In a first, experimental gene therapy cuts Huntington's progression by 75%

A Phase I/II trial of AMT-130 reports unprecedented results, with patients showing a 75 per cent slowdown in Huntington's disease progression over three years

For decades, Huntington’s disease has been seen as unstoppable, a genetic fate with no way to slow its decline. Now, a new Phase I/II study of AMT-130 brings unprecedented hope. According to findings announced in uniQure’s topline report (September 2025), patients receiving the high-dose gene therapy experienced a 75 per cent slower disease progression, offering what experts call the strongest evidence yet of a disease-modifying treatment.  Researchers believe this could mark the beginning of a new treatment era for Huntington’s.

What is Huntington’s disease?

Huntington’s is a rare, inherited brain disorder caused by a faulty gene that produces a toxic protein, gradually damaging brain cells. Symptoms usually appear between the ages of 35 and 55 and include involuntary movements, memory decline, mood swings, and eventual loss of independence. Current treatments can only manage symptoms; none can slow progression.

 

How does the new gene therapy work?

The treatment, AMT-130, uses a harmless virus to deliver genetic instructions directly into the brain. These instructions trigger cells to produce microRNAs — molecules that “silence” the faulty Huntington gene and reduce production of its toxic protein.

 

The procedure involves MRI-guided neurosurgery, where doctors infuse the therapy into the brain’s striatum, one of the first regions affected by the disease. Once delivered, the microRNAs continue working inside the cells, potentially offering long-lasting protection.

What did the study reveal?

The Phase I/II trial included 29 patients with early Huntington’s. Among those given a high dose of AMT-130: 

• Disease progression slowed by 75 per cent 
• Daily function decline slowed by 60 per cent 
• Cognitive test scores improved significantly 
• A biomarker of brain damage fell 8.2 per cent from baseline 

AMT-130 was generally well tolerated, with side effects mostly linked to the surgical procedure and no new serious drug-related events.

What do experts say about the results?

Dr Sarah Tabrizi, professor of clinical neurology at University College London and lead investigator, called the findings “the most convincing in the field to date”. She said, “These data indicate that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.”

When could the therapy be available?

uniQure plans to submit a Biologics License Application (BLA) to the US FDA in early 2026, aiming for a launch later that year if approved. The therapy has already received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations, which may speed up review.

What does this mean for families?

If approved, AMT-130 could become the first disease-modifying treatment for Huntington’s. Instead of merely treating symptoms, it may slow the disease itself, potentially giving patients extra years of independence and better quality of life.

 

Experts, however, caution that while results are promising, the trial was small. More data will be needed to confirm long-term safety and effectiveness.

What’s next for Huntington’s research?

Beyond gene therapy, scientists are also exploring Crispr-based gene editing as a possible future cure. For now, AMT-130 represents a major leap forward, transforming hope into tangible progress.

 

As uniQure’s Chief Medical Officer, Dr Walid Abi-Saab put it, “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease.” 

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