Breakthrough in Huntington's Treatment: FDA Paves Way for First Gene Therapy Approval

Lexington, Wednesday, 17 June 2026.
The FDA has endorsed uniQure’s plan to submit a Biologics License Application for AMT-130, a pioneering gene therapy for Huntington’s disease, by Q3 2026. This marks a historic milestone as AMT-130 could become the first gene therapy approved for this devastating neurodegenerative disorder. The FDA’s support is based on 3-year Phase I/II trial data, showing unprecedented stability in patients over four years—defying typical disease progression. With no existing treatments to delay Huntington’s, this therapy offers new hope to 75,000 patients in the U.S. and EU. Investors are already responding, with uniQure’s stock surging on the news, signaling strong confidence in its commercial potential.