STAT+: Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial

Groundbreaking Data Positions Uniqure’s AMT-130 as Potential Pioneer Genetic Treatment for Huntington’s Disease

An innovative gene therapy from Uniqure has demonstrated a remarkable 75% reduction in the progression of Huntington’s disease over three years, as disclosed in a recent study. This significant finding could facilitate the first approval of a genetic treatment for this rare neurodegenerative disorder.

For those affected by Huntington’s disease, a potent, one-time treatment that considerably slows the deterioration of motor skills and cognitive function during mid-life could essentially safeguard valuable years of meaningful relationships and professional opportunities that would typically be forfeited due to the illness.

In an extensive three-year study, Huntington’s patients who received the high dosage of Uniqure’s gene therapy, known as AMT-130, exhibited an average decline of just 0.38 points on the cUHDRS scale, a measure of disease progression. In contrast, individuals from an external control group lost an average of 1.52 points, based on a large natural history study. The benefits seen with AMT-130 were statistically significant and aligned with the primary objectives of the research.