UniQure’s gene therapy still slows Huntington’s disease after four years, but benefit declines

UniQure reported that its one‑time gene therapy, AMT‑130, continues to decelerate the progression of Huntington’s disease four years after being surgically delivered into patients' brains.

A new analysis shows that while the treatment remains effective, the size of the therapeutic benefit has lessened compared with a similar evaluation performed a year earlier.

The reduced magnitude of effect is prompting questions about the durability of the therapy just as the U.S. Food and Drug Administration begins its review of UniQure’s marketing application.