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

STAT PlusOpinionAdam’s Biotech Scorecard 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. Manage alerts for this article Email this article Share this article By Adam FeuersteinSept.

10, 2026 Senior Writer, Biotech Adam Feuerstein[email protected]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.Advertisement 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.

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2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, Pharmaceuticals, rare diseases, STAT+ Submit a correction requestReprints Adam Feuerstein Senior Writer, Biotech 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. STAT+ Newsletter Senior writer Adam Feuerstein's unfiltered, uncompromising analysis on the biotech world Recommended Adam's Biotech Scorecard September 10, 2026 STAT Plus: Roivant is one of the savviest dealmakers in biotech. It also has a secret weapon Adam's Biotech Scorecard September 3, 2026 STAT Plus: Abivax has still not been acquired.

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