Experimental gene therapy found to slow Huntington’s disease progression, company says

UniQure said it plans to submit its data on the new therapy to the US Food and Drug Administration in the first quarter of 2026. Kristoffer Tripplaar/Sipa/AP

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