There’s a fine line between hope and disappointment in the tumultuous world of gene therapy R&D. Just ask Pfizer, whose Duchenne muscular dystrophy treatment was discontinued last summer. Or Sarepta, whose own DMD therapy Elevidys met with safety concerns this year that drew regulators’ attention. Or Johnson & Johnson’s late-stage failure in a rare eye disease earlier in 2025.

But scientific progress is a force to be reckoned with, and the Huntington’s disease community is hinging hope on a gene therapy from uniQure that has shown in a small study to be a potential breakthrough. Still, experts are urging caution, especially as the jury is still out with respect to how the gene therapy will perform in larger, peer-reviewed clinical trials.

Today, we’re exploring what uniQure’s early success means for the Huntington’s patient community and R&D in the space.

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