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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.
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 80% reduction in disease progression compared to a control group, as measured by the Unified Huntington’s Disease Rating Scale (cUHDRS).
Additionally, levels of neurofilament light protein (NfL) in cerebrospinal fluid, a key biomarker of neurodegeneration, decreased markedly in patients receiving the therapy.
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.
QURE Price Action: UniQure shares were up by 61.2% at $10.78 according to Benzinga Pro.
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Image: Shutterstock/ solarseven.
Posted In: QURE
