UniQure said the timetable to submit AMT‑130, its in vivo gene therapy for Huntington’s disease, is now unclear after a recent FDA meeting. Regulators signaled that existing Phase 1/2 data using an external control may not meet standards for an approval filing, forcing the company to consider additional studies or longer follow‑up. UniQure disclosed the change publicly and shares dropped sharply as the company weighed next steps. The development follows prior communications in which UniQure and regulators had discussed accelerated paths for AMT‑130. The company must now decide whether to generate new controlled evidence or revise its regulatory strategy. Investors and partners will watch for timelines on any supplemental trials, protocol designs, and potential capital needs. Clinical takeaways: AMT‑130 targets a high unmet-need neurodegenerative indication where regulatory precedent for gene therapies remains evolving; changes in evidentiary expectations can materially delay filings and amplify financing risk for small biotechs.