It's rare that clinical trial findings are as exciting as what biotech company UniQure has just announced. The company is based in Amsterdam and Lexington, Mass.
Early results, after three years of a clinical trial conducted in the US, UK, and Europe, suggest that the treatment, for now called AMT-130, significantly slows the progression of Huntington's Disease (HD). And although when I originally wrote this post in September it looked like the treatment could be available in the US as early as 2026, since then what is left of the Food and Drug Administration has cast doubt on the significance of the trial results, according to a Uniqure news release. But the treatment will be available elsewhere, and in the US when experts return to the agency, eventually.
A Variation on the Gene Therapy Theme: MicroRNAs
The strategy uses microRNA technology to stamp tiny pieces of RNA onto the mRNA molecules that carry the information in the overextended gene behind HD, which encodes a protein called huntingtin. The oversized protein gums up parts of the brain that are critical for movement.
To continue reading, go to DNA Science, where this post first appeared.