The FDA escalated scrutiny of uniQure’s AMT‑130 Huntington’s disease gene therapy by urging a prospective, randomized, double‑blind, sham‑surgery‑controlled trial, prompting fierce public dispute and sharp market moves. STAT reported the agency’s retreat from traditional public advisory meetings in controversial cases; Endpoints/StockWatch documented volatile uniQure share swings as investors digested the agency’s position and leadership turbulence. The demand for sham‑controlled evidence has immediate implications for trial design, regulatory timelines, and investor expectations for intracerebral gene therapies.