A gene therapy intended as a one-time treatment for Huntington’s disease significantly slowed progression of the disorder in a pivotal Phase 1/2 trial, paving the way for a regulatory submission in the first quarter of 2026. The developer, uniQure NV, showed that it is possible to safely impact the disease by targeting both the disease-causing huntingtin protein and the normal protein using a vector-based gene therapy and a gene encoding a microRNA (miRNA).
uniQure announced top-line results from the trial on 24 September. They showed that the therapy, AMT-130, administered at a high dose, was able to achieve a 75% disease slowing at 36 months as measured by a Huntington’s disease rating scale and compared with a score-matched external control. The trial enrolled 29 patients.
“I believe these ground-breaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease where an urgent need persists,” said Sarah Tabrizi, director of the University College London Huntington’s Disease Center, in a prepared statement.
Huntington’s disease is a rare, inherited neurodegenerative disorder that can cause chorea, behavioural abnormalities and cognitive decline, resulting in a progressive physical and mental deterioration. Mutations in the huntingtin gene lead to the production and aggregation of abnormal proteins in the brain. AMT-130 has been designed to carry and deliver a gene encoding a miRNA that will recognise, bind and non-selectively lower both the mutant and the normal huntingtin proteins. The therapy is administered to patients by way of a neurosurgical procedure.
Based in Amsterdam, the Netherlands, and Lexington, Mass, US, uniQure has an approved gene therapy on the market for haemophilia B and a pipeline of candidate therapies for refractory temporal lobe epilepsy, amyotrophic lateral sclerosis, and Fabry disease.
Separately, the company also announced plans on 24 September to raise $200 million from a public share offering.
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