...