~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License ...

~ FDA agrees that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval ~ As part of uniQure’s Regenerative Medicine ...

Management reiterated the expectation for top line pivotal data from AMT-130 and initial Fabry data in September, with a planned BLA submission for AMT-130 in the first quarter of 2026. The team ...