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UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

在亨廷顿基因治疗试验的低剂量队列数据公布后,UnQure股价上涨
Benzinga Real-time News ·  2022/06/24 02:10
  • UniQure N.V. (NASDAQ:QURE) announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase 1/2 trial of AMT-130 gene therapy for Huntington's disease. 
  • Six of the ten enrolled patients received AMT-130, and four received an imitation surgical procedure in this randomized, blinded clinical trial in the U.S.
  • AMT-130 was generally well-tolerated in treated patients at the lower dose of 6x1012 vector genomes.
  • No serious adverse events related to AMT-130 were reported in these patients.
  • Related: Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data.
  • Measurements of CSF NfL, a key biomarker of neuronal damage, increased as expected following the AMT-130 surgical procedure and approached baseline at 12 months.
  • Measurements of mHTT protein in the CSF of evaluable treated patients showed decreases compared to baseline through 12 months.
  • Biomarker and clinical data, including 24-month follow-up in the low-dose U.S. cohort and 12-month follow-up in the high-dose U.S. cohort expected in 1H of 2023.
  • Price Action: QURE shares are 24.40% at $19.15 during the market session on the last check Thursday.
  • UnQure N.V.纳斯达克公司(Temasek Holdings:QUURE)宣布了正在进行的AMT-130基因疗法治疗亨廷顿病1/2期低剂量队列试验的10名患者的安全性和生物标志物数据。
  • 在美国的这项随机、盲目临床试验中,10名入选患者中有6名接受了AMT-130,4名接受了模拟外科手术。
  • 在较低剂量的6×1012载体基因组治疗患者中,AMT-130的耐受性通常很好。
  • 在这些患者中,没有与AMT-130相关的严重不良事件的报道。
  • 相关: 瑞穗对UniQure即将发布的小剂量亨廷顿基因疗法试验数据的评论.
  • 脑脊液NFL是神经元损伤的关键生物标志物,在AMT-130手术后,其测量结果如预期般增加,并在12个月时接近基线。
  • 可评估的治疗患者的脑脊液中mHTT蛋白的测量结果显示,在12个月中与基线相比有所下降。
  • 生物标记物和临床数据,包括低剂量美国队列的24个月随访和预计2023年上半年的高剂量美国队列12个月的随访。
  • 价格行动:在周四最后一次检查的市场时段,Qure的股价上涨了24.40%,至19.15美元。

译文内容由第三方软件翻译。


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