uniQure’s experimental Huntington’s gene therapy AMT-130 remains at the center of an escalating regulatory standoff with the U.S. Food and Drug Administration. The FDA signaled that data from uniQure’s earlier Phase I/II external-control study are insufficient and issued a strong recommendation for a prospective, randomized, double-blind, sham-surgery-controlled trial. The agency’s public stance triggered extreme share volatility as investors reassessed approval risk and commercial forecasts. Events accelerated when the FDA’s chief medical and scientific officer at CBER, Vinay Prasad, announced he will depart at the end of April. Prasad’s tenure included high-profile rejections and reversals on gene-therapy and vaccine reviews, and his exit adds uncertainty to the agency’s review process for advanced biologics. Companies and investors are recalibrating development plans for intracranial gene therapies and weighing the operational and ethical implications of sham-surgery controls in neurosurgical trials.