UniQure’s Huntington’s gene therapy shows 75% disease slowdown, eyes FDA approval

UniQure will seek FDA approval in 2026 for its gene therapy AMT-130 after new trial data showed a 75% reduction in disease progression for 12 Huntington’s patients over three years.

Why it matters: This could become the first gene therapy for Huntington’s, a fatal, currently untreatable neurodegenerative disorder, offering hope of significantly extended quality of life. This same hope has also extended to UniQure´s stock price, as it has tripled since the news.

Backstory: UniQure, a gene therapy pioneer, has endured setbacks, developing but later abandoning Glybera, the first gene therapy approved in Europe. Its current lead program, AMT-130, targets the mutant huntingtin protein causing Huntington’s disease. Earlier trial data were underwhelming, but new long-term results flipped expectations.

Zoom in: High-dose AMT-130 patients showed 75% slower progression vs. external controls and a 60% decrease in the speed of the decline of total functional capacity. Given these positive results, UniQure is expected to file with the FDA in 2026, supported by the new $125M credit line from Hercules Capital for future development.

Big picture: The gene therapy field faces FDA scrutiny under new leadership, making regulatory approval less predictable. However, AMT-130 has received two FDA designations that could accelerate its path.