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FDA Reverses Course on UniQure Huntington's Gene Therapy, Clearing Path to U.S. Approval Filing

6/21/2026

Food and Drug Administration has reversed its opposition to a closely watched gene therapy for Huntington's disease developed by biotech company UniQure, clearing a direct path for UniQure to file for U.S.

The agency's turnaround represents a meaningful inflection for a disease community that has watched therapeutic candidates stall or fail, and positions UniQure for a pivotal regulatory moment in the American market.

Filing Window for UniQure The reversal is significant precisely because the FDA had previously blocked the path.

Regulatory opposition — not just a lengthy review — is the harder obstacle; removing it shifts the question from "if" to "when" UniQure submits its filing.

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