UniQure AMT-130 Huntington's Gene Therapy Shows Durable Disease Slowdown at Four Years

UniQure's AMT-130, an AAV5-delivered miRNA gene therapy targeting Huntingtin mRNA in the striatum, continues to slow Huntington's disease progression at the four-year mark — the longest durability data yet reported for any Huntington's gene therapy. The results reinforce AMT-130's clinical potential and support its ongoing Phase I/II trial as UniQure advances toward a pivotal program.