First Successful Gene Therapy for Huntington's Disease Slows Disease Progression by 75%

image via Medical Daily

September 24, 2025, 4:06 PM

• •AMT-130 is administered via a one-time neurosurgical procedure.
• •High-dose patients saw 75% slower disease progression.
• •Secondary outcomes showed a 60% slower decline in functional abilities.
• •uniQure plans to submit a BLA to the FDA in early 2026.
• •FDA granted AMT-130 Breakthrough Therapy designation

The gene therapy AMT-130, delivered via a one-time neurosurgical procedure, demonstrated significant benefits for Huntington's disease patients. High-dose patients showed a 75% slower disease progression over 36 months compared to external controls, with a 60% slower decline in functional abilities. The treatment was generally well-tolerated, prompting uniQure to plan a Biologics License Application to the FDA in early 2026. This marks a significant advancement in treating a devastating disease, with the FDA granting Breakthrough Therapy designation.

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