STAT+: After promising early data, gene therapy trial for Huntington’s takes puzzling turn
Last year, after a string of high-profile trial failures, the Huntington’s disease community got positive news in the form of very early data from an experimental gene therapy for the rare neurodegenerative condition.
On Wednesday, however, the company behind the drug released updated figures that were more mixed — even befuddling.
The study from Uniqure, a Dutch biotech, randomized early stage Huntington’s patients to receive low-dose treatment, a high-dose treatment, or a sham injection. The one-time treatment consists of small strands of RNA, delivered via a benign virus injected into the brain, that are designed to block mHTT, the mutant gene that drives the disease.
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