uniQure has reported fresh four-year data for its Huntington’s disease gene therapy AMT-130, saying the treatment continues to slow disease progression in a small group of patients.

The company said 12 high-dose patients showed a 44 per cent slowing on the composite cUHDRS scale, though that result was not statistically significant. On the Total Functional Capacity measure, slowing was 61 per cent with a nominal p-value of 0.008.

The data come from ongoing Phase 1/2 studies and have not been peer-reviewed. With only a dozen high-dose patients, experts caution that small studies can produce results that do not hold up in larger trials.

uniQure has submitted marketing applications to regulators, seeking approval for the one-time gene therapy. Huntington’s is a devastating inherited disorder with no disease-modifying treatment.

Patient groups welcomed the continued follow-up while urging caution. Regulators will now weigh whether the data are strong enough to support approval. The company said longer follow-up of the treated patients is planned.