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UniQure to seek FDA approval for Huntington's gene therapy after regulatory reversal

Market News
17 Jun 2026
CNBC
View Source
Bullish
pluang ai news

UniQure announced plans to submit its gene therapy for Huntington's disease to the FDA for approval in Q3, following a recent FDA meeting that supported accelerated approval based on a three-year Phase 1/2 study. This marks a reversal from earlier FDA criticism under former Commissioner Marty Makary, who had questioned the trial data and demanded a placebo-controlled study. UniQure's therapy, which slows disease progression by 75%, was previously challenged due to ethical concerns about sham brain surgery controls. The FDA now agrees to an accelerated approval pathway with a commitment from UniQure to conduct a follow-up study comparing the therapy to standard care. Shares surged 70% on the news, highlighting renewed investor confidence.

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