uniQure Breakthrough Designation to AMT-130 for Huntington’s

uniQure (NASDAQ: QURE) has received FDA Breakthrough Therapy designation for AMT-130, its gene therapy treatment for Huntington's disease, a rare inherited neurodegenerative disorder with no current disease-modifying therapies.

The designation is based on promising interim data from Phase I/II trials showing dose-dependent slowing of disease progression. The company presented 24-month data in July 2024, demonstrating meaningful disease progression slowdown based on cUHDRS scores compared to natural history.

AMT-130 has already received Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations. The treatment has been administered to 45 patients to date, with additional regulatory guidance on the Biologics License Application expected in Q2 2025.

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