UniQure's Gene Therapy Slows Huntington's

UniQure's experimental gene therapy, AMT-130, has demonstrated a 75% reduction in the progression of Huntington s disease over 36 months in an early-to-mid stage study. This groundbreaking development, announced by UniQure, has been met with optimism from the medical community, including experts like Sarah Tabrizi of University College London and Walid Abi-Saab, chief medical officer of UniQure. The therapy, administered via direct injection into the brain, was generally well-tolerated. UniQure plans to submit a marketing application to the United States — Food and Drug Administration in early 2026, with a potential launch later that year. This news has led to a significant surge in UniQure's stock price, reflecting the high market anticipation for a treatment for Huntington s disease, which currently has no FDA-approved cure. Organizations like the Huntington s Disease Society of America, represented by Judging Amy, have expressed immense hope for the approximately 42,000 Americans living with the disease.