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Gene therapy for Huntington's disease reaches FDA review
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Fusion42 · 3 November 2026 · Fusion42 review
uniQure's gene therapy ifezuntirgene inilparvovec for Huntington's disease has entered FDA Priority Review with a target decision in May 2027. This is the first potential disease-modifying treatment for Huntington's targeting the mutant HTT gene via a single administration using an AAV5 vector to silence the gene in striatal neurons.
This Wire brief sits within Fusion42's coverage of Biotech, and 4 sources have reported it between 2 Nov 2026 and 3 Nov 2026.
◆ ◆ The Wire takeaway
The FDA's Priority Review opens a rare disease market that lacked approved therapy for decades. If you build gene therapies for hard-to-treat neurodegenerative diseases, you can now sell a novel approach that targets root causes with single-dose treatments.
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4 sources · first reported 2 Nov 2026 · latest 3 Nov 2026
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