STAT+: What to expect from UniQure’s upcoming Huntington’s gene therapy readout — SkimNews
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- UniQure is expected to report four-year results from its AMT-130 gene therapy trial for Huntington's disease before the end of September.
- The AMT-130 trial's three-year results were reported almost one year ago, establishing the benchmark for this longer-term readout.
- The high dose of AMT-130, given to 12 patients, slowed Huntington's disease progression by 75% versus matched participants in an external control group.
- The external control group was drawn from a large natural-history study rather than a randomized concurrent comparator arm — a design choice the broader field is watching closely.
Why it matters: Four-year durability is the key milestone for any one-time gene therapy. UniQure's earlier 75% slowing at three years — measured against an external natural-history control rather than a randomized arm — will now be tested as patients enter the years where untreated Huntington's typically shows steeper progression. The comparison method itself has been a focal point for skeptics.
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