The U.S. Food and Drug Administration told uniQure it expects a prospective, randomized, double‑blind, sham‑surgery‑controlled study to support marketing approval for AMT‑130, the company’s one‑time AAV‑based Huntington’s disease therapy. Regulators said data from Phase I/II compared to external controls are insufficient and recommended a controlled study design during a Type A meeting. Investigators reported Phase I/II results showing slowed disease progression, but the FDA’s requirement raises ethical and operational concerns because sham neurosurgical controls could leave participants untreated for years. Clinicians and patient advocates warn that prolonged placebo exposure may render some patients ineligible for later therapy. The agency’s stance introduces a high‑stakes design choice for irreversible, potentially life‑prolonging gene therapies and could set precedent for how surgical gene‑delivery candidates are evaluated going forward.