CNN —

An experimental gene therapy was found to slow the progression of Huntington's disease in a "pivotal" Phase 1/2 study , according to drugmaker uniQure, marking a major step toward a potential first genetic treatment for the condition.

The Amsterdam-based company announced Wednesday that patients who received a high dose of its AMT-130 therapy for Huntington's disease saw disease progression slow by 75% after 36 months.

The treatment was "generally well-tolerated," with a "manageable" safety profile, the company said. The full study results have not yet been published in a peer-reviewed journal.

UniQure said it plans to submit its data on AMT-130 to the U.S. Food and Drug Administration in the first quarter of 2026. If the treatment is approved by the FDA, it could launch later

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