AMT-130

Kessler Topaz Meltzer & Check, LLP 已代表在 2025 年 9 月 24 日至 2025 年 10 月 31 日期间购买了 uniQure N.V.（NASDAQ: QURE）股票的投资者提起了一项证券欺诈集体诉讼。诉讼指控 uniQure 在其亨廷顿舞蹈症基因治疗药物 AMT-130 及其临床试验结果方面误导了投资者。投资者可以在 2026 年 4 月 13 日之前申请成为主原告，并被鼓励联系 KTMC 寻求法律帮助您在 2025 年 9 月 24 日至 2025 年 10 月 31 日期间购买了 uniQure 普通股吗？ 受影响的 uniQure N.V. 投资者摘要 谁：uniQure N.V. (NASDAQ: QURE) 什么：提起证券欺诈集体诉讼 集体诉讼期间：2025 年 9 月 24 日至 2025 年 10 月 31 日 申请主原告资格的截止日期：2026 年 4 月 13 日 主要诉讼指控：关于公司亨廷顿病基因治疗药物的重大虚假陈述和/或遗漏。 投资者行动：联系 Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) 以获取无成本的索赔选项 , /PRNewswire/ -- Kessler Topaz Meltzer & Check, LLP 通知投资者，该公司已代表在 2025 年 9 月 24 日至 2025 年 10 月 31 日期间购买或获得 uniQure 普通股的投资者，向 uniQure N.V. (NASDAQ: QURE) 提起证券欺诈集体诉讼。此案标题为 Scocco v. uniQure N.V., et al.，案件编号为 1:26-cv-01124，已在纽约南区美国地方法院提起。 重要截止日期提醒：在集体诉讼期间购买或以其他方式获得 uniQure 普通股的投资者，最迟可于 2026 年 4 月 13 日向法院申请担任主原告。 联系 KTMC 讨论您的法律权利： 如果您购买或获得了 uniQure 普通股并遭受损失，鼓励您联系 KTMC 律师 Jonathan Naji，Esq.，联系方式： 📞 (484) 270-1453 📧 [email protected] 🌐 https://www.ktmc.com/qure-uniqure-nv-class-action-lawsuit?utm_source=PR_Newswire&utm_medium=pressrelease&utm_campaign=qure&mktm=PR 与律师交谈没有费用或义务。 **UNIQURE N.V.**集体诉讼 - 投诉指控摘要： uniQure 是一家开发罕见疾病基因治疗的生物技术公司，包括亨廷顿病（HD）。uniQure 的主要药物候选者是 AMT-130，一种新型基因治疗，旨在减缓 HD 的进展。在集体诉讼期间，uniQure 误导投资者关于其 AMT-130 的 I/II 期临床试验（关键研究）以及 uniQure 向 FDA 提交生物制剂许可申请（BLA）以获得使用 AMT-130 治疗 HD 患者的批准的前景和时间表。 投诉指控，在集体诉讼期间，被告做出了重大虚假和/或误导性陈述，并未披露关于 uniQure 的业务和运营的重大不利事实。具体而言，被告误导和/或未披露： (1) uniQure 的关键研究设计——包括将关键研究结果与 ENROLL-HD 外部历史数据集进行比较——并未得到 FDA 的完全批准； (2) 被告淡化了尽管关键研究结果声称非常成功，uniQure 仍需推迟其 BLA 时间表以进行额外研究以补充其 BLA 提交的可能性； (3) 因此，被告关于 uniQure 的业务、运营和前景的陈述缺乏合理依据。 QURE 投资者现在可以做什么： 在 2026 年 4 月 13 日前申请担任主原告。 联系 KTMC 进行免费案件评估。 保留自己选择的律师或不采取任何行动。 UNIQURE N.V.投资者的主原告流程： uniQure 投资者可在 2026 年 4 月 13 日之前 通过 Kessler Topaz Meltzer & Check, LLP 或其他律师寻求被任命为集体的主原告代表，或选择不采取任何行动，保持缺席集体成员身份。主原告是代表所有集体成员指导诉讼的代表方。主原告通常是具有最大财务利益的投资者或小组投资者，并且也符合拟议集体投资者的典型性。主原告选择律师代表主原告和集体，这些律师如果获得法院批准，则为主律师或集体律师。您分享任何索赔的能力不受是否担任主原告的决定影响。 Kessler Topaz Meltzer & Check, LLP 鼓励 uniQure 投资者联系该公司以获取更多信息。 关于 KESSLER TOPAZ MELTZER & CHECK, LLP (KTMC)： Kessler Topaz Meltzer & Check, LLP (KTMC) 是一家领先的美国原告方律师事务所，专注于证券欺诈集体诉讼和全球投资者保护。该事务所代表个人投资者以及机构，如主要养老金基金、资产管理公司和国际投资者。KTMC 在证券诉讼中主导了一些最大的索赔，并获得了同行和法律媒体的众多荣誉，包括《国家法律杂志》的原告热名单和原告法的开拓者，BTI 咨询集团的最受畏惧律师事务所荣誉名单，《法律智库》的年度集体诉讼律师事务所，以及《Lawdragon》的领先原告金融律师和《Law360》的原告律师行业巨头。该事务所在宾夕法尼亚州和加利福尼亚州设有办事处，业务遍及全球。有关 Kessler Topaz Meltzer & Check, LLP 的更多信息，请访问 www.ktmc.com。 联系方式： Jonathan Naji, Esq. (484) 270-1453 280 King of Prussia Road Radnor, PA 19087 [email protected] 在某些司法管辖区可能被视为律师广告。过去的结果并不保证未来的结果。 来源 Kessler Topaz Meltzer & Check, LLP 您在 2025 年 9 月 24 日至 2025 年 10 月 31 日期间购买了 uniQure 普通股吗？ 受影响的 uniQure N.V. 投资者摘要 谁：uniQure N.V. (NASDAQ: QURE) 什么：提起证券欺诈集体诉讼 集体诉讼期间：2025 年 9 月 24 日至 2025 年 10 月 31 日 申请主原告资格的截止日期：2026 年 4 月 13 日 主要诉讼指控：关于公司亨廷顿病基因治疗药物的重大虚假陈述和/或遗漏。 投资者行动：联系 Kessler Topaz Meltzer & Check, LLP (www.ktmc.com) 以获取无成本的索赔选项 , /PRNewswire/ -- Kessler Topaz Meltzer & Check, LLP 通知投资者，该公司已代表在 2025 年 9 月 24 日至 2025 年 10 月 31 日期间购买或获得 uniQure 普通股的投资者，向 uniQure N.V. (NASDAQ: QURE) 提起证券欺诈集体诉讼。此案标题为 Scocco v. uniQure N.V., et al.，案件编号为 1:26-cv-01124，已在纽约南区美国地方法院提起。 重要截止日期提醒：在集体诉讼期间购买或以其他方式获得 uniQure 普通股的投资者，最迟可于 2026 年 4 月 13 日向法院申请担任主原告。 