The US Food and Drug Administration (FDA) has indicated it will not license a gene therapy for Huntington's disease.
UniQure's AMT-130 microRNA treatment was fast-tracked for licence review in April, and the Phase I/II clinical trial results released in September appeared to show that the treatment can slow the progression of Huntington's disease (see BioNews 1308). However, these results are now deemed insufficient for the FDA to review a licence application.
Read more at Fierce Biotech.
Related Articles
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
First evidence that gene therapy can slow Huntington's disease
A microRNA treatment is the first therapy to show evidence of slowing Huntington's disease progression....