Gene Therapy Slows Huntington’s Disease Progression for First Time

In a breakthrough clinical trial, scientists have shown that a single injection of a gene therapy named AMT-130 can slow the progression of Huntington’s disease by as much as 75%.

The trial involved 29 patients, compared against a historical control group from a long-term study. Among those who received the higher dose and were followed for three years, disease worsening was reduced by 75% relative to the control group. Biomarker measurements also indicated less nerve damage.

AMT-130 was well tolerated, with no major safety concerns reported. The treatment requires a one-time neurosurgical delivery to a targeted brain region.

Regulatory approval is still pending, though developers plan to seek accelerated approval in the U.S. first, then Europe and the U.K. This marks the first time a therapy has shown durable disease-modifying effects in Huntington’s disease in humans.

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