联系 KTMC 讨论您的法律权利： 如果您购买或获得了 uniQure 普通股并遭受损失，鼓励您联系 KTMC 律师 Jonathan Naji，Esq.，联系方式： 📞 (484) 270-1453 📧 [email protected] 🌐 https://www.ktmc.com/qure-uniqure-nv-class-action-lawsuit?utm_source=PR_Newswire&utm_medium=pressrelease&utm_campaign=qure&mktm=PR 与律师交谈没有费用或义务。 **UNIQURE N.V.**集体诉讼 - 投诉指控摘要： uniQure 是一家开发罕见疾病基因治疗的生物技术公司，包括亨廷顿病（HD）。uniQure 的主要药物候选者是 AMT-130，一种新型基因治疗，旨在减缓 HD 的进展。在集体诉讼期间，uniQure 误导投资者关于其 AMT-130 的 I/II 期临床试验（关键研究）以及 uniQure 向 FDA 提交生物制剂许可申请（BLA）以获得使用 AMT-130 治疗 HD 患者的批准的前景和时间表。 投诉指控，在集体诉讼期间，被告做出了重大虚假和/或误导性陈述，并未披露关于 uniQure 的业务和运营的重大不利事实。具体而言，被告误导和/或未披露： (1) uniQure 的关键研究设计——包括将关键研究结果与 ENROLL-HD 外部历史数据集进行比较——并未得到 FDA 的完全批准； (2) 被告淡化了尽管关键研究结果声称非常成功，uniQure 仍需推迟其 BLA 时间表以进行额外研究以补充其 BLA 提交的可能性； (3) 因此，被告关于 uniQure 的业务、运营和前景的陈述缺乏合理依据。 QURE 投资者现在可以做什么： 在 2026 年 4 月 13 日前申请担任主原告。 联系 KTMC 进行免费案件评估。 保留自己选择的律师或不采取任何行动。 UNIQURE N.V.投资者的主原告流程： uniQure 投资者可在 2026 年 4 月 13 日之前 通过 Kessler Topaz Meltzer & Check, LLP 或其他律师寻求被任命为集体的主原告代表，或选择不采取任何行动，保持缺席集体成员身份。主原告是代表所有集体成员指导诉讼的代表方。主原告通常是具有最大财务利益的投资者或小组投资者，并且也符合拟议集体投资者的典型性。主原告选择律师代表主原告和集体，这些律师如果获得法院批准，则为主律师或集体律师。您分享任何索赔的能力不受是否担任主原告的决定影响。 Kessler Topaz Meltzer & Check, LLP 鼓励 uniQure 投资者联系该公司以获取更多信息。 关于 KESSLER TOPAZ MELTZER & CHECK, LLP (KTMC)： Kessler Topaz Meltzer & Check, LLP (KTMC) 是一家领先的美国原告方律师事务所，专注于证券欺诈集体诉讼和全球投资者保护。该事务所代表个人投资者以及机构，如主要养老金基金、资产管理公司和国际投资者。KTMC 在证券诉讼中主导了一些最大的索赔，并获得了同行和法律媒体的众多荣誉，包括《国家法律杂志》的原告热名单和原告法的开拓者，BTI 咨询集团的最受畏惧律师事务所荣誉名单，《法律智库》的年度集体诉讼律师事务所，以及《Lawdragon》的领先原告金融律师和《Law360》的原告律师行业巨头。该事务所在宾夕法尼亚州和加利福尼亚州设有办事处，业务遍及全球。有关 Kessler Topaz Meltzer & Check, LLP 的更多信息，请访问 www.ktmc.com。 联系方式： Jonathan Naji, Esq. (484) 270-1453 280 King of Prussia Road Radnor, PA 19087 [email protected] 在某些司法管辖区可能被视为律师广告。过去的结果并不保证未来的结果。 来源 Kessler Topaz Meltzer & Check, LLP

Clinical Advisory Board formed to support progression of HRN001 into first-in-human studies in 2027 Dr Andy Billinton, CSO will present preclinical data at the upcoming CHDI Foundation Huntington’s Disease Therapeutics Conference 16 February 2026: Harness Therapeutics (‘Harness’), a biotechnology company unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases, today announces the nomination of HRN001 as its lead drug candidate for Huntington's disease and the formation of a clinical advisory board to support the programme's advancement towards clinical evaluation. Huntington’s disease (HD) is a devastating, inherited neurodegenerative disorder that leads to progressive cognitive, psychiatric and motor decline, with death often occurring within 15 years of symptom onset. Despite significant advances in understanding HD disease biology, there are currently no approved disease-modifying treatments available. HD is caused by the expansion of CAG repeats in the huntingtin (HTT) gene. Ongoing somatic expansion of these repeats is now recognised as a key driver of disease onset and progression. FAN1 nuclease has emerged as one of the most compelling targets to suppress somatic expansion, demonstrating the strongest genetic association to disease onset in genome-wide association studies. Our first-in-class candidate HRN001 is a potent and specific antisense oligonucleotide targeting FAN1, designed to drive controlled upregulation of this key DNA repair