STAT+: What to expect from UniQure’s upcoming Huntington’s gene therapy readout

The biotech company has already submitted the drug to regulators in the U.S. and U.K. Sipa via AP Images 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. This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. Before the end of September, UniQure is expected to report four-year results from the closely tracked clinical trial of AMT-130, its gene therapy for Huntington’s disease. Three-year results were reported almost one year ago. The high dose of AMT-130, administered to 12 patients, slowed the progression of Huntington’s by 75% compared to matched participants in an external control group based on a large, natural history study. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. 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.