STAT+: UniQure’s gene therapy continues to slow Huntington’s progression after four years

But the magnitude of the treatment’s benefit waned compared to a similar analysis from a year ago Sipa via AP Images Sept. 29, 2026 Adam Feuerstein, a senior writer and biotech columnist, is the author of Adam’s Biotech Scorecard, a subscriber-only newsletter about the crossroads of drug development, business, Wall Street, and biotechnology. Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54. An experimental gene therapy for Huntington’s disease, developed by the biotech company UniQure, continued to slow the progression of the neurodegenerative disease four years after it was surgically implanted into the brains of patients, the company reported Tuesday. However, the magnitude of the treatment’s benefit has waned compared to a similar analysis conducted one year ago, which may raise questions about its durability just as the Food and Drug Administration begins to review UniQure’s marketing application. In the new analysis, a high dose of UniQure’s one-time gene therapy called AMT-130 slowed the progression of Huntington’s by 44% compared to matched participants in an external control group based on a large, natural history study. The difference was not statistically significant. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.