Gene therapy appears to slow Huntington progression
UniQure announced results from a small clinical trial of AMT-130, a one-time AAV-delivered gene therapy infused directly into the brain, reporting that 12 high-dose recipients declined 75% less on the Unified Huntington’s Disease Rating Scale over 3 years compared with a ~1,600-patient untreated comparison group; academic leaders and outside neuroscientists called the findings encouraging while noting the trial is small and not yet peer-reviewed.
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In a first, a gene therapy seems to slow Huntington disease
New information:
- UniQure’s AMT-130 was infused directly into the caudate nucleus and putamen in a one-time neurosurgical procedure.
- Twelve high-dose trial participants followed for 3 years showed a 0.38-point decline on the Unified Huntington’s Disease Rating Scale versus a 1.52-point decline in the comparison group (≈75% difference).
- The trial enrolled 29 symptomatic Huntington patients (stage 2–early stage 3) and compared outcomes to nearly 1,600 untreated patients at similar disease stages.