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UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency

CNBC · Jun 17, 2026, 4:12 PM · Also reported by 3 other sources

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  • Uni Qure plans to seek FDA approval of its experimental gene therapy for Huntington's disease, the company said Wednesday, months after previous agency leaders criticized the evidence backing the application.
  • As a result of the meeting, UniQure plans to submit its application to the FDA in the third quarter of this year.

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Uni Qure plans to seek FDA approval of its experimental gene therapy for Huntington's disease, the company said Wednesday, months after previous agency leaders criticized the evidence backing the application.

Uni Qure said the FDA in a recent meeting communicated that a three-year analysis from a Phase 1/2 study would support an accelerated approval of UniQure's gene therapy for Huntington's, a rare hereditary disease that gradually destroys nerve cells in the brain. As a result of the meeting, UniQure plans to submit its application to the FDA in the third quarter of this year.

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