UniQure's UQ1.F experimental gene therapy for Huntington's disease slowed progression of the brain disorder by 75% in an early-to-mid stage study, it said on Wednesday.

Huntington's is a rare inherited brain disorder, which steadily worsens and typically leads to death 10 to 30 years after symptoms begin. There are no FDA-approved treatments for the condition.

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose, based on a widely used clinical scale.

It also slowed decline of functional abilities in patients by 60%, a key secondary goal of the trial.

"These groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need

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