24岁的徐清(化名)从未踢过一场足球，尽管他房间里贴满了球队海报。作为血友病B患者，他常年戴着 护膝，每周至少注射三次凝血因子，一年100多针维系着脆弱的生活平衡。 血友病B是一种由于凝血因子Ⅸ基因缺陷引起的X染色体连锁的隐性遗传性出血性疾病，患者要时刻防 止出血风险，常被称为"玻璃人"，因为，他们的人生宛如脆弱的玻璃艺术品，轻轻一触，便可能支离破 碎。 传统治疗要求患者终身定期输注凝血因子Ⅸ，且需每周多次静脉注射。"扎到后面，血管不够用了。"徐 清坦言，病友们急需更有效的治疗药物。 好消息在2025年4月迎来。国家药监局官网更新了公告，让中国数万名血友病B患者看到了终结终生注 射治疗的希望——波哌达可基注射液(商品名：信玖凝)正式获批上市，该药物是我国首个腺相关病毒 (AAV)基因治疗产品，用于中重度血友病B(先天性凝血因子IX缺乏症)成年患者的治疗。 据信念医药联合创始人、董事长兼首席科学家肖啸博士介绍，国产的波哌达可基注射液使用工程化改造 的AAV843载体，实现了更高的肝脏靶向性和更快的基因表达速度——临床数据显示，患者在单次给药 后第三天即可检测到凝血因子Ⅸ活性显著提升，一周内即达峰值，且该表达水平 ...

Core Insights - uniQure N.V. is advancing its investigational gene therapy AMT-130 for Huntington's disease, with a Biologics License Application (BLA) submission planned for the first quarter of 2026, following alignment with the FDA on key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information [1][2][8] Regulatory Update - The FDA has supported the use of the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an acceptable clinical endpoint for accelerated approval, with the primary efficacy analysis focusing on the 3-year change in cUHDRS in high-dose AMT-130 patients compared to an external control arm [3][4] - The ENROLL-HD dataset, which includes approximately 33,000 patients, will serve as the external control dataset for the primary analysis, enhancing the robustness of the statistical analysis plan due to its larger sample size and lower attrition rates [4][5] Chemistry, Manufacturing and Controls (CMC) - The FDA has agreed that the validation of the AMT-130 manufacturing process can leverage prior knowledge from the HEMGENIX® process, along with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch [6][7] Next Steps - The company plans to submit an updated statistical analysis plan to the FDA in Q2 2025, initiate the PPQ run and present topline Phase I/II data in Q3 2025, hold a pre-BLA meeting in Q4 2025, and submit the BLA in Q1 2026 with a request for priority review designation [15] Clinical Program Overview - uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to evaluate the safety and efficacy of AMT-130, with a total of 26 patients in the U.S. study and 13 patients in the European study, exploring both low and high doses [10][11] - AMT-130 has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and Breakthrough Therapy designation, marking it as the first therapy for Huntington's disease to achieve RMAT designation [11] Huntington's Disease Context - Huntington's disease is a rare neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies currently available to slow its progression [12] Company Background - uniQure is focused on gene therapy, with a pipeline that includes treatments for Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease, building on its historic achievement in gene therapy for hemophilia B [13]

~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington’s disease. Following recent Typ ...