UniQure said it received formal FDA meeting minutes indicating agency staff view its current dataset for AMT‑130, a Huntington’s gene‑silencing therapy, as likely insufficient for approval. The company confirmed it will seek an urgent follow‑up meeting and assess next steps; its shares dropped sharply on the news. AMT‑130 uses an AAV vector to deliver genetic material designed to lower mutant huntingtin protein levels. Mid‑stage results had shown a large effect size in a small cohort, prompting UniQure to plan an early regulatory filing, but FDA concerns center on trial size, durability, and generalizability of the data. UniQure now faces scenarios including expanded enrollment, longer follow‑up, or a new trial; the company said it will press for clarification with regulators in Q1. The setback underscores regulatory uncertainty for complex CNS gene therapies.