Experimental gene therapy slows Huntington’s progression in trial

UniQure’s 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 persists,” said Sarah Tabrizi, director of the University College London’s Huntington’s Disease Center.

AMT-130 was generally well-tolerated, with no new serious side effects reported since late 2022, the company said. UniQure plans to submit a marketing application to the US FDA in early 2026, with hopes of launching the therapy later that year if approved. About 41,000 people in the US are living with the disease, and more than 200,000 are at risk of inheriting it, according to Huntington’s disease Society of America.

(This is a Reuters's story)