UniQure said the FDA no longer agrees that its Phase I/II data for AMT-130—an experimental one-time gene therapy for Huntington’s disease—are adequate to support a Biologics License Application. The company reported that agency feedback at a recent pre-BLA meeting reversed earlier guidance and left the timing of any U.S. filing unclear. UniQure disclosed that high-dose AMT-130 appeared to slow disease progression in a small cohort, but FDA officials questioned the strength of the evidence from a single-arm study using an external control. The company said it will seek clarification from the agency and expects formal meeting minutes within 30 days. Investors reacted sharply: the stock plunged more than 60% on the announcement, underscoring how regulatory pivots can swiftly erase market value for gene therapy developers.