nuclease. It leverages Harness' proprietary MISBA® (microRNA site blocking ASO) platform, which enables precise upregulation of target protein levels without risk of over-expression. HRN001 has demonstrated robust upregulation of FAN1 and slowing of somatic expansion in models of HD, as well as favourable PK and tolerability characteristics. Preclinical development will continue throughout 2026 to support clinical entry in 2027. Harness is exploring the potential of the MISBA® platform in other triplet repeat disorders and across a broader pipeline of neurodegenerative disorders. To support the progression of HRN001 towards the clinic, Harness has established a clinical advisory board (CAB) comprising leading experts in the HD field, including: Dr. Irina Antonijevic (Chair) – Chief Medical Officer, Trace Neuroscience Dr. Anne Rosser – Professor of Clinical Neuroscience, Cardiff University Dr. Jeffrey Long – Professor of Psychiatry and Biostatistics, University of Iowa Health Care Dr. Ralf Reilmann – Founding Director and Chief Executive Officer, George-Huntington-Institute Dr. Roger Barker – Professor of Clinical Neuroscience, University of Cambridge Dr. Sarah Tabrizi – Professor of Clinical Neurology, University College London Dr. William Gray – Professor of Functional Neurosurgery, Cardiff University The CAB will provide strategic guidance on clinical development, trial design, and translational strategy as the programme advances towards the clinic. Dr Andy Billinton, Chief Scientific Officer of Harness Therapeutics, will present the company's work in HD at the upcoming annual CHDI Huntington’s Disease Therapeutics Conference in Palm Springs, California, 23rd-26th February, providing further insights into the scientific rationale and development progress for HRN001. Dr Jan Thirkettle, CEO of Harness Therapeutics, commented: "The nomination of HRN001 represents a pivotal milestone for Harness and underscores our commitment to the Huntington’s disease community. By precisely upregulating FAN1, a target with compelling genetic validation in delaying disease onset, HRN001 represents a differentiated, first-in-class therapeutic approach for addressing somatic expansion, a fundamental driver of disease progression. The formation of a Clinical Advisory Board brings deep clinical and translational expertise to the programme. The CAB will work closely with Harness as we advance HRN001 towards the clinic and seek to deliver a truly disease-modifying therapy for patients and families living with Huntington’s disease." Dr Irina Antonijevic, Chair of Harness Therapeutics’ Clinical Advisory Board, added: "FAN1 is one of the most compelling and consistently validated genetic modifiers of Huntington’s disease identified to date, with a clear mechanistic link to somatic expansion and disease progression. Harness' approach with HRN001 offers a novel and highly targeted way to therapeutically modulate this pathway. We look forward to advising the Company, as it advances HRN001 toward the clinic, translating this promising science into a clinical programme that could meaningfully alter the course of this devasting disease." CHDI presentation details Title: Upregulation of