STAT+: UniQure’s gene therapy continues to slow Huntington’s progression after four years

UniQure’s experimental gene therapy for Huntington’s disease, AMT-130, continued to slow progression of the neurodegenerative disease four years after it was surgically implanted into patients’ brains, the company reported Tuesday. The update matters because it comes as the FDA begins reviewing UniQure’s marketing application, so the durability of the treatment’s effect is central to its regulatory prospects.

In the new analysis, a high dose of the one-time gene therapy slowed Huntington’s progression by 44% compared with matched participants in an external control group drawn from a large natural history study. However, that difference was not statistically significant.

The magnitude of the treatment’s benefit has also waned compared with a similar analysis conducted one year earlier, which may raise questions about whether AMT-130’s effect is durable. The combination of a continued but smaller apparent benefit and the lack of statistical significance could complicate the ongoing FDA review.