uniQure (NASDAQ:QURE) entered 2026 with a dual narrative of clinical progress and regulatory complexity.
Reporting financial results for the year ended December 31, 2025, the gene therapy pioneer confirmed it holds $622.5 million in cash and investments.
This significant liquidity, extending into the second half of 2029, is critical as the company prepares for an expanded clinical trial program following recent feedback from the U.S. FDA.
The company’s lead program, AMT-130 for Huntington’s disease, recently delivered 36-month data showing a 75% slowing of disease progression compared to a natural history cohort.
Despite these results, the FDA notified uniQure following a January 2026 meeting that existing Phase I/II data—which relied on external controls—would not suffice for a marketing application.
The agency has strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study to provide primary evidence of effectiveness.
In its broader pipeline, uniQure presented updated data for AMT-191 in Fabry disease, showing durable and dose-dependent increases in enzyme activity.
However, the company has implemented a protocol-defined dosing pause in its mid- and high-dose cohorts following two asymptomatic Grade 3 liver enzyme elevations.