Breakthrough Gene Therapy Slows Huntington’s Disease Progression by 75%

A groundbreaking clinical trial has revealed that a new gene therapy may dramatically slow the progression of Huntington’s disease, offering long-awaited hope to thousands affected by this devastating neurological disorder. The therapy, developed by biotech company uniQure, showed a 75% reduction in disease progression over three years in patients who received high doses—marking the most promising results in Huntington’s research to date.

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🧬 What Is Huntington’s Disease?

Huntington’s disease is a rare, inherited brain disorder caused by a mutation in the huntingtin gene. It typically begins between ages 35 and 55 and leads to progressive motor dysfunction, cognitive decline, and emotional disturbances. There is currently no cure, and existing treatments only manage symptoms.

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💉 The New Therapy: AMT-130

The experimental treatment, called AMT-130, uses a harmless virus to deliver microRNA directly into the brain. This RNA sequence silences the faulty huntingtin gene, preventing the production of the toxic protein responsible for neuron death. Once injected, the virus’s instructions remain in the brain cells permanently, turning them into “mini factories” that produce their own therapeutic agent.

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📊 Trial Results

• 29 patients participated in the Phase I/II trial.
• High-dose group showed a 75% slower progression compared to untreated patients.
• Moderate-dose group saw more modest benefits.
• Improvements were measured across movement, cognition, and daily functioning.
• The therapy was well-tolerated, with no serious side effects reported.

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🧠 How It Works

The therapy targets the striatum, a brain region critical for movement and especially vulnerable to Huntington’s. While other areas like the frontal cortex are also affected by the disease, they were not directly targeted in this trial. Still, the results suggest a meaningful delay in symptom onset and progression.

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🗓️ What’s Next?

uniQure plans to submit its data to the U.S. Food and Drug Administration in early 2026. If approved, AMT-130 could become the first disease-modifying treatment for Huntington’s, potentially extending independence and quality of life for patients.

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Sources:

• WISH-TV: New gene therapy shows promise in Huntington’s disease trial
• Nature: Huntington’s disease treated for first time using gene therapy
• Oman Observer: New treatment shows promise for Huntington’s disease
• USA Today: Experimental gene therapy slows Huntington's disease progression in trial