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    SPK-10001

    Other names: RG6662

    Modality: Huntingtin mRNA gene therapy

    Disease State: Huntington’s Disease (HD)

    Summary

    SPK-10001 is an investigational gene therapy designed to deliver microRNA, with a proposed mechanism of action to downregulate both mutant huntingtin protein (mHtt) and wild-type huntingtin protein (wHtt) in transduced neurons.1

    Clinical Trials

    SPK-10001
    NCT ID Study Phase Enrollment Status Study Title
    NCT06826612 Phase I / Phase II Recruiting A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease View details
    1. Roche. Product development pipeline. Roche. Updated October 23, 2025. Accessed January 26, 2026. https://www.roche.com/​solutions/​pipeline

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    TOP

    • HD
      Huntington’s disease

    • mRNA
      Messenger RNA

    • mHtt
      Mutant huntingtin protein

    • wHtt
      Wild-type huntingtin protein

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