A uniQure gene therapy slowed progression of Huntington’s disease by 75% after three years, statistically significant clinical trial results the company says will support plans for a regulatory submission next year. If approved, the gene therapy would become the first treatment for this progressive neurodegenerative disorder.

The uniQure gene therapy, AMT-130, is not a cure for Huntington’s. But the results reported Wednesday indicate disease-modifying effects along with a manageable safety profile — benchmarks that have eluded many drug research efforts in this rare disease.

Huntington’s stems from mutations to the gene that codes for huntingin, a protein important for neuronal function. The buildup of mutant huntingin in the brain damages and kills neurons. Disease symptoms include i

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