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UniQure's Huntington's gene therapy shows dose-dependent efficacy; BLA path ahead

Sep 29, 2026, 11:55 AM EDT1 sourcesAI-analyzed

Why it may matterVerify against the original reporting

Immediate reaction reflects concerns over non-significant 48-month cUHDRS signal, offset by regulatory optimism from the BLA pathway; mixed signals create near-term volatility.

AI summary

What happened, with direct paths to the underlying reporting

UniQure released 36-month data showing dose-dependent slowing (80% cUHDRS, 67% TFC) and a 6% neurofilament decline. Four-year results were mixed: 48-month TFC slowed 61%, cUHDRS 44% but not statistically significant. FDA confirmed 36-month data from 12 high-dose patients can anchor a BLA under accelerated approval.

36-month data: high-dose shows 80% cUHDRS, 67% TFC slowing.
FDA Type B meeting: 36-month data acceptable for BLA under accelerated approval.
48-month results: 61% TFC, 44% cUHDRS slowing; cUHDRS not statistically significant.
Post-hoc analysis with earlier controls shows significant slowing.
Safety: generally manageable; one unrelated suicide case.

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