FAN1 with ASOs as a potential therapeutic strategy for triplet repeat disorders Session: Somatic Instability/Mismatch Repair Date: Tuesday 24 February Presenter: Dr Andy Billinton, CSO of Harness Therapeutics CAB biographies Dr. Irina Antonijevic (Chair) is an experienced clinician and drug development leader, widely recognized for her leadership in advancing innovative therapies from early development through clinical stages, particularly in areas with high unmet medical needs such as neurodegenerative and psychiatric disorders. She currently serves as Chief Medical Officer at Trace Neuroscience, bringing over three decades of experience in clinical psychiatry, neurology, academic research, and therapeutic drug development. Dr. Antonijevic is a board-certified physician trained in both psychiatry and neurology, with deep expertise in translational medicine and clinical development strategies. Dr. Anne Rosser is Professor of Clinical Neuroscience at Cardiff University and an Honorary Consultant Neurologist at the University Hospital of Wales, leading the South Wales clinical service for Huntington’s disease. She co-leads the Cardiff University Brain Repair Group, a multidisciplinary research team focused on developing cell-based and regenerative therapies for neurodegenerative diseases. Dr. Rosser previously served as Chair of the European Huntington’s Disease Network (EHDN) and is currently Chair of the Scientific Committee of the Lister Institute of Preventive Medicine. Dr. Jeffrey D. Long is Professor of Psychiatry and Biostatistics at the University of Iowa Health Care / Carver College of Medicine where he applies statistical modeling to neurodegenerative diseases, with a special emphasis on Huntington’s disease. He develops and refines disease progression indexes, critical for understanding natural disease trajectories and planning clinical trials. Dr Long also serves as Lead Biostatistician for NIH-funded longitudinal studies, such as REDICT-HD, and plays a key role as Head Biostatistician in the Data Coordinating Center of the NeuroNEXT clinical trials network. Dr. Ralf Reilmann is Founding Director and Chief Executive Officer of the George-Huntington-Institute (GHI) in Münster, Germany, a private research institute devoted to clinical care and research in Huntington’s disease. Under his leadership, the Institute participates in key Huntington’s observational studies including ENROLL-HD and clinical trials exploring disease-modifying therapies and gene therapy approaches. Dr. Reilmann is a board-certified neurologist, widely recognized for his commitment to improving trial outcomes, patient care, and global collaboration in Huntington’s research. Dr. Roger Barker Professor of Clinical Neuroscience at the University of Cambridge and Honorary Consultant Neurologist at Addenbrooke’s Hospital, Cambridge. He serves as Lead Academic Scientist for the Alzheimer’s Research UK Drug Discovery Institute in Cambridge and Lead Consultant for the East of England Huntington’s Disease Service. His research focuses on clinical characterization of neurodegenerative disorders and the development of new therapeutic approaches, especially cell- and gene-based