Biotech company uniQure reported on 24 September that its experimental gene therapy AMT-130 produced a statistically significant 75% slowing of Huntington’s disease progression at 36 months in patients who received the high dose. These are topline results announced by the company, not yet peer reviewed, and the treatment is not approved anywhere. Still, for a fatal inherited brain disorder with no disease-modifying treatment, this is the first time any therapy has been reported to meaningfully slow it.
What happened
uniQure released results from its pivotal Phase I/II study of AMT-130. According to the company, 29 patients were treated: 17 at a high dose and 12 at a low dose. Of those, 12 patients in each dose group had reached 36 months of follow-up, and the headline numbers come from that three-year mark.
The main measure was the cUHDRS, a clinical rating scale used in Huntington’s research. On it, the high dose showed a “statistically significant 75% disease slowing at 36 months”, with a p-value of 0.003. On a second measure, Total Functional Capacity, the company reported a “statistically significant 60% slowing of disease progression” (p=0.033).
There was also a biological signal. Neurofilament light in the cerebrospinal fluid, a marker of nerve damage, showed a “mean reduction from baseline … of -8.2%”. uniQure said it plans to submit a BLA, a formal application for regulatory approval, in the first quarter of 2026.
How it works
Huntington’s is caused by a faulty gene that a person inherits, and the damage it does to brain cells builds up over years. That slow, steady decline is what the trial measured. A gene therapy aims to act on the root cause inside the cells rather than only easing symptoms, and AMT-130 is designed as a one-time treatment.
The phrase “75% slowing” is easy to misread, so here is a simple way to picture it. Imagine two walkers heading down the same long hill. One walks at the usual pace. The other still walks downhill, but covers only a quarter of the distance in the same time. Both are still descending. That is what slowing means here: the disease still progresses, just more slowly on this scale. It does not mean the disease stopped or reversed.
The other detail that matters is the comparison. The treated patients were not compared with a placebo group in the same trial. They were compared with an external natural history control, meaning data on how the disease usually progresses in people who did not receive the therapy. That approach is common in rare, severe diseases, but it is a weaker comparison than a randomized placebo-controlled trial, and readers should keep it in mind.
By the numbers
| Item | Figure | Source |
|---|---|---|
| Slowing on cUHDRS, high dose, 36 months | 75% (p=0.003) | uniQure, 24 Sep 2025 |
| Slowing on Total Functional Capacity | 60% (p=0.033) | uniQure, 24 Sep 2025 |
| Patients treated | 29 (17 high dose, 12 low dose) | uniQure, 24 Sep 2025 |
| Patients with 36 months of follow-up | 12 per dose group | uniQure, 24 Sep 2025 |
| Change in CSF neurofilament light | Mean of -8.2% from baseline | uniQure, 24 Sep 2025 |
| Planned BLA submission | First quarter of 2026 | uniQure, 24 Sep 2025 |
Why it matters
What caught my attention first was not the 75% figure itself but the word “first”. Huntington’s families have lived for generations with a diagnosis that comes with no treatment able to change its course. A reported slowing of this size, at three years, on two separate measures, is a different kind of headline from what this field has seen before.
The neurofilament result also matters in my view. Clinical scales depend partly on how patients perform on tests, while a fluid marker of nerve damage gives a more biological reading. Seeing both move in the same direction makes the story more coherent, even if an 8.2% average reduction is modest on its own.
At the same time, the limits are real. The data are topline and company reported, not yet peer reviewed. The groups are small, with 12 patients per dose at 36 months. And the comparison is with an external natural history control, not placebo. None of this cancels the result, but it is why careful scientists will want the full dataset. Nothing here is medical advice, and anyone affected should talk to their own specialist.
What comes next
The next step uniQure has named is regulatory: a BLA submission planned for the first quarter of 2026. Until regulators review the full data, AMT-130 remains an experimental therapy. I will be watching for the complete results and for how independent experts read the natural history comparison.
Sources
- uniQure via GlobeNewswire, “uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease”, 24 September 2025, https://www.globenewswire.com/news-release/2025/09/24/3155348/0/en/uniQure-Announces-Positive-Topline-Results-from-Pivotal-Phase-I-II-Study-of-AMT-130-in-Patients-with-Huntington-s-Disease.html
- uniQure investor relations, same topline results announcement, 24 September 2025, https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-topline-results-pivotal-phase-iii
- Huntington’s Disease Society of America, PDF copy of the uniQure topline results release, 24 September 2025, https://hdsa.org/wp-content/uploads/2025/09/uniQure-Announces-Positive-Topline-Results-from-Pivotal-Phase-I_II-Study-of-AMT-130-in-Patients-with-Huntingtons-Disease.pdf