UniQure's Huntington's gene therapy shows sustained but narrowing benefit at four years
Updated data from UniQure's AMT-130 programme show continued slowing of Huntington's disease progression four years post-treatment, though the magnitude of effect has diminished compared with earlier readouts.
UniQure has released updated long-term data for AMT-130, its investigational gene therapy for Huntington's disease, showing that treated patients continue to show slower disease progression relative to untreated comparators at the four-year mark. The therapy uses an adeno-associated virus to deliver a microRNA targeting the huntingtin (HTT) gene, aiming to reduce levels of the mutant HTT protein that drives neurodegeneration.
According to reporting by STAT News, whilst the directional benefit is maintained, the magnitude of the effect appears smaller than data presented at the three-year timepoint. UniQure has not yet offered a full mechanistic explanation for the attenuation; possibilities under discussion in the field include waning transgene expression, disease progression outpacing the therapy's capacity to slow it at later stages, or regression-to-the-mean artefacts from the non-randomised comparison cohort.
AMT-130 is currently in a Phase I/II trial. Huntington's disease is a dominantly inherited, progressive neurodegenerative condition caused by a CAG repeat expansion in HTT; there are currently no approved disease-modifying treatments. The four-year data will be important context for any future regulatory submission, and the field will be watching whether the effect plateau stabilises or continues to narrow with longer follow-up.
This story was published by STAT News on 29 September 2026. The underlying trial data have not yet appeared in peer-reviewed form; this summary is based on conference or company reporting as covered in the press.
Plain-language version
For patients, families, and general readers. Educational only — not medical advice.
Huntington's disease is a hereditary condition that gradually affects movement, thinking, and behaviour. It is caused by a change in a gene called HTT that is passed down in families. There is currently no treatment that can stop or reverse the disease.
A company called UniQure has been testing a gene therapy called AMT-130 that aims to reduce the harmful effects of the faulty gene. New information released after four years of follow-up suggests that people who received the treatment continued to show slower progression of symptoms compared with those who did not, which is encouraging. However, the size of that benefit appears to be somewhat smaller than data reported a year earlier, and researchers are still working to understand why.
The treatment is still being studied in a clinical trial and has not yet been approved by any medicines regulator. Scientists and clinicians will continue to monitor participants to understand whether the effect remains stable over time.
This is an educational summary, not medical advice. If anything here raises questions for you, please speak with your GP or a clinical professional.
Sources
Read the original reporting — these are the public sources this summary draws from.
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Primary source Stat News · 2026-09-29STAT+: UniQure's gene therapy continues to slow Huntington's progression after four years