A human trial shows that synthetic strands of DNA meant to “silence” a problematic gene can slow down the production of a protein that leads to irreversible damage in the brain.
Huntington’s disease is caused by a mutation in the HTT gene that causes the body to create a rogue version of a protein called huntingtin; this altered protein damages parts of the brain. The new drug, called Ionis-HTTRx, is a small piece of synthetic DNA that blocks the production of that protein by silencing the HTT gene. The drug has to be injected into the fluid around the spine using a large needle.
Now the company behind the drug, Ionis Pharmaceuticals, reports that a phase 1 trial of 46 patients has shown that the drug successfully lowers the level of the toxic protein in the nervous system. And the results have caught the eye of Big Pharma: Roche has announced that it’s licensing the drug from Ionis for $45 million and will take on its development going forward.
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