STAT+: Why a Huntington’s patient isn’t fazed by the waning effect of UniQure’s gene therapy — SkimNews
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- Lauren Holder, a Huntington's disease community advocate living with early-stage HD, told STAT she isn't fazed by the apparent waning of UniQure's gene therapy effect.
- AMT-130, UniQure's one-time gene therapy for Huntington's, slowed disease progression by 44% in a four-year analysis of 12 treated patients.
- The same therapy had shown 75% slowing of disease progression at the three-year update reported last year.
- The drop from 75% to 44% between years three and four hasn't alarmed UniQure but has prompted outside questions about the therapy's durability.
- Huntington's disease is a progressive, fatal neurodegenerative condition, and Holder's willingness to accept a potentially time-limited benefit underscores the desperation in the patient community for any disease-modifying option.
Why it matters: The durability question is the central commercial and clinical hurdle for one-time gene therapies in neurodegenerative disease. UniQure's confidence in AMT-130's 44% four-year slowing stands against outside skepticism about whether the benefit holds, and a patient community advocate publicly accepting a time-limited effect signals the threshold families are willing to accept for a fatal, untreatable condition.
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