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

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 https://www.statnews.com/2026/09/29/uniqure-huntingtons-gene-therapy-slows-disease-progression-four-years/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-9u8GCVduAdt-avf7m3Kw1QbeXiCMqIrbuy2DSY82drp-okseb1VwLj82pHtTRXZd8R4Ihh7blfLBz8WQZZXWXAinQadA&_hsmi=441553206&utm_content=441553206&utm_source=hs_email By Adam FeuersteinSept. 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. UniQure's Huntington's benefit is less pronounced four years in UniQure’s experimental Huntington’s gene therapy AMT-130 continued to slow disease progression four years after treatment. But the magnitude of the benefit declined from a year earlier — which could potentially complicate the therapy’s regulatory review, STAT’S Adam Feuerstein writes. Among 12 patients given a high dose, AMT-130 slowed progression by 44% compared with an external natural history control group, a difference that was not statistically significant. At three years, however, the company had reported a slowing of 75%, which was statistically significant. This decrease in efficacy can partly be attributed to faster-progressing patients in the historical control group dropping out of the study, UniQure CEO Matthew Kapusta said. Still, other measures were more encouraging: AMT-130 maintained a 61% slowing in loss of functional capacity, while levels of NfL, a marker of neuronal damage, stayed close to the baseline.