Encouraging Biomarker Data Lift UniQure’s Huntington’s Disease Hopes

Results Exceeded Expectations

Hopes rise as first four evaluable patients experienced no serious adverse events, and a sustained decline in level of key Huntington’s disease protein with the novel gene therapy product.

Encouraging early safety and biomarker data from uniQure’s gene therapy candidate, AMT-130, for Huntington’s disease have lifted hopes of progress against the disease.

The readout from 10 patients in the low-dose cohort of an ongoing Phase I/II trial in patients with early-stage Huntington’s disease showed no serious adverse events linked to the therapy over 12 months

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