The FDA has told uniQure that existing single-arm data for its Huntington’s gene therapy AMT‑130 are insufficient and has strongly recommended a randomized, double‑blind, sham surgery–controlled trial before a marketing application can be accepted. Company statements and regulatory summaries indicate the requirement could add two to three years to development and carries ethical and operational complexity. A sham‑surgery trial uses a blinded surgical control to isolate treatment effect; regulators sometimes demand this design for invasive, one‑time interventions when external controls are judged inadequate. Investors reacted sharply to the news, deepening scrutiny of approval pathways for high‑risk gene editing and gene‑delivery programs.