FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease
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Posted on: 6 March 2026, source: CNBC UniQure needs to run another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said on a call with reporters Thursday. The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of st