An experimental gene therapy for Huntington’s disease, developed by the biotech company UniQure, continued to slow the progression of the neurodegenerative disease four years after it was surgically implanted into the brains of patients, the company reported Tuesday.
However, the magnitude of the treatment’s benefit has waned compared to a similar analysis conducted one year ago, which may raise questions about its durability just as the Food and Drug Administration begins to review UniQure’s marketing application.
In the new analysis, a high dose of UniQure’s one-time gene therapy called AMT-130 slowed the progression of Huntington’s by 44% compared to matched participants in an external control group based on a large, natural history study. The difference was not statistically significant.
