Why Are UniQure Shares Surging Today?
Why Are UniQure Shares Surging Today?
UniQure N.V. (NASDAQ:QURE) shares are skyrocketing Wednesday following the release of promising interim data from its Phase I/II clinical trials of AMT-130, a gene therapy targeting Huntington's disease.
UniQure N.V.（纳斯达克：QURE）股票正在飙升，这是因为其AMt-130的I/II期临床试验发布了有希望的中期数据，该基因疗法旨在治疗亨廷顿病。
What's Going On: The latest results, which include up to 24 months of follow-up data from 29 patients across the U.S. and Europe, demonstrated a significant slowing of disease progression. Patients treated with AMT-130 exhibited an impressive 80% reduction in disease progression compared to a control group, as measured by the Unified Huntington's Disease Rating Scale (cUHDRS).
最新的结果显示，来自美国和欧洲的29名患者的长达24个月的随访数据显示，疾病进展显著减缓。与对照组相比，接受AMt-130治疗的患者，根据统一亨廷顿病评分量表(cUHDRS)所测量的疾病进展率减少了令人印象深刻的80%。
Additionally, levels of neurofilament light protein (NfL) in cerebrospinal fluid, a key biomarker of neurodegeneration, decreased markedly in patients receiving the therapy.
此外，接受该疗法治疗的患者的脑脊液中神经丝轻链蛋白（NfL）水平显著下降，这是神经退行性疾病的关键生物标志物。
What Else: The company's achievement of FDA's Regenerative Medicine Advanced Therapy (RMAT) designation further underscores the therapy's potential to address unmet medical needs expediently and prepares for discussions with the FDA in late 2024 to explore accelerated approval pathways.
公司获得的FDA再生医学先进疗法（RMAT）认证进一步突显了该疗法解决未满足的医疗需求的潜力，并为2024年底与FDA探讨加速批准途径做好了准备。
QURE Price Action: UniQure shares were up by 61.2% at $10.78 according to Benzinga Pro.
UniQure股价上涨61.2%，至10.78美元，据Benzinga Pro报道。
Image: Shutterstock/ solarseven.
图片：shutterstock / solarseven。