Huntington's Gene Therapy Slows Disease by 75% in 3-Year Trial
Huntington's disease gene therapy showed a major milestone this week. A one-time treatment called AMT-130 slowed measured progression by about 75 percent at three years in a high dose group, according to new trial data. Below is a clear breakdown of what happened, how it works, and what comes next.
What happened
Researchers led by Prof. Sarah Tabrizi at University College London reported that uniQure’s gene therapy AMT-130 slowed clinical decline in Huntington’s over 36 months. The company’s topline readout says the pivotal Phase I/II study met its primary endpoint on the composite Unified Huntington’s Disease Rating Scale, with a 75 percent slowing in the high dose group versus a matched external control dataset. Independent newsrooms reported the same headline result and noted the treatment involves a long neurosurgical infusion into the striatum.
- Company release with detailed stats and endpoints. Read the topline.
- Independent coverage confirms the 75 percent slowing and surgical details. Reuters and The Guardian.
How the therapy works
The surgery is a single session that slowly infuses the vector through micro-catheters into two brain regions. Reports place procedure time about 12 to 20 hours for careful, stepwise delivery. Source.
uniQure describes AMT-130 as an AAV5 vector carrying an engineered microRNA that silences the huntingtin gene. Program page.
Key results
The pivotal Phase I/II program pooled U.S. and European cohorts and compared treated patients to propensity score-matched natural-history controls. Twelve high-dose patients at 36 months drove the primary outcome.
| Measure | High dose AMT-130 (36 mo) | Matched external control | Reported effect |
|---|---|---|---|
| cUHDRS change | -0.38 | -1.52 | 75% slowing, p=0.003 |
| Total Functional Capacity | -0.36 | -0.88 | 60% slowing, p=0.033 |
| CSF NfL | Below baseline at 36 mo | — | Supportive biomarker |
Source with full topline bullet points: uniQure press release. Read more. Independent coverage: Reuters, The Guardian, expert explainer HDBuzz.
What the primary scale means
Limits and open questions
- Dataset size: The 36-month high-dose readout covers 12 treated patients compared to matched controls. Larger confirmatory data will matter for regulators. Topline.
- Study design: Outcomes were compared to an external control from Enroll-HD rather than a concurrent placebo over three years. This is acceptable in rare diseases but leaves room for debate. Details.
- Procedure burden: Infusion takes many hours in the operating room and requires specialized centers. Report.
- Safety and durability: Serious risks have been manageable to date and common effects were related to the procedure, which resolved, but lifetime durability and rare events need continued follow-up. Reuters.
Availability timeline
uniQure plans to file with the U.S. FDA in the first quarter of 2026. If approved, a U.S. launch could follow later that year with limited center rollout. Europe would likely trail. Pricing and coverage are not yet known. Company guidance.
FAQs
Is this a cure for Huntington’s?
No. The data show slowed progression over three years in a high dose group. It is a major step, not a cure. Longer follow-up and broader access will tell us more. Reuters.
How is AMT-130 given?
Through a single neurosurgical infusion into the caudate and putamen with MRI guidance and slow convection-enhanced delivery. Reports place the operating time around 12 to 20 hours. The Guardian.
What are the main risks so far?
Most issues have related to the procedure and resolved. No new drug-related serious adverse events were reported since late 2022 in this dataset, according to the company. Topline.
When could patients realistically access it?
If regulators accept the filing and approve in 2026, early access would likely be through a small number of specialized centers first. Coverage and cost will shape real-world adoption. Company guidance.
Sources
- uniQure topline Phase I/II readout for AMT-130. GlobeNewswire.
- Independent news coverage of the results. Reuters and The Guardian.
- Program background on AAV5 microRNA huntingtin lowering. uniQure program. Research context: PMC review.
- Lay explainer of the new data. HDBuzz.
Conclusion
Today’s data readout is a real inflection point. A one-time gene therapy that slows Huntington’s across multiple measures is the kind of clinical signal families have waited for. It is early, complex, and not yet approved. I will keep tracking how regulators receive the filing and how access might roll out.