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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

在亨廷頓基因治療試驗的低劑量隊列數據公佈後,UnQure股價上漲
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.納斯達克公司(Temasek Holdings:QUURE)宣佈了正在進行的AMT-130基因療法治療亨廷頓病1/2期低劑量隊列試驗的10名患者的安全性和生物標誌物數據。
  • 在美國的這項隨機、盲目臨牀試驗中,10名入選患者中有6名接受了AMT-130,4名接受了模擬外科手術。
  • 在較低劑量的6×1012載體基因組治療患者中,AMT-130的耐受性通常很好。
  • 在這些患者中,沒有與AMT-130相關的嚴重不良事件的報道。
  • 相關: 瑞穗對UniQure即將發佈的小劑量亨廷頓基因療法試驗數據的評論.
  • 腦脊液NFL是神經元損傷的關鍵生物標誌物,在AMT-130手術後,其測量結果如預期般增加,並在12個月時接近基線。
  • 可評估的治療患者的腦脊液中mHTT蛋白的測量結果顯示,在12個月中與基線相比有所下降。
  • 生物標記物和臨牀數據,包括低劑量美國隊列的24個月隨訪和預計2023年上半年的高劑量美國隊列12個月的隨訪。
  • 價格行動:在週四最後一次檢查的市場時段,Qure的股價上漲了24.40%,至19.15美元。

譯文內容由第三人軟體翻譯。


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