Uniqure 公布亨廷顿病基因疗法 AMT-130 的四年随访更新，AMT-130 相较自然病史使疾病进展减缓 44%，但不再具有统计学显著性；此前三年数据为减缓 75% 且具统计学显著性。接受治疗的患者在第 3 至第 4 年进展加快，而自然病史患者趋于稳定，Uniqure 对此给出了解释。该更新发布之际，FDA 正开始对 AMT-130 进行审评。
AMT-130 四年随访疗效从统计学显著转为不显著，读者可据此理解基因治疗持久性争议与 FDA 审评时点的关联。
$QURE Huntington's gene therapy update issued. The new data will likely spark debate about durability, just as the FDA begins its review.
At 4 yrs, AMT-130 slowed disease progression by 44% vs natural history, no longer statistically significant.
Recall, at 3 yrs, AMT-130 showed a 75% slowing of disease progresssion, stat sig.
Treated patients progressed faster from year 3 to 4, natural history patients stabilized. Why? Uniqure offers reasons.
There is a lot more data to chew on. Uniqure shared the update with me last night under embargo, and I interviewed CEO Matt Kapusta + an indpt HS expert.
My story is below:
https://www.statnews.com/2026/09/29/uniqure-huntingtons-gene-therapy-slows-disease-progression-four-years/
来源：Adam Feuerstein ✡️ · x.com