UniQure’s AMT-130 Huntington’s gene therapy came under renewed scrutiny after the company reported additional follow-up data suggesting the magnitude of benefit has waned compared with results disclosed a year earlier. The update is likely to affect how regulators assess durability as the FDA begins its marketing application review. In the newer analysis, UniQure said high-dose AMT-130 slowed disease progression by 44% versus matched participants from an external natural history study, but the difference was not statistically significant. Analysts had focused on a four-year check-in that is critical for durability judgments. The market response reflected concerns that a weaker-than-expected durability profile could complicate the overall benefit-risk narrative for a one-time implanted therapy. For gene therapy developers, the episode reinforces how long-term endpoint durability and statistical robustness can become decisive even when earlier readouts showed clinical momentum.