treatments. Dr. Barker has led and coordinated major research programmes, including European projects and stem cell trials for Parkinson's disease as well as a range of trials in Huntington's disease. Dr. Sarah Tabrizi is Professor of Clinical Neurology at University College London, internationally recognised for her research in neurodegenerative diseases, particularly Huntington’s disease. She holds several prestigious roles across UCL’s neuroscience community and the UK's top clinical research institutions recognised with many major awards. Her work has transformed the Huntington’s field, establishing frameworks for disease staging, biomarker development, and therapeutic trials that serve as models for other neurodegenerative conditions. Dr. Tabrizi’s bench-to-bedside approach integrates deep biological insight with clinical trials to bring hope to patients with previously untreatable conditions. Dr. William Gray is Professor of Functional Neurosurgery at Cardiff University and Honorary Consultant Neurosurgeon at the University Hospital of Wales. He brings dual expertise in clinical neurosurgery and neuroscience research, especially focused on disorders like epilepsy, Parkinson’s disease, Huntington’s disease, and frontotemporal dementia. He serves as Director of the Advanced NeuroTherapies Centre (ANTC), developing and delivering emerging therapies directly to the brain, is the Academic Lead in Neurosurgery in Wales, and Head of the Brain Repair and Intracranial Neurotherapeutics (BRAIN) Unit. Dr. Gray is the UK Chief Investigator for the uniQure AMT-130 gene therapy trial in Huntington’s disease and Principal Investigator for gene therapy trials in frontotemporal dementia and Parkinson’s disease. He also co-leads the European Huntington’s Disease Network ATMP Working Group and chairs its Surgical Delivery Task Force. Harness Therapeutics is a biotechnology company focused on unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases. Its MISBA® platform technology enables precise upregulation of target protein levels by modulating mechanisms controlling protein synthesis. Harness’ recently announced MISBA® Duo technology enables the design of single constructs which simultaneously upregulate one target whilst downregulating a second. Using its deep understanding of post-transcriptional regulation and sophisticated neuron-based models, Harness’ approach allows drugging of potentially disease-modifying targets, which would not be addressable using gene therapy or other modalities. Its lead candidate in Huntington’s Disease, HRN001, targets FAN1 nuclease, a key protective protein, shown to slow disease progression. Its pipeline also includes programmes addressing compelling first in class targets for ALS, Alzheimer's Disease, and Parkinson’s Disease. Harness has assembled a world-class team, supported by leading experts in neurodegeneration and RNA biology, and is based in Cambridge, UK. Its leading life science investors include the foundational investors Takeda Ventures and SV Health Investors’ Dementia Discovery Fund, alongside Epidarex Capital and Ono Ventures Investment. The content above comes from the network. if any infringement, please contact us